KR930703453A - 유전자 치료법을 위한 안전한 벡터 - Google Patents

유전자 치료법을 위한 안전한 벡터

Info

Publication number
KR930703453A
KR930703453A KR1019930702050A KR930702050A KR930703453A KR 930703453 A KR930703453 A KR 930703453A KR 1019930702050 A KR1019930702050 A KR 1019930702050A KR 930702050 A KR930702050 A KR 930702050A KR 930703453 A KR930703453 A KR 930703453A
Authority
KR
South Korea
Prior art keywords
vector
aav
globin
bone marrow
cell
Prior art date
Application number
KR1019930702050A
Other languages
English (en)
Inventor
아룬 스리바스타바
Original Assignee
티모시 존 렉칼트
리서치 코포레이션 테크놀로지스, 인코포레이티드
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 티모시 존 렉칼트, 리서치 코포레이션 테크놀로지스, 인코포레이티드 filed Critical 티모시 존 렉칼트
Publication of KR930703453A publication Critical patent/KR930703453A/ko

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Obesity (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

유전자 치료요법은 새로운 유전자 정보를 세포로 옮겨 안정하게 삽입시키는 것을 의미한다. 본 발명은 유전자 치료요법을 위한 안전한 벡터에 관한 것으로, 실질적인 세포독성이 없이 포유동물의 염색체에 위치 특이적으로 삽입될수 있으며, 원하는 이원적 유전자의 세포 특이적 발현을 가능하게 하는 하이브리드 파르보바이러스 벡터를 제공한다.
이 하이브리드 벡터는 유전자 치료, 특히 혈색소병 및 기타 조혈 작용 관련 질병의 치료에, 또 세포 특이적인 다약물 대상의 전달이 유용하다. 또한 본 발명은 약히학적 산물의 전달방법 및 재조합 단백질 생상방법도 제공한다.

Description

유전자 치료법을 위한 안전한 벡터
본 내용은 요부공개 건이므로 전문내용을 수록하지 않았음
제1도는 AAV 2게놈의 ITR 뉴클레오타이드 서열을 나타낸다. 제2도는 Shade등(1986)이 정한 숫자에 의한 뉴클레오타이드 번호 200-424까지의 B19뉴클레오타이드 서열을 나타낸다. 제3도는 본 발명에 의한 하이브리드 벡터를 만드는 다이아그램이다.

Claims (44)

  1. 아데노-연관 바이러스 2의 역방향 말단 반복부(inverted terminal repeats)2개와, 이원적 유전자에 작동 가능하도록 연결된, 세포 특이적 발현을 가능하게 하는 프로모터로 이루어지는 최소한 하나의 카세트로 이루어지며, 상기 카세트가 상기 역방향 말단 반복후 사이에 위치하는, 부위 특이적 삽입(intergration)과 세포 특이적 유전자 발현을 위한 발현 벡터.
  2. 제1항에 있어서, 상기 역방향 말단 반복부가 각각 SEQ ID NO:1로 이루어지는 것을 특징으로 하는 벡터.
  3. 제1항에 있어서, 상기 역방향 말단 반복부가 각각 SEQ ID NO:1의 뉴클레오타이드 1-125로 이루어지는 것을 특징으로 하는 벡터.
  4. 제1항에 있어서, 상기 프로모터가 B19 파르보바이러스 프로모터인 것을 특징으로 하는 벡터.
  5. 제4항에 있어서, 상기 B19 파르보바이러스 프로모터가 p6 프로모터인 것을 특징으로 하는 벡터.
  6. 제4항에 있어서, 상기 B19 파르보바이러스 프로모터가 뉴클레오타이드 SEQ ID NO:2로 이루어지는 것을 특징으로 하는 벡터.
  7. 제1항에 있어서, 상기 이원적 유전자가 생물학적 기능이 있는 단백질을 지정하는 것을 특징으로 하는 벡터.
  8. 제1항에 있어서, 상기 이원적 유전자가 생물학적 기능이 없는 단백질을 지정하는 것을 특징으로 하는 벡터.
  9. 제1항에 있어서, 상기 이원적 유전자가 안티센스 RNA를 지정하는 것을 특징으로 하는 벡터.
  10. 제1항에 있어서, 상기 이원적 유전자가 α-글로빈, β-글로빈, γ-글로빈, 과립구 매그로파지-콜로니자극인자(GM-CSF), 종양 괴사인자(TNF) 및 인터루킨 1-11, 네오마이신 내성, 루시퍼레이즈, 아데닌 포스 포라이보실 전이효소(APRT), 망막아종, 인슐린, 매스트 세포 성장인자, p53을 지정하는 유전자 및 아데노신 탈아민효소를 지정하는 유전자로 구성되는 군으로부터 선택되는 것을 특징으로 하는 벡터.
  11. 제1항에 있어서, 상기 이원적 유전자가 P-당단백질인 것을 특징으로 하는 벡터.
  12. 제9항에 있어서, 상기 안티센스 RNA가 α-글로빈을 지정하는 DNA 또는 RNA의 단편인 것을 특징으로 하는 벡터.
  13. 제5항에 있어서, 상기 벡터가 AAV-B19-GM-CSF, AAV-B19-APRT, AAV-B19-Neo, AAV-B19-RB, AAV-B19-β- 글로빈, AAV-B19-α글로빈 또는 AAV-B19-γ글로빈인 것을 특징으로 하는 벡터.
  14. 제5항에 있어서, 상기 벡터가 AAV-B19-mdr인 것을 특징으로 하는 벡터.
  15. 제1항 내지 제14항중 어느 한 항에 있어서, 상기 벡터가 숙주 세포내에 들어있는 것을 특징으로 하는 벡터.
  16. 제1항 내지 제12항중 어느 한 항에 있어서, 상기 벡터가 바이러스내에 들어있는 것을 특징으로 하는 벡터.
  17. 제16항에 있어서, 상기 벡터가 숙주세포내에 들어있는 것을 특징으로 하는 벡터.
  18. 제15항 또는 제17항에 있어서, 상기 숙주세포가 조혈 스템 세포 또는 조혈 조상 세포인 것을 특징으로 하는 벡터.
  19. a)환자로부터 골수 세포를 얻고, b)이 골수세포로 제1항에 따른 벡터를 형질도입시키고, c)이 형질도입시킨 골수세포를 상기 환자에게 다시 도입시키는 것으로 이루어지는 제1항에 따른 벡터를 유전자 치료에 사용하는 방법.
  20. 제19항에 있어서, 상기 형질도입이 상기 벡터를 트랜스펙션시키는 것에 의해 상기 벡터를 포함하는 바이러스를 감염시키는 것에 의해 수행되는 것을 특징으로 하는 방법.
  21. 제19항에 있어서, 상기 유전자 치료가 낫꼴 세포 빈혈이나 당뇨병의 치료인 것을 특징으로 하는 방법.
  22. 제19항에 있어서, 상기 유전자 치료가 지중해 빈혈의 치료인 것을 특징으로 하는 방법.
  23. 제22항에 있어서, 상기 벡터가 AAV-B19-α글로빈 또는 AAV-B19-β-글로빈인 것을 특징으로 하는 방법.
  24. 제19항에 있어서, 상기 유전자 치료가 조혈 관련 질병인 것을 특징으로 하는 방법.
  25. 제24항에 있어서, 상기 벡터가 AAV-B19-GM-CSF인 것을 특징으로 하는 방법.
  26. 제25항에 있어서, 상기 상기 유전자 치료가 암의 치료인 것을 특징으로 하는 방법.
  27. 제26항에 있어서, 상기 벡터가 AAV-B19-RB인 것을 특징으로 하는 방법.
  28. 제19항에 있어서, 상기 유전자 치료가 혈색소병의 치료인 것을 특징으로 하는 방법.
  29. 제28항에 있어서, 상기 벡터가 AAV-B19-β-글로빈, AAV-B19-α-글로빈 또는 AAV-B19-γ-글로빈 것을 특징으로 하는 방법.
  30. a)포유류로부터 골수 세포를 얻고, b)이 골수세포로 제1항에 따른 벡터를 형질도입시키고, c)이 형질도입시킨 골수세포를 상기 포유류에게 다시 도입시키는 것으로 이루어지는 제1항에 따른 벡터를 이용한 약리학적 산물 전달방법.
  31. 제30항에 있어서, 상기 형질도입이 상기 벡터를 트랜스펙션시키는 것에 의해 또는 상기 벡터를 포함하는 바이러스를 감염시키는 것에 의해 수행되는 것을 특징으로 하는 방법.
  32. 제31항에 있어서, 상기 산물이 γ-글로빈, 인슐린, 매그로파지 콜로니 자극인자, 과립구 콜로니 자극인자, 적혈구 조혈소 종양 괴사인자, 매스트 세포 성장인자, 또는 인터루킨 1-11, p53중의 하나 또는 안티센스 RNA분자인 것을 특징으로 하는 방법.
  33. 제31항에 있어서, 상기 산물이 과립구 매그로파지-콜로니 집락인자인 것을 특징으로 하는 방법.
  34. 제33항에 있어서, 상기 벡터가 AAV-B19-GM-CSF인 것을 특징으로 하는 방법.
  35. 제31항에 있어서, 상기 산물이 α-글로빈인 것을 특징으로 하는 방법.
  36. 제35항에 있어서, 상기 벡터가 AAV-B19-α-글로빈인 것을 특징으로 하는 방법.
  37. 제31항에 있어서, 상기 산물이 β-글로빈인 것을 특징으로 하는 방법.
  38. 제37항에 있어서, 상기 벡터가 AAV-B19-β-글로빈인 것을 특징으로 하는 방법.
  39. 제31항에 있어서, 상기 산물이 망막아종인 것을 특징으로 하는 방법.
  40. 제39항에 있어서, 상기 벡터가 AAV-B19-RB인 것을 특징으로 하는 방법.
  41. a)환자로부터 골수 세포를 얻고, b)이 골수세포로 제11항에 따른 벡터를 형질도입시키고, c)이 형질도입시킨 골수세포를 상기 환자에게 다시 도입시키는 것으로 이루어지는 제11항에 따른 벡터를 이용하여 환자에게 세포 특이적인 다약물 내성을 부여하는 방법.
  42. a)환자로부터 골수 세포를 얻고, b)이 골수세포로 제14항에 따른 벡터를 형질도입시키고, c)이 형질도입시킨 골수세포를 상기 환자에게 다시 도입시키는 것으로 이루어지는 제14항에 따른 벡터를 이용하여 환자의 적혈구 세포에 다약물 내성을 부여하는 방법.
  43. 제41항 또는 제42항에 있어서, 상기 형질도입이 상기 벡터를 트랜스펙션시키는 것에 의해 또는 상기 벡터를 포함하는 바이러스를 감염시키는 것에 의해 수행되는 것을 특징으로 하는 방법.
  44. 제19항, 31항, 42항 또는 43항중 어느 한항에 있어서, 상기 골수세포가 형질도입되기 전에 스템세포에 대해 강화되는 것을 특징으로 하는 방법.
    ※ 참고사항 : 최초출원 내용에 의하여 공개하는 것임.
KR1019930702050A 1991-11-08 1992-11-06 유전자 치료법을 위한 안전한 벡터 KR930703453A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US07/789,917 US5252479A (en) 1991-11-08 1991-11-08 Safe vector for gene therapy
US789,917 1991-11-08
PCT/US1992/009769 WO1993009239A1 (en) 1991-11-08 1992-11-06 Adeno-associated virus-2 basal vectors

Publications (1)

Publication Number Publication Date
KR930703453A true KR930703453A (ko) 1993-11-30

Family

ID=25149101

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1019930702050A KR930703453A (ko) 1991-11-08 1992-11-06 유전자 치료법을 위한 안전한 벡터

Country Status (13)

Country Link
US (1) US5252479A (ko)
EP (1) EP0566732B1 (ko)
JP (2) JPH06504680A (ko)
KR (1) KR930703453A (ko)
AT (1) ATE236263T1 (ko)
AU (1) AU657829B2 (ko)
CA (1) CA2098483C (ko)
DE (1) DE69232983T2 (ko)
DK (1) DK0566732T3 (ko)
ES (1) ES2192555T3 (ko)
IL (1) IL103677A0 (ko)
PT (1) PT101040B (ko)
WO (1) WO1993009239A1 (ko)

Families Citing this family (525)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6605712B1 (en) * 1990-12-20 2003-08-12 Arch Development Corporation Gene transcription and ionizing radiation: methods and compositions
US6410010B1 (en) 1992-10-13 2002-06-25 Board Of Regents, The University Of Texas System Recombinant P53 adenovirus compositions
US5747469A (en) 1991-03-06 1998-05-05 Board Of Regents, The University Of Texas System Methods and compositions comprising DNA damaging agents and p53
CA2108144A1 (en) 1991-03-06 1992-09-07 Jack A. Roth Methods and compositions for the selective inhibition of gene expression
US6261834B1 (en) * 1991-11-08 2001-07-17 Research Corporation Technologies, Inc. Vector for gene therapy
US5587308A (en) * 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6268213B1 (en) * 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
JPH08504095A (ja) 1992-09-18 1996-05-07 カンジ，インコーポレイテッド 腫瘍抑制遺伝子を有するレトロウイルスベクターによる遺伝子療法
US5830461A (en) * 1992-11-25 1998-11-03 University Of Pittsburgh Of The Commonwealth System Of Higher Education Methods for promoting wound healing and treating transplant-associated vasculopathy
US5658565A (en) * 1994-06-24 1997-08-19 University Of Pittsburgh Of The Commonwealth System Of Higher Education Inducible nitric oxide synthase gene for treatment of disease
US6340674B1 (en) 1993-03-26 2002-01-22 Thomas Jefferson University Method of inhibiting the proliferation and causing the differentiation of cells with IGF-1 receptor antisense oligonucleotides
PT1314431E (pt) * 1993-04-30 2008-10-24 Wellstat Biologics Corp Composições purificadas de vírus da doença de newcastle
US6686200B1 (en) * 1993-08-31 2004-02-03 Uab Research Foundation Methods and compositions for the large scale production of recombinant adeno-associated virus
US6652850B1 (en) 1993-09-13 2003-11-25 Aventis Pharmaceuticals Inc. Adeno-associated viral liposomes and their use in transfecting dendritic cells to stimulate specific immunity
US5834441A (en) * 1993-09-13 1998-11-10 Rhone-Poulenc Rorer Pharmaceuticals Inc. Adeno-associated viral (AAV) liposomes and methods related thereto
US5693531A (en) * 1993-11-24 1997-12-02 The United States Of America As Represented By The Department Of Health And Human Services Vector systems for the generation of adeno-associated virus particles
FR2716893B1 (fr) * 1994-03-03 1996-04-12 Rhone Poulenc Rorer Sa Virus recombinants, leur préparation et leur utilisation thérapeutique.
IL113052A0 (en) 1994-03-23 1995-06-29 Rhone Poulenc Rorer Sa Recombinant viruses, their preparation and their use in gene therapy
DE4411402A1 (de) * 1994-03-31 1995-10-05 Juergen Schrader DNA-Expressionsvektoren zur Verwendung in der gentherapeutischen Behandlung von Gefäßerkrankungen
WO1995027494A1 (en) * 1994-04-11 1995-10-19 Sloan-Kettering Institute For Cancer Research DEFECTIVE HERPES AND DEFECTIVE ADENO-ASSOCIATED VIRUS VECTORS WITH p53 FOR THE TREATMENT OF CANCER
EP0755454B1 (en) 1994-04-13 2008-02-13 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
WO1995032225A1 (en) * 1994-05-23 1995-11-30 The Salk Institute For Biological Studies Method for site-specific integration of nucleic acids and related products
US6814962B1 (en) * 1994-06-02 2004-11-09 Aventis Pharma S.A. Recombinant viruses and their use for treatment of atherosclerosis and other forms of coronary artery disease and method, reagent, and kit for evaluating susceptibility to same
US5658785A (en) * 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US20020159979A1 (en) 1994-06-06 2002-10-31 Children's Hospital, Inc. Adeno-associated virus materials and methods
US6204059B1 (en) * 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
MX9701075A (es) 1994-08-12 1998-03-31 Myriad Genetics Inc Mutaciones en vivo y polimorfismos en el gen de susceptibilidad al cancer de pecho y ovario enlazado con 17q.
WO1996005308A1 (en) 1994-08-12 1996-02-22 Myriad Genetics, Inc. Method for diagnosing a predisposition for breast and ovarian cancer
US5871986A (en) 1994-09-23 1999-02-16 The General Hospital Corporation Use of a baculovirus to express and exogenous gene in a mammalian cell
US5652225A (en) * 1994-10-04 1997-07-29 St. Elizabeth's Medical Center Of Boston, Inc. Methods and products for nucleic acid delivery
US5856152A (en) * 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
PT787200E (pt) * 1994-10-28 2005-08-31 Univ Pennsylvania Adenovirus melhorado e metodos para a sua utilizacao
US5872005A (en) * 1994-11-03 1999-02-16 Cell Genesys Inc. Packaging cell lines for adeno-associated viral vectors
US5714170A (en) 1994-11-16 1998-02-03 Thomas Jefferson University Method of inducing resistance to tumor growth
US5998205A (en) 1994-11-28 1999-12-07 Genetic Therapy, Inc. Vectors for tissue-specific replication
US6638762B1 (en) 1994-11-28 2003-10-28 Genetic Therapy, Inc. Tissue-vectors specific replication and gene expression
FR2731710B1 (fr) * 1995-03-14 1997-04-30 Rhone Poulenc Rorer Sa Virus recombinants exprimant la lecithine cholesterol acyltransferase et utilisations en therapie genique
WO1996028560A1 (fr) * 1995-03-16 1996-09-19 Hisamitsu Pharmaceutical Co., Inc. Nouvelles souches cellulaires
FR2732357B1 (fr) 1995-03-31 1997-04-30 Rhone Poulenc Rorer Sa Vecteurs viraux et utilisation pour le traitement des desordres hyperproliferatifs, notamment de la restenose
DE19513152A1 (de) * 1995-04-07 1996-10-10 Bundesrep Deutschland Verwendung eines "Immundefizienzvirus-supprimierenden Lymphokins (ISL)" zur Hemmung der Virusvermehrung, insbesondere von Retroviren
US7745416B2 (en) * 1995-04-11 2010-06-29 The Regents Of The University Of California Method for in vivo regulation of cardiac muscle contractility
US6638264B1 (en) * 1995-05-16 2003-10-28 Biostratum Incorporation Perfusion apparatus and methods for pharmaceutical delivery
DE69624355T2 (de) * 1995-05-16 2003-06-12 Karl Tryggvason Verfahren zur arzneistoffabgabe mittels gentherapie
WO1996037626A1 (en) * 1995-05-22 1996-11-28 Chiron Corporation Position-specific integration of vector constructs into eukaryotic genomes mediated by a chimeric integrase protein
US5756283A (en) * 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US5990388A (en) * 1995-06-07 1999-11-23 Research Corporation Technologies, Inc. Resistance to viruses and viroids in transgenic plants and animals expressing dsRNA-binding protein
US6110456A (en) * 1995-06-07 2000-08-29 Yale University Oral delivery or adeno-associated viral vectors
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
US20020068049A1 (en) * 1998-09-10 2002-06-06 Henderson Daniel R. Tissue specific adenoviral vectors
EP0840622A4 (en) * 1995-07-21 2003-01-02 Aventis Pharma Inc ADENO-ASSOCIATED VIRUS LIPOSOMES AND THEIR USE FOR THE TRANSFECTION OF DENDRITIC CELLS FOR THE STIMULATION OF SPECIFIC IMMUNITY
US5770720A (en) * 1995-08-30 1998-06-23 Barnes-Jewish Hospital Ubiquitin conjugating enzymes having transcriptional repressor activity
US5795872A (en) * 1995-09-19 1998-08-18 Pharmadigm, Inc. DNA construct for immunization
US6162796A (en) * 1995-09-27 2000-12-19 The Rockefeller University Method for transferring genes to the heart using AAV vectors
US6084085A (en) * 1995-11-14 2000-07-04 Thomas Jefferson University Inducing resistance to tumor growth with soluble IGF-1 receptor
PL186018B1 (pl) * 1995-11-30 2003-09-30 Regents Board Of Sposoby i kompozycje do diagnozowania i leczenia nowotworu
EP0785216B2 (en) 1995-12-18 2006-06-07 The University of Utah Research Foundation Chomosome 13-linked breast cancer susceptibility gene BRCA2
US5837492A (en) 1995-12-18 1998-11-17 Myriad Genetics, Inc. Chromosome 13-linked breast cancer susceptibility gene
CA2240737C (en) 1995-12-22 2009-09-29 University Of Utah Research Foundation A long qt syndrome gene which encodes kvlqt1 and its association with mink
US5962313A (en) 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US5846528A (en) * 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5858351A (en) * 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US5952221A (en) * 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
DE19608751B4 (de) * 1996-03-06 2006-05-18 Medigene Ag Verwendung eines Adeno-assoziierten Virus-Vektors zur Steigerung der Immunogenität von Zellen
GB9605124D0 (en) 1996-03-11 1996-05-08 Royal Free Hosp School Med Method of treating muscular disorders
US6723531B2 (en) 1996-04-05 2004-04-20 The Salk Institute For Biological Studies Method for modulating expression of exogenous genes in mammalian systems, and products related thereto
AU3130597A (en) * 1996-05-16 1997-12-05 Duke University Tristetraprolin
US5928914A (en) * 1996-06-14 1999-07-27 Albert Einstein College Of Medicine Of Yeshiva University, A Division Of Yeshiva University Methods and compositions for transforming cells
US6534314B1 (en) 1996-06-14 2003-03-18 Massachusetts Institute Of Technology Methods and compositions for transforming cells
US5780447A (en) * 1996-06-14 1998-07-14 St. Jude Children's Research Hospital Recombinant adeno-associated viral vectors
JP2002512501A (ja) 1996-07-03 2002-04-23 メリアル インコーポレイテッド 外来性ｄｎａを含む組換えイヌアデノウィルス（ｃａｖ）
JP2001500376A (ja) * 1996-09-06 2001-01-16 カイロン コーポレイション 組換えａａｖベクターを使用する治療分子の肝臓特異的送達のための方法および組成物
US20020037867A1 (en) * 1999-02-26 2002-03-28 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
US5866552A (en) * 1996-09-06 1999-02-02 The Trustees Of The University Of Pennsylvania Method for expressing a gene in the absence of an immune response
AU4266597A (en) 1996-09-11 1998-04-14 General Hospital Corporation, The Use of a non-mammalian dna virus to express an exogenous gene in a mammalian cell
US6544523B1 (en) 1996-11-13 2003-04-08 Chiron Corporation Mutant forms of Fas ligand and uses thereof
WO1998024924A1 (en) * 1996-12-05 1998-06-11 Introgene B.V. Genetic modification of primate hemopoietic repopulating stem cells
US7008776B1 (en) 1996-12-06 2006-03-07 Aventis Pharmaceuticals Inc. Compositions and methods for effecting the levels of high density lipoprotein (HDL) cholesterol and apolipoprotein AI very low density lipoprotein (VLDL) cholesterol and low density lipoprotein (LDL) cholesterol
GB9701425D0 (en) * 1997-01-24 1997-03-12 Bioinvent Int Ab A method for in vitro molecular evolution of protein function
US6218597B1 (en) 1997-04-03 2001-04-17 University Technology Corporation Transgenic model and treatment for heart disease
CA2288306A1 (en) 1997-04-28 1998-11-05 Rhone-Poulenc Rorer S.A. Adenovirus-mediated intratumoral delivery of an angiogenesis antagonist for the treatment of tumors
US6541036B1 (en) 1997-05-29 2003-04-01 Thomas Jefferson University Treatment of tumors with oligonucleotides directed to insulin-like growth factor-I receptors (IGF-IR)
US6156303A (en) * 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6635623B1 (en) 1997-06-13 2003-10-21 Baylor College Of Medicine Lipoproteins as nucleic acid vectors
US20050096288A1 (en) * 1997-06-13 2005-05-05 Aragene, Inc. Lipoproteins as nucleic acid vectors
GB9712512D0 (en) 1997-06-16 1997-08-20 Bioinvent Int Ab A method for in vitro molecular evolution of protein function
US6300488B1 (en) 1997-07-10 2001-10-09 The Salk Institute For Biological Studies Modified lepidopteran receptors and hybrid multifunctional proteins for use in transcription and regulation of transgene expression
US6251677B1 (en) 1997-08-25 2001-06-26 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
AU1269699A (en) * 1997-10-08 1999-04-27 Advanced Research And Technology Institute, Inc. Chimeric parvovirus-based recombinant vector system that specifically targets the erythroid lineage
US6346415B1 (en) 1997-10-21 2002-02-12 Targeted Genetics Corporation Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors
IL135578A0 (en) 1997-10-29 2001-05-20 Genzyme Corp Compositions and methods for treating lysosomal storage disease
CA2671261A1 (en) 1997-11-06 1999-05-20 Novartis Vaccines And Diagnostics S.R.L. Neisserial antigens
JP2002500039A (ja) 1998-01-08 2002-01-08 アベンティス ファーマスーティカルズ プロダクツ インコーポレイテッド 機能あるヒトリポタンパク質（ａ）を発現するトランスジェニックウサギ
US6780609B1 (en) 1998-10-23 2004-08-24 Genome Therapeutics Corporation High bone mass gene of 1.1q13.3
ATE446368T1 (de) 1998-01-14 2009-11-15 Novartis Vaccines & Diagnostic Antigene aus neisseria meningitidis
US6984635B1 (en) 1998-02-13 2006-01-10 Board Of Trustees Of The Leland Stanford Jr. University Dimerizing agents, their production and use
EP2261355A3 (en) 1998-05-01 2012-01-11 Novartis Vaccines and Diagnostics, Inc. Neisseria meningitidis antigens and compositions
US6225456B1 (en) 1998-05-07 2001-05-01 University Technololy Corporation Ras suppressor SUR-5
US6333318B1 (en) 1998-05-14 2001-12-25 The Salk Institute For Biological Studies Formulations useful for modulating expression of exogenous genes in mammalian systems, and products related thereto
US6506889B1 (en) 1998-05-19 2003-01-14 University Technology Corporation Ras suppressor SUR-8 and related compositions and methods
US7153655B2 (en) * 1998-06-16 2006-12-26 Alligator Bioscience Ab Method for in vitro molecular evolution of protein function involving the use of exonuclease enzyme and two populations of parent polynucleotide sequence
US6135976A (en) 1998-09-25 2000-10-24 Ekos Corporation Method, device and kit for performing gene therapy
US6743906B1 (en) 1998-10-02 2004-06-01 Board Of Regents, The University Of Texas PPP2R1B is a tumor suppressor
US6221349B1 (en) 1998-10-20 2001-04-24 Avigen, Inc. Adeno-associated vectors for expression of factor VIII by target cells
US6200560B1 (en) * 1998-10-20 2001-03-13 Avigen, Inc. Adeno-associated virus vectors for expression of factor VIII by target cells
US7129043B1 (en) * 1998-10-22 2006-10-31 Duke University Methods of screening for risk of proliferative disease and methods for the treatment of proliferative disease
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6855549B1 (en) 1998-11-23 2005-02-15 The University Of Iowa Research Foundation Methods and compositions for increasing the infectivity of gene transfer vectors
US6441156B1 (en) * 1998-12-30 2002-08-27 The United States Of America As Represented By The Department Of Health And Human Services Calcium channel compositions and methods of use thereof
US6387368B1 (en) 1999-02-08 2002-05-14 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV virus and methods of use thereof
US6495376B1 (en) 1999-02-18 2002-12-17 Beth Israel Deaconess Medical Center Methods and compositions for regulating protein-protein interactions
US7794703B1 (en) * 1999-02-19 2010-09-14 Hai Xing Chen Method for production and delivery of a protein in vivo
US20030215423A1 (en) * 1999-04-01 2003-11-20 Merck & Co., Inc. Gene therapy for obesity
US20080145347A1 (en) * 1999-04-29 2008-06-19 Steiner Mitchell S p-Hyde sequences in the Rat
DK1228217T3 (da) 1999-04-30 2013-02-25 Novartis Vaccines & Diagnostic Konserverede neisseria-antigener
GB9911683D0 (en) 1999-05-19 1999-07-21 Chiron Spa Antigenic peptides
US6165754A (en) * 1999-06-08 2000-12-26 Cornell Research Foundation, Inc. Method of expressing an exogenous nucleic acid
US7504253B2 (en) * 1999-06-11 2009-03-17 The Burnham Institute For Medical Research Nucleic acid encoding proteins involved in protein degradation, products and methods related thereof
GB9916529D0 (en) 1999-07-14 1999-09-15 Chiron Spa Antigenic peptides
ATE422556T1 (de) * 1999-08-20 2009-02-15 Univ Texas Hdac4- und hdac5-abhängige regulation der genexpression im herzen
US7057015B1 (en) 1999-10-20 2006-06-06 The Salk Institute For Biological Studies Hormone receptor functional dimers and methods of their use
CA2864069A1 (en) 1999-10-29 2001-05-03 Novartis Vaccines And Diagnostics S.R.L. Neisserial antigenic peptides
AU3642601A (en) * 1999-11-05 2001-05-30 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
WO2001036603A2 (en) * 1999-11-17 2001-05-25 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
ATE446365T1 (de) 1999-11-18 2009-11-15 Novartis Vaccines & Diagnostic Menschliches fgf-21 gen und genexpressionsprodukte
US6241710B1 (en) 1999-12-20 2001-06-05 Tricardia Llc Hypodermic needle with weeping tip and method of use
ES2507100T3 (es) 2000-01-17 2014-10-14 Novartis Vaccines And Diagnostics S.R.L. Vacuna OMV suplementada contra meningococo
EP1854476A3 (en) 2000-02-09 2008-05-07 Bas Medical, Inc. Use of relaxin to treat diseases related to vasoconstriction
AU3988401A (en) * 2000-02-25 2001-09-03 Ludwig Inst Cancer Res Materials and methods involving hybrid vascular endothelial growth factor dnas and proteins
US20020082205A1 (en) 2000-03-08 2002-06-27 Nobuyuki Itoh Human FGF-23 gene and gene expression products
GB0018307D0 (en) 2000-07-26 2000-09-13 Aventis Pharm Prod Inc Polypeptides
EP1950297A2 (en) 2000-05-31 2008-07-30 Novartis Vaccines and Diagnostics, Inc. Compositions and methods for treating neoplastic disease using chemotherapy and radiation sensitizers
US7198924B2 (en) 2000-12-11 2007-04-03 Invitrogen Corporation Methods and compositions for synthesis of nucleic acid molecules using multiple recognition sites
EP2189473A3 (en) 2000-10-27 2010-08-11 Novartis Vaccines and Diagnostics S.r.l. Nucleic and proteins from streptococcus groups A & B
JP2004536555A (ja) 2000-11-03 2004-12-09 ダナ ファーバー キャンサー インスティテュート 癌感受性の診断のための方法及び組成物並びに欠損ｄｎａ修復メカニズム及びその処置
US20030188326A1 (en) 2000-11-03 2003-10-02 Dana Farber Cancer Institute Methods and compositions for the diagnosis of cancer susceptibilities and defective DNA repair mechanisms and treatment thereof
US6958213B2 (en) 2000-12-12 2005-10-25 Alligator Bioscience Ab Method for in vitro molecular evolution of protein function
US20020086292A1 (en) 2000-12-22 2002-07-04 Shigeaki Harayama Synthesis of hybrid polynucleotide molecules using single-stranded polynucleotide molecules
DK1355918T5 (da) 2000-12-28 2012-02-20 Wyeth Llc Rekombinant beskyttelsesprotein af streptococcus pneumoniae
US6613534B2 (en) 2001-03-20 2003-09-02 Wake Forest University Health Sciences MAP-2 as a determinant of metastatic potential
GB0107661D0 (en) 2001-03-27 2001-05-16 Chiron Spa Staphylococcus aureus
GB0107658D0 (en) 2001-03-27 2001-05-16 Chiron Spa Streptococcus pneumoniae
US8034791B2 (en) 2001-04-06 2011-10-11 The University Of Chicago Activation of Egr-1 promoter by DNA damaging chemotherapeutics
WO2002081641A2 (en) 2001-04-06 2002-10-17 Georgetown University Gene scc-112 and diagnostic and therapeutic uses thereof
ATE541937T1 (de) * 2001-04-06 2012-02-15 Univ Chicago Chemotherapeutische einleitung der egr-1-promoter-aktivität in gentherapie
US20040242523A1 (en) * 2003-03-06 2004-12-02 Ana-Farber Cancer Institue And The Univiersity Of Chicago Chemo-inducible cancer gene therapy
AU2002258728A1 (en) 2001-04-06 2002-10-21 Georgetown University Gene brcc-3 and diagnostic and therapeutic uses thereof
WO2002081639A2 (en) 2001-04-06 2002-10-17 Georgetown University Gene brcc2 and diagnostic and therapeutic uses thereof
WO2002097056A2 (en) * 2001-05-31 2002-12-05 The Rockefeller University Method for generating replication defective viral vectors that are helper free
AUPR546801A0 (en) 2001-06-05 2001-06-28 Commonwealth Scientific And Industrial Research Organisation Recombinant antibodies
US7449562B2 (en) * 2001-06-29 2008-11-11 Novartis Ag PERV screening method and use thereof
MX339524B (es) 2001-10-11 2016-05-30 Wyeth Corp Composiciones inmunogenicas novedosas para la prevencion y tratamiento de enfermedad meningococica.
JP4413617B2 (ja) 2001-12-12 2010-02-10 カイロン ソチエタ ア レスポンサビリタ リミタータ Ｃｈｌａｍｙｄｉａｔｒａｃｈｏｍａｔｉｓに対する免疫化
KR100981471B1 (ko) 2002-03-15 2010-09-10 더 큐레이터스 오브 더 유니버시티 오브 미주리 효소 활성이 감소된 형별불능 헤모필러스 인플루엔자의ｐ4 변형 단백질
US7384738B2 (en) * 2002-03-28 2008-06-10 Bremel Robert D Retrovirus-based genomic screening
US7244565B2 (en) 2002-04-10 2007-07-17 Georgetown University Gene shinc-3 and diagnostic and therapeutic uses thereof
MXPA04010282A (es) 2002-04-18 2005-08-18 Acuity Pharmaceuticals Inc Medios y metodos para la inhibicion especifica de genes en celulas y tejido de cns y/u ojo.
US7262012B2 (en) * 2002-05-17 2007-08-28 Alligator Bioscience Ab Method for in vitro molecular evolution of protein function using varied exonuclease digestion in two polynucleotide populations
JP4563171B2 (ja) 2002-05-24 2010-10-13 シェーリング コーポレイション 中和ヒト抗ｉｇｆｒ抗体
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
US7785608B2 (en) 2002-08-30 2010-08-31 Wyeth Holdings Corporation Immunogenic compositions for the prevention and treatment of meningococcal disease
WO2007068784A1 (en) 2005-12-14 2007-06-21 Licentia Ltd Novel neurotrophic factor protein and uses thereof
CN102304570B (zh) 2002-11-13 2015-01-21 托马斯杰斐逊大学 用于癌症诊断和治疗的组合物和方法
RU2377253C2 (ru) 2002-12-02 2009-12-27 Амген Фремонт,Инк. Антитела, специфичные к фактору некроза опухолей, и их применение
US20050048041A1 (en) * 2003-01-13 2005-03-03 Rao Mahendra S. Persistent expression of candidate molecule in proliferating stem and progenitor cells for delivery of therapeutic products
ATE477337T1 (de) 2003-01-16 2010-08-15 Univ Pennsylvania Zusammensetzungen und verfahren zur sirna-hemmung von icam-1
GB0308198D0 (en) 2003-04-09 2003-05-14 Chiron Srl ADP-ribosylating bacterial toxin
US20050028229A1 (en) * 2003-04-14 2005-02-03 Mitchell Weiss Alpha-hemoglobin stabilizing protein transgenic mouse and methods of use thereof
US7514533B2 (en) * 2003-05-21 2009-04-07 Ares Trading S.A. TNF-like secreted protein
TW200518669A (en) * 2003-09-12 2005-06-16 Vertex Pharma Animal model for protease activity and liver damage
US20060078558A1 (en) * 2003-11-12 2006-04-13 Whitsett Jeffrey A Diagnosis, prognosis and treatment of pulmonary diseases
WO2005054438A2 (en) 2003-12-01 2005-06-16 Invitrogen Corporation Nucleic acid molecules containing recombination sites and methods of using the same
WO2005067708A2 (en) 2004-01-14 2005-07-28 Daniolabs Limited Zebrafish model for autoimmune diseases
US7432057B2 (en) 2004-01-30 2008-10-07 Michigan State University Genetic test for PSE-susceptible turkeys
EP1713938A2 (en) 2004-02-09 2006-10-25 Thomas Jefferson University DIAGNOSIS AND TREATMENT OF CANCERS WITH MicroRNA LOCATED IN OR NEAR CANCER-ASSOCIATED CHROMOSOMAL FEATURES
EP1732947B1 (en) * 2004-03-05 2011-04-27 Vegenics Pty Ltd Growth factor binding constructs materials and methods
EP2899278A1 (en) * 2004-03-12 2015-07-29 Alnylam Pharmaceuticals Inc. iRNA agents targeting VEGF
US7319015B2 (en) * 2004-03-16 2008-01-15 The Regents Of The University Of Michigan Methods and compositions for using alveolar macrophage phospholipase A2
US7582442B2 (en) 2004-03-16 2009-09-01 The Regents Of The University Of Michigan Methods and compositions for using aleveolar macrophage phospholipase A2
US8268324B2 (en) 2004-03-29 2012-09-18 Galpharma Co., Ltd. Modified galectin 9 proteins and use thereof
US7604798B2 (en) 2004-07-15 2009-10-20 Northwestern University Methods and compositions for importing nucleic acids into cell nuclei
WO2006014798A2 (en) * 2004-07-27 2006-02-09 Mount Sinai School Of Medicine Methods and compositions for using sax2
EP1781284B1 (en) * 2004-08-25 2010-10-13 The University of Chicago Use of the combination comprising temozolomide and tnf-alpha for treating glioblastoma
FI20050753A (fi) 2004-09-03 2006-03-04 Licentia Oy Uudet peptidit
GB0423126D0 (en) * 2004-10-18 2004-11-17 Ares Trading Sa Protein
JP2008520583A (ja) * 2004-11-15 2008-06-19 マウント シナイ スクール オブ メディスン オブ ニューヨーク ユニバーシティー Ｗｎｔ自己分泌シグナル伝達を改変するための組成物および方法
GB0426960D0 (en) 2004-12-08 2005-01-12 Ares Trading Sa TGR-3 like protein receptor
US7531523B2 (en) * 2005-02-17 2009-05-12 Vertex Pharmaceuticals Incorporated Sodium channel protein type III alpha-subunit splice variant
GB0504767D0 (en) * 2005-03-08 2005-04-13 Ares Trading Sa Lipocalin protein
US20060216315A1 (en) * 2005-03-16 2006-09-28 Yoo Tai J Cockroach allergen gene expression and delivery systems and uses
US20100028312A1 (en) * 2005-03-24 2010-02-04 Caritas St. Elizabeth Medical Center of Boston Inc Stably transformed bone marrow-derived cells and uses thereof
US9181184B2 (en) 2005-05-17 2015-11-10 Amicus Therapeutics, Inc. Method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
US20090270479A1 (en) * 2005-07-12 2009-10-29 Antonio Giordano Genetic and Epigenetic Alterations In the Diagnosis and Treatment of Cancer
US20100119525A1 (en) * 2005-08-01 2010-05-13 Mount Sinai Schoool Of Medicine Of New York University Method for extending longevity using npc1l1 antagonists
CA2618796C (en) 2005-08-11 2018-01-02 Arpi Matossian-Rogers Peptides for treatment and diagnosis of autoimmune disease
AU2006279462A1 (en) 2005-08-15 2007-02-22 Vegenics Limited Modified VEGF and PDGF with improved angiogenic properties
AU2006291165B2 (en) 2005-09-12 2013-03-14 The Ohio State University Research Foundation Compositions and methods for the diagnosis and therapy of BCL2-associated cancers
US7972813B2 (en) * 2005-09-30 2011-07-05 Vertex Pharmaceuticals Incorporated Tetrodotoxin-resistant sodium channel alpha subunit
US20080200408A1 (en) * 2005-09-30 2008-08-21 Mccormack Kenneth Deletion mutants of tetrodotoxin-resistant sodium channel alpha subunit
GB2432366B (en) * 2005-11-19 2007-11-21 Alligator Bioscience Ab A method for in vitro molecular evolution of protein function
CA2524619A1 (en) * 2005-11-22 2007-05-22 Ottawa Health Research Institute Novel stem cells, nucleotide sequences and proteins therefrom
CA2634286A1 (en) 2005-12-22 2007-08-09 Samuel Jotham Reich Compositions and methods for regulating complement system
NZ569883A (en) 2005-12-28 2011-12-22 Translational Therapeutics Inc Use of triazole containing compounds for inhibiting 4E activity and proliferation of a cell
EP2484782B1 (en) 2006-01-05 2015-11-18 The Ohio State University Research Foundation MicroRNA-based methods and compositions for the diagnosis and treatment of solid cancers
EP2591794A1 (en) 2006-01-05 2013-05-15 The Ohio State University Research Foundation MicroRNA expressions abnormalities in pancreatic endocrine and acinar tumors
EP2502630B1 (en) 2006-01-05 2015-03-11 The Ohio State University Research Foundation MicroRNA-based methods and compositions for the diagnosis, prognosis and treatment of lung cancer
US20090214496A1 (en) * 2006-01-30 2009-08-27 Licentia Ltd. Bmx/etk tyrosine kinase gene therapy materials and methods
US8229398B2 (en) * 2006-01-30 2012-07-24 Qualcomm Incorporated GSM authentication in a CDMA network
ES2448491T3 (es) 2006-03-02 2014-03-14 The Ohio State University Research Foundation Perfil de expresión de microARN asociado con cáncer de páncreas
ES2446362T3 (es) 2006-03-20 2014-03-07 The Ohio State University Research Foundation Huellas de microARN durante megacariocipoyesis humana
WO2007108557A1 (ja) 2006-03-20 2007-09-27 Japan Science And Technology Agency Ｉｐ３受容体結合タンパク質による細胞内標的分子の制御
NZ571568A (en) 2006-03-31 2010-11-26 Alnylam Pharmaceuticals Inc Double-stranded RNA molecule compositions and methods for inhibiting expression of Eg5 gene
EP2007880A2 (en) 2006-04-07 2008-12-31 Neuro Therapeutics AB Survival and development of neural cells
WO2007127919A2 (en) * 2006-04-28 2007-11-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a gene from the jc virus
US7723564B2 (en) 2006-05-10 2010-05-25 Board Of Regents Of The University Of Nebraska Compositions and methods for modulation of KSR1 and KSR2 interactions
CN101484588B (zh) 2006-05-11 2013-11-06 阿尔尼拉姆医药品有限公司 抑制pcsk9基因表达的组合物和方法
EP2018184B1 (en) 2006-05-17 2013-09-11 The Ludwig Institute for Cancer Research Anti-VEGF-B antibody for the treatment or prophylaxis of diabetes type II or metabolic syndrome
EP2392583A1 (en) * 2006-05-19 2011-12-07 Alnylam Europe AG. RNAi modulation of Aha and therapeutic uses thereof
CA2653451C (en) * 2006-05-22 2015-12-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of ikk-b gene
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
PL2054431T3 (pl) 2006-06-09 2012-07-31 Novartis Ag Konformery adhezyn bakteryjnych
US20080152654A1 (en) * 2006-06-12 2008-06-26 Exegenics, Inc., D/B/A Opko Health, Inc. COMPOSITIONS AND METHODS FOR siRNA INHIBITION OF ANGIOGENESIS
ES2434070T3 (es) 2006-07-13 2013-12-13 The Ohio State University Research Foundation MIR-103-2 para el diagnóstico de adenocarcinoma de colon con mal pronóstico de supervivencia
CN101495633B (zh) 2006-07-28 2015-01-07 塞诺菲-安万特股份有限公司 用于治疗肿瘤的组合物和方法
US7872118B2 (en) * 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
CA2663581C (en) 2006-09-21 2016-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the hamp gene
EP2530090A3 (en) 2006-10-19 2013-01-23 CSL Limited Anti-IL-13R alpha 1 antibodies and their uses thereof
JP5238710B2 (ja) 2006-10-19 2013-07-17 シーエスエル、リミテッド インターロイキン−１３レセプターアルファ１の高親和性抗体アンタゴニスト
US20080113915A1 (en) * 2006-11-09 2008-05-15 Duke University SAPAP3 knockout mouse and clinical modeling associated with the SAPAP3 gene
AR064642A1 (es) 2006-12-22 2009-04-15 Wyeth Corp Polinucleotido vector que lo comprende celula recombinante que comprende el vector polipeptido , anticuerpo , composicion que comprende el polinucleotido , vector , celula recombinante polipeptido o anticuerpo , uso de la composicion y metodo para preparar la composicion misma y preparar una composi
MX2009008413A (es) * 2007-02-06 2009-11-02 Tai June Yoo Tratamiento y prevencion de enfermedades neurodegenerativas usando terapia genica.
WO2008115387A2 (en) * 2007-03-15 2008-09-25 University Hospitals Of Cleveland Screening, diagnosing, treating and prognosis of pathophysiologic states by rna regulation
PE20090064A1 (es) 2007-03-26 2009-03-02 Novartis Ag Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende
US7759320B2 (en) 2007-03-29 2010-07-20 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a gene from the Ebola
CA2692503C (en) 2007-07-05 2013-09-24 Novartis Ag Dsrna for treating viral infection
WO2009012263A2 (en) * 2007-07-18 2009-01-22 The Trustees Of Columbia University In The City Of New York Tissue-specific micrornas and compositions and uses thereof
AU2008282318B2 (en) 2007-07-31 2014-02-27 The Ohio State University Research Foundation Methods for reverting methylation by targeting methyltransferases
JP2010535473A (ja) 2007-08-03 2010-11-25 ズィ、オハイオウ、ステイト、ユーニヴァーサティ、リサーチ、ファウンデイシャン ｎｃＲＮＡをコードする超保存領域
AU2008288806B2 (en) 2007-08-22 2014-11-27 The Ohio State University Research Foundation Methods and compositions for inducing deregulation of EphA7 and Erk phosphorylation in human acute leukemias
EP3048177A1 (en) 2007-09-06 2016-07-27 The Ohio State University Research Foundation Microrna signatures in human ovarian cancer
WO2009049129A1 (en) 2007-10-11 2009-04-16 The Ohio State University Research Foundation Methods and compositions for the diagnosis and treatment of esphageal adenocarcinomas
FI20070808A0 (fi) 2007-10-25 2007-10-25 Mart Saarma GDNF:n silmukointivariantit ja niiden käytöt
JP5530933B2 (ja) 2007-12-10 2014-06-25 アルナイラム ファーマシューティカルズ， インコーポレイテッド 第ｖｉｉ因子遺伝子発現阻害のための組成物及び方法
US20090196854A1 (en) * 2008-02-04 2009-08-06 Kytos Biosystems S.A. Methods and compositions for use of crl 5803 cells for expression of biotherapeutics and encapsulated cell-based delivery
AU2009221775B2 (en) * 2008-03-05 2015-05-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of Eg5 and VEGF genes
FI20080326A0 (fi) 2008-04-30 2008-04-30 Licentia Oy Neurotroofinen tekijä MANF ja sen käytöt
JP2011526916A (ja) 2008-06-30 2011-10-20 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン 全身性紅斑性狼瘡を同定および処置するための診断標的および治療標的としてのリソソームホスホリパーゼａ２（ｌｐｌａ２）活性
WO2010028054A1 (en) 2008-09-02 2010-03-11 Alnylam Europe Ag. Compositions and methods for inhibiting expression of mutant egfr gene
CA2739170A1 (en) 2008-09-25 2010-04-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
ES2656516T3 (es) 2008-10-20 2018-02-27 Alnylam Pharmaceuticals, Inc. Composiciones y métodos para inhibir la expresión de transtiretina
NZ614064A (en) 2008-11-05 2015-04-24 Wyeth Llc Multicomponent immunogenic composition for the prevention of beta-hemolytic streptococcal (bhs) disease
US8470792B2 (en) 2008-12-04 2013-06-25 Opko Pharmaceuticals, Llc. Compositions and methods for selective inhibition of VEGF
AU2009324534B2 (en) 2008-12-10 2015-07-30 Alnylam Pharmaceuticals, Inc. GNAQ targeted dsRNA compositions and methods for inhibiting expression
WO2010099341A1 (en) 2009-02-26 2010-09-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of mig-12 gene
CN102421900B (zh) 2009-03-12 2015-07-22 阿尔尼拉姆医药品有限公司 用于抑制Eg5和VEGF基因表达的脂质配制的组合物以及方法
CA2755870C (en) 2009-03-20 2019-04-09 Angioblast Systems, Inc. Production of reprogrammed pluripotent cells
CA2758290C (en) 2009-04-27 2018-04-10 Novartis Ag Antagonistic activin receptor iib (actriib) antibodies for increasing muscle growth
EP2424987B1 (en) * 2009-05-01 2017-11-15 CuRNA, Inc. Treatment of hemoglobin (hbf/hbg) related diseases by inhibition of natural antisense transcript to hbf/hbg
GB0908425D0 (en) 2009-05-15 2009-06-24 Medical Res Council Medical use
US9050276B2 (en) 2009-06-16 2015-06-09 The Trustees Of Columbia University In The City Of New York Autism-associated biomarkers and uses thereof
AP3574A (en) 2009-08-14 2016-02-08 Alnylam Pharmaceuticals Inc Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
JP2013509422A (ja) 2009-10-30 2013-03-14 シーエヌエス セラピューティクス，インク． 改良されたニュールツリン分子
AU2010313112B2 (en) 2009-11-02 2014-06-26 The Regents Of The University Of California Vault complexes for cytokine delivery
US8551781B2 (en) 2009-11-19 2013-10-08 The Regents Of The University Of California Vault complexes for facilitating biomolecule delivery
EP2507373A4 (en) * 2009-12-04 2013-08-07 Opko Pharmaceuticals Llc COMPOSITIONS AND METHODS FOR INHIBITING VEGF
CA2792291A1 (en) 2010-03-29 2011-10-06 Kumamoto University Sirna therapy for transthyretin (ttr) related ocular amyloidosis
JP2013523162A (ja) 2010-04-06 2013-06-17 アルナイラム ファーマシューティカルズ， インコーポレイテッド Ｃｄ２７４／ｐｄ−ｌ１遺伝子の発現を阻害するための組成物および方法
EP2563907B1 (en) 2010-04-28 2019-06-19 The J. David Gladstone Institutes Methods for generating cardiomyocytes
CA2798703A1 (en) 2010-05-10 2011-11-17 The Regents Of The University Of California Endoribonuclease compositions and methods of use thereof
WO2011153323A2 (en) 2010-06-02 2011-12-08 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
WO2011154553A2 (en) 2010-06-11 2011-12-15 Cellartis Ab Novel micrornas for the detection and isolaton of human embryonic stem cell-derived cardiac cell types
AU2011274510A1 (en) 2010-07-09 2013-01-24 Exelixis, Inc. Combinations of kinase inhibitors for the treatment of cancer
GB201012651D0 (en) 2010-07-28 2010-09-15 Univ Edinburgh Peptides
HRP20210242T4 (hr) 2010-08-23 2024-05-10 Wyeth Llc Stabilne formulacije antigena iz bakterije neisseria meningitidis rlp2086
CN103096920B (zh) 2010-09-10 2016-03-23 惠氏有限责任公司 脑膜炎奈瑟球菌orf2086抗原的非脂质化变体
BR112013009196A2 (pt) 2010-10-15 2020-08-25 The Trustees Of Columbia University In The City Of New York usos de polipeptídeo para redução da aquisição de ácido graxo e da ingestão de alimento, bem como para promoção de saciedade relacionados à obesidade
PL2635299T3 (pl) 2010-11-02 2020-03-31 The Trustees Of Columbia University In The City Of New York Sposoby leczenia zaburzeń utraty włosów
EP2635346B1 (en) 2010-11-05 2017-03-29 The Board of Trustees of the Leland Stanford Junior University Optogenetic control of reward-related behaviors
EP2635108B1 (en) 2010-11-05 2019-01-23 The Board of Trustees of the Leland Stanford Junior University Light-activated chimeric opsins and methods of using the same
EP2698635A3 (en) 2010-12-01 2014-05-28 The University Of North Carolina At Chapel Hill Methods and compositions for targeting sites of neovascular growth
EP2648763A4 (en) 2010-12-10 2014-05-14 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR EXPRESSION INHIBITION OF GENES KLF-1 AND BCL11A
EP2649182A4 (en) 2010-12-10 2015-05-06 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHOD FOR INCREASING AN ERYTHROPOIETIN (EPO) PREPARATION
WO2012109495A1 (en) 2011-02-09 2012-08-16 Metabolic Solutions Development Company, Llc Cellular targets of thiazolidinediones
MX343008B (es) 2011-03-29 2016-10-21 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion del gen de proteasa transmembrana, serina 6 (tmprss6).
WO2012142526A1 (en) 2011-04-14 2012-10-18 Modiano Jaime Use of tumor fas expression to determine response to anti-cancer therapy
CA2840175C (en) 2011-05-19 2021-11-16 The Regents Of The University Of Michigan Integrin alpha-2 binding agents and use thereof to inhibit cancer cell proliferation
US20140134728A1 (en) 2011-06-01 2014-05-15 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods for adjusting expression of mitochondrial genome by microrna
WO2012177906A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
CA3191066A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibition of expression of apolipoprotein c-iii (apoc3) genes
WO2012177949A2 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein c (proc) genes
EP4092120A1 (en) 2011-06-21 2022-11-23 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (anglptl3) irna compositions and methods of use thereof
EP3597750B1 (en) 2011-06-23 2022-05-04 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
WO2013019857A2 (en) 2011-08-01 2013-02-07 Alnylam Pharmaceuticals, Inc. Method for improving the success rate of hematopoietic stem cell transplants
US9279002B2 (en) 2011-09-15 2016-03-08 Los Angeles Biomedical Research Institute At Harbor-Ucla Medical Center Immunotherapy and diagnosis of mucormycosis using CotH
CN104364390B (zh) 2011-10-14 2016-08-24 俄亥俄州立大学 与卵巢癌相关的方法和材料
US20140288156A1 (en) 2011-10-27 2014-09-25 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods for the treatment and diagnosis of atherosclerosis
AU2012321102C1 (en) 2011-10-27 2016-11-10 Pharma Cinq, Llc Vectors encoding rod-derived cone viability factor
WO2013079687A1 (en) 2011-11-30 2013-06-06 The Chancellor, Masters And Scholars Of The University Of Oxford Inkt cell modulators and methods of using the same
GB201120860D0 (en) 2011-12-05 2012-01-18 Cambridge Entpr Ltd Cancer immunotherapy
CN104093833B (zh) 2011-12-16 2017-11-07 斯坦福大学托管董事会 视蛋白多肽及其使用方法
AU2013221317B2 (en) 2012-02-16 2017-07-06 Pangu Biopharma Limited Histidyl-tRNA synthetases for treating autoimmune and inflammatory diseases
EP2817068B1 (en) 2012-02-21 2017-04-12 The Board of Trustees of the Leland Stanford Junior University Compositions for treating neurogenic disorders of the pelvic floor
BR122016004924A2 (pt) 2012-03-09 2019-07-30 Pfizer Inc. Polipeptídeo isolado e composições imunogênicas compreendendo os mesmos
SA115360586B1 (ar) 2012-03-09 2017-04-12 فايزر انك تركيبات لعلاج الالتهاب السحائي البكتيري وطرق لتحضيرها
WO2013139861A1 (en) 2012-03-20 2013-09-26 Luc Montagnier Methods and pharmaceutical compositions of the treatment of autistic syndrome disorders
HUE040127T2 (hu) 2012-03-29 2019-02-28 Univ Columbia Eljárások hajhullási rendellenességek kezelésére
US20150190532A1 (en) 2012-04-04 2015-07-09 The Trustees Of Columbia University In The City Of New York Smooth muscle specific inhibition for anti-restenotic therapy
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
JP2015518475A (ja) 2012-04-10 2015-07-02 インサーム（インスティテュ ナシオナル ドゥ ラ サンテ エ ドゥ ラルシェルシェ メディカル）Ｉｎｓｅｒｍ（Ｉｎｓｔｉｔｕｔ Ｎａｔｉｏｎａｌ Ｄｅｌａ Ｓａｎｔｅ Ｅｔｄｅ Ｌａ Ｒｅｃｈｅｒｃｈｅ Ｍｅｄｉｃａｌｅ） 非アルコール性脂肪性肝炎の治療方法
WO2013153139A1 (en) 2012-04-11 2013-10-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment and diagnosis of acute leukemia
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
MX362866B (es) 2012-05-25 2019-02-20 Univ California Metodos y composiciones para la modificacion de adn objetivo dirigida por arn y para la modulacion de la transcripcion dirigida por arn.
WO2013184209A1 (en) 2012-06-04 2013-12-12 Ludwig Institute For Cancer Research Ltd. Mif for use in methods of treating subjects with a neurodegenerative disorder
US20150150821A1 (en) 2012-07-09 2015-06-04 The Regents Of The University Of California Vault Immunotherapy
IN2015DN00552A (ko) 2012-07-19 2015-06-26 Redwood Bioscience Inc
US20140037599A1 (en) * 2012-08-03 2014-02-06 The Trustees Of The University Of Pennsylvania Compositions and Methods of Treating T Cell Deficiency
PT2885010T (pt) 2012-08-16 2020-03-11 Ipierian Inc Métodos de tratamento de uma tauopatia
CA2884245C (en) 2012-09-06 2023-03-14 The University Of Chicago Antisense polynucleotides to induce exon skipping and methods of treating dystrophies
AU2013334229B2 (en) 2012-10-25 2018-02-15 Bioverativ Usa Inc. Anti-complement C1s antibodies and uses thereof
DK2914291T3 (da) 2012-11-02 2022-05-16 Bioverativ Usa Inc Anti-komplement-c1s-antistoffer og anvendelser deraf
EP3659518A1 (en) 2012-11-21 2020-06-03 Circuit Therapeutics, Inc. System and method for optogenetic therapy
SI2929031T1 (en) 2012-12-05 2018-02-28 Alnylam Pharmaceuticals, Inc. PCSK9 IRNA assemblies and procedures for their use
EP2956557B1 (en) 2013-02-14 2018-07-11 The J. David Gladstone Institutes Compositions and methods of use thereof for identifying anti-viral agents
PT3613439T (pt) 2013-02-15 2021-05-12 Univ California Recetor de antigénio quimérico e métodos de utilização do mesmo
PT2956476T (pt) 2013-02-18 2020-02-21 Vegenics Pty Ltd Moléculas de ligação e usos destas
JP6446377B2 (ja) 2013-03-08 2018-12-26 ファイザー・インク 免疫原性融合ポリペプチド
US10981961B2 (en) 2013-03-11 2021-04-20 University Of Florida Research Foundation, Incorporated Delivery of card protein as therapy for occular inflammation
US9481903B2 (en) 2013-03-13 2016-11-01 Roche Molecular Systems, Inc. Systems and methods for detection of cells using engineered transduction particles
EP3699592A1 (en) 2013-03-13 2020-08-26 Geneweave Biosciences Inc. Non-replicative transduction particles and transduction particle-based reporter systems
MX366660B (es) 2013-03-14 2019-07-18 Alnylam Pharmaceuticals Inc Composiciones de arni contra el componente c5 del complemento y métodos para su uso.
EP2968997B1 (en) 2013-03-15 2019-06-26 The Board of Trustees of the Leland Stanford Junior University Optogenetic control of behavioral state
JP2016515508A (ja) 2013-03-15 2016-05-30 ザ・トラスティーズ・オブ・コロンビア・ユニバーシティ・イン・ザ・シティ・オブ・ニューヨーク 融合タンパク質及びその方法
WO2014160871A2 (en) 2013-03-27 2014-10-02 The General Hospital Corporation Methods and agents for treating alzheimer's disease
WO2014179331A2 (en) 2013-04-29 2014-11-06 The Board Of Trustees Of The Leland Stanford Junior University Devices, systems and methods for optogenetic modulation of action potentials in target cells
EP2999785B1 (en) 2013-05-22 2018-04-04 Alnylam Pharmaceuticals, Inc. Serpina1 irna compositions and methods of use thereof
IL293657A (en) 2013-05-22 2022-08-01 Alnylam Pharmaceuticals Inc Preparations of tmprss6 irna and methods of using them
WO2014191630A2 (en) 2013-05-28 2014-12-04 Helsingin Yliopisto Non-human animal model encoding a non-functional manf gene
EP3071598B1 (en) 2013-05-29 2021-07-07 Vybion, Inc. Single chain intrabodies that alter huntingtin mutant degradation
US9701985B2 (en) 2013-06-04 2017-07-11 Virginia Commonwealth University mda-9/syntenin promoter to image and treat metastatic cancer cells
WO2014209553A1 (en) 2013-06-04 2014-12-31 Virginia Commonwealth University Use of a truncated ccn1 promoter for cancer diagnostics, therapeutics and theranostics
WO2014197599A1 (en) 2013-06-04 2014-12-11 The Johns Hopkins University Peg-prom mediated surface expression of avidin/streptavidin
WO2014197535A1 (en) 2013-06-04 2014-12-11 Virginia Commonwealth University Recombinant cancer therapeutic cytokine
EP3003393A4 (en) 2013-06-04 2017-01-11 The Johns Hopkins University Tripartite cancer theranostic nucleic acid constructs
JP6446443B2 (ja) 2013-06-10 2018-12-26 アイピエリアン，インコーポレイティド タウオパチーの処置方法
EP3013424A4 (en) 2013-06-25 2017-03-29 University of Canberra Methods and compositions for modulating cancer stem cells
AU2014306679A1 (en) 2013-08-14 2016-03-10 Circuit Therapeutics, Inc. Compositions and methods for controlling pain
CA2923129C (en) 2013-09-08 2020-06-09 Pfizer Inc. Neisseria meningitidis compositions and methods thereof
SG11201601408PA (en) 2013-09-18 2016-04-28 Univ Canberra Stem cell modulation ii
MX2016004230A (es) 2013-10-02 2016-10-21 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion del gen lect2.
SG10201910929QA (en) 2013-10-04 2020-01-30 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of the alas1 gene
JP2016537028A (ja) 2013-11-18 2016-12-01 クリスパー セラピューティクス アーゲー Ｃｒｉｓｐｒ−ｃａｓシステムの材料及び方法
EP3079727A1 (en) 2013-12-12 2016-10-19 INSERM - Institut National de la Santé et de la Recherche Médicale Methods for the prevention and treatment of diabetic cardiomyopathy using mir-424/322
EP3080266B1 (en) 2013-12-12 2021-02-03 The Regents of The University of California Methods and compositions for modifying a single stranded target nucleic acid
KR102344559B1 (ko) 2013-12-12 2021-12-31 알닐람 파마슈티칼스 인코포레이티드 보체 성분 ｉＲＮＡ 조성물 및 이의 이용 방법
US9682123B2 (en) 2013-12-20 2017-06-20 The Trustees Of Columbia University In The City Of New York Methods of treating metabolic disease
EP3960860A3 (en) 2014-02-11 2022-06-08 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
EP3107939B1 (en) 2014-02-19 2020-06-17 University of Florida Research Foundation, Inc. Delivery of nrf2 as therapy for protection against reactive oxygen species
AU2015237265A1 (en) 2014-03-28 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Engineered light-activated anion channel proteins and methods of use thereof
TW201607559A (zh) 2014-05-12 2016-03-01 阿尼拉製藥公司 治療ｓｅｒｐｉｎｃ１相關疾患之方法和組成物
JP6811094B2 (ja) 2014-05-22 2021-01-13 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． アンジオテンシノーゲン（ＡＧＴ）ｉＲＮＡ組成物およびその使用
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
CN113248623A (zh) 2014-06-18 2021-08-13 阿尔伯特爱因斯坦医学院 Syntac多肽及其用途
EP3177640B1 (en) 2014-08-08 2020-05-06 The Board of Trustees of the Leland Stanford Junior University High affinity pd-1 agents and methods of use
CA2958704A1 (en) 2014-08-25 2016-03-03 University Of Canberra Compositions for modulating cancer stem cells and uses therefor
TW201614073A (en) 2014-08-25 2016-04-16 Geneweave Biosciences Inc Non-replicative transduction particles and transduction particle-based reporter systems
EA201790533A1 (ru) 2014-09-07 2017-07-31 Селекта Байосайенсиз, Инк. Способы и композиции для ослабления иммунных ответов против вирусного вектора для переноса, предназначенного для модулирования экспрессии генов
PT3198018T (pt) 2014-09-24 2021-02-24 Hope City Variantes dos vetores de vírus adenoassociados para edição do genoma de alta eficácia e métodos da mesma
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
CA2966620A1 (en) 2014-11-05 2016-05-12 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
WO2016081444A1 (en) 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
WO2016105517A1 (en) 2014-12-23 2016-06-30 The Trustees Of Columbia University In The City Of New York Fusion proteins and methods thereof
WO2016130806A2 (en) 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
AU2016221318B2 (en) 2015-02-19 2020-06-25 Pfizer Inc. Neisseria meningitidis compositions and methods thereof
CA3091771A1 (en) 2015-03-13 2016-09-22 The Jackson Laboratory A three-component crispr/cas complex system and uses thereof
DK3277814T3 (da) * 2015-04-03 2020-08-24 Univ Massachusetts Oligonukleotidforbindelser til målretning mod huntingtin-mrna
HUE061076T2 (hu) 2015-04-06 2023-05-28 Bioverativ Usa Inc Humanizált anti-C1s ellenanyagok alkalmazási eljárások
US10669596B2 (en) 2015-04-07 2020-06-02 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Methods for inducing cell division of postmitotic cells
DK3283500T3 (en) 2015-04-08 2020-11-16 Univ Chicago Compositions and methods for correcting limb girdle muscular dystrophy type 2C using exon skipping
WO2016191418A1 (en) 2015-05-26 2016-12-01 Salk Institute For Biological Studies Motor neuron-specific expression vectors
US10392607B2 (en) 2015-06-03 2019-08-27 The Regents Of The University Of California Cas9 variants and methods of use thereof
EP3307316A1 (en) 2015-06-12 2018-04-18 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2017023861A1 (en) 2015-08-03 2017-02-09 The Regents Of The University Of California Compositions and methods for modulating abhd2 activity
WO2017037203A1 (en) 2015-09-02 2017-03-09 Immutep S.A.S. Anti-LAG-3 Antibodies
WO2017044807A2 (en) 2015-09-09 2017-03-16 The Trustees Of Columbia University In The City Of New York Reduction of er-mam-localized app-c99 and methods of treating alzheimer's disease
AU2016339053A1 (en) 2015-09-24 2018-04-12 Crispr Therapeutics Ag Novel family of RNA-programmable endonucleases and their uses in genome editing and other applications
US10188750B1 (en) 2015-10-23 2019-01-29 University Of South Florida Self-replicating cell selective gene delivery compositions, methods, and uses thereof
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
SG10201913805RA (en) 2016-01-08 2020-03-30 Univ California Conditionally active heterodimeric polypeptides and methods of use thereof
CA3011894A1 (en) 2016-01-31 2017-08-03 University Of Massachusetts Branched oligonucleotides
AU2017221441A1 (en) 2016-02-17 2018-10-04 Children's Medical Center Corporation FFA1 (GPR40) as a therapeutic target for neural angiogenesis diseases or disorders
KR20180132070A (ko) 2016-03-03 2018-12-11 큐 바이오파마, 인크. T-세포 조절 다량체 폴리펩타이드 및 이의 사용 방법
US11111505B2 (en) 2016-03-19 2021-09-07 Exuma Biotech, Corp. Methods and compositions for transducing lymphocytes and regulating the activity thereof
US11325948B2 (en) 2016-03-19 2022-05-10 Exuma Biotech Corp. Methods and compositions for genetically modifying lymphocytes to express polypeptides comprising the intracellular domain of MPL
WO2020047527A2 (en) 2018-09-02 2020-03-05 F1 Bioventures, Llc Methods and compositions for genetically modifying lymphocytes in blood or in enriched pbmcs
SG10202104509PA (en) 2016-03-19 2021-06-29 Exuma Biotech Corp Methods and compositions for transducing lymphocytes and regulated expansion thereof
BR112018070357A2 (pt) 2016-04-04 2019-01-29 Bioverativ Usa Inc anticorpos anticomplemento contra o fator bb e seus usos
WO2017182981A1 (en) 2016-04-20 2017-10-26 Washington University Ppar agonist or lxr agonist for use in the treatment of systemic lupus erythematosus by modulation of lap activity
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
SG11201808552XA (en) 2016-05-18 2018-10-30 Cue Biopharma Inc T-cell modulatory multimeric polypeptides and methods of use thereof
IL262606B2 (en) 2016-05-18 2023-04-01 Albert Einstein College Medicine Inc pd-l1 variant polypeptides, T-cell modulatory multimeric polypeptides, and methods of using them
KR102392236B1 (ko) 2016-05-18 2022-05-03 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
SG11201809643UA (en) 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
WO2017214518A1 (en) 2016-06-10 2017-12-14 Alnylam Pharmaceuticals, Inc. COMPLETMENT COMPONENT C5 iRNA COMPOSTIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
AU2017292936C1 (en) 2016-07-08 2024-05-02 Exuma Biotech, Corp. Methods and compositions for transducing lymphocytes and regulating the activity thereof
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
CN109982710A (zh) 2016-09-13 2019-07-05 杰克逊实验室 靶向增强的dna去甲基化
WO2018057648A1 (en) 2016-09-20 2018-03-29 The Board Of Trustees Of The Leland Stanford Junior University Peptide regulators of mitochondrial fusion and methods of use
EP3299460A1 (en) 2016-09-26 2018-03-28 Johann Wolfgang Goethe-Universität Frankfurt am Main Novel compounds and methods for modulating ubiquitination
MX2019004259A (es) 2016-10-12 2019-09-27 Bioverativ Usa Inc Anticuerpos anti-c1s y metodos de uso de los mismos.
US11154591B2 (en) 2016-10-14 2021-10-26 The Trustees Of Columbia University In The City Of New York Methods of treating alcohol abuse disorder
US11332713B2 (en) 2016-11-16 2022-05-17 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
TWI790217B (zh) 2016-12-16 2023-01-21 美商阿尼拉製藥公司 使用甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物於治療或預防ＴＴＲ相關疾病之方法
IL297617B2 (en) 2016-12-22 2023-11-01 Cue Biopharma Inc Multimeric polypeptides modulate T cells and methods for their use
EP3565829A4 (en) 2017-01-09 2021-01-27 Cue Biopharma, Inc. MULTIMER POLYPEPTIDES T-LYMPHOCYTE MODULATORS AND THEIR METHODS OF USE
EP3571294A1 (en) 2017-01-18 2019-11-27 F1 Oncology, Inc. Methods of transducing and expanding immune cells and uses thereof
KR20190130559A (ko) 2017-01-18 2019-11-22 에프1 온콜로지, 인코포레이티드 Axl 또는 ror2에 대한 키메라 항원 수용체 및 이의 사용 방법
AU2018215585B2 (en) 2017-01-31 2022-03-17 Pfizer Inc. Neisseria meningitidis compositions and methods thereof
CA3054064A1 (en) 2017-03-03 2018-09-07 F1 Oncology, Inc. Methods and compositions for transducing and expanding lymphocytes and regulating the activity thereof
WO2018204803A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
CN110913866A (zh) 2017-05-05 2020-03-24 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
GB201710973D0 (en) 2017-07-07 2017-08-23 Avacta Life Sciences Ltd Scaffold proteins
WO2019018342A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. NETWORK EQUIPMENT TRACK GUIDE SYSTEM
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Ａａｖを送達するための組成物および方法
JP2020534352A (ja) 2017-09-07 2020-11-26 キュー バイオファーマ，インコーポレーテッド コンジュゲーション部位を有するｔ細胞調節多量体ポリペプチド及びその使用方法
JP2020534811A (ja) 2017-09-08 2020-12-03 マベリック セラピューティクス， インコーポレイテッドＭａｖｅｒｉｃｋ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｉｎｃ． Ｆｃ領域を含有する条件的に活性化された結合部分
EP3679067A2 (en) 2017-09-08 2020-07-15 Maverick Therapeutics, Inc. Constrained conditionally activated binding proteins
CN117737009A (zh) 2017-10-10 2024-03-22 南特生物科学公司 对病毒生产有效载荷具有低毒性的经修饰的ec7细胞
CN111527105A (zh) 2017-10-11 2020-08-11 美国比奥维拉迪维股份有限公司 诱导补体活性的方法
JP7502991B2 (ja) 2017-10-16 2024-06-19 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症（ａｌｓ）の治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CA3079437A1 (en) * 2017-10-18 2019-04-25 City Of Hope Adeno-associated virus compositions for restoring hbb gene function and methods of use thereof
WO2019089922A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
EP3714054A1 (en) 2017-11-20 2020-09-30 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
MA51138A (fr) 2017-12-14 2020-10-21 Bayer Healthcare Llc Nouveaux systèmes d'endonucléases arn-programmables et leurs utilisations dans l'édition de génome et d'autres applications
US11008602B2 (en) 2017-12-20 2021-05-18 Roche Molecular Systems, Inc. Non-replicative transduction particles and transduction particle-based reporter systems
EP3737689A4 (en) 2018-01-09 2021-12-01 Cue Biopharma, Inc. MULTIMERIC T CELL-MODULATING POLYPEPTIDES AND METHOD OF USING THEREOF
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US11667949B2 (en) 2018-02-15 2023-06-06 The Trustees Of Princeton University Reporter construct and biosensor for interferon second messenger 2-5A
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RECOVERING F8 GENE FUNCTION AND METHODS OF USE THEREOF
AU2019234926A1 (en) 2018-03-15 2020-10-08 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
JP2021518139A (ja) 2018-03-19 2021-08-02 クリスパー セラピューティクス アーゲー 新規ｒｎａプログラム可能エンドヌクレアーゼ系およびその使用
JP2021533744A (ja) 2018-08-09 2021-12-09 マベリック セラピューティクス, インコーポレイテッドMaverick Therapeutics, Inc. 条件的活性化型結合タンパク質を共発現及び精製するための方法
WO2020037125A1 (en) 2018-08-16 2020-02-20 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of the lect2 gene
CN110857440B (zh) 2018-08-23 2021-02-19 武汉纽福斯生物科技有限公司 重组人ⅱ型线粒体动力蛋白样gtp酶基因序列及其应用
CA3111076A1 (en) 2018-08-30 2020-03-05 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with myocardin and ascl1
CN111118016B (zh) 2018-10-30 2022-04-15 上海朗昇生物科技有限公司 治疗视网膜色素变性疾病的基因治疗载体
WO2020104649A2 (en) 2018-11-23 2020-05-28 Sanofi Novel rna compositions and methods for inhibiting angptl8
TW202039542A (zh) 2018-12-19 2020-11-01 美商庫爾生物製藥有限公司 多聚體ｔ細胞調節多肽及其使用方法
EP3902930A1 (en) 2018-12-27 2021-11-03 F. Hoffmann-La Roche AG Non-replicative transduction particles and transduction particle-based reporter systems for detection of acinetobacter baumannii
US11572595B2 (en) 2018-12-31 2023-02-07 Roche Molecular Systems, Inc. Non-replicative transduction particles with one or more non-native tail fibers and transduction particle-based reporter systems
CN114390938A (zh) 2019-03-05 2022-04-22 武田药品工业有限公司 受约束的条件性活化的结合蛋白
AU2020231391A1 (en) 2019-03-05 2021-10-28 Takeda Pharmaceutical Company Limited Conditionally activated binding proteins containing Fc regions and moieties targeting tumor antigens
EP3935080A4 (en) 2019-03-06 2023-04-05 Cue Biopharma, Inc. T-CELL-MODULATING MULTIMERIC POLYPEPTIDES AND METHODS OF USE THEREOF
MX2021010559A (es) 2019-03-07 2021-12-15 Univ California Polipéptidos efectores de crispr-cas y métodos de uso de estos.
WO2020186059A2 (en) 2019-03-12 2020-09-17 Crispr Therapeutics Ag Novel high fidelity rna-programmable endonuclease systems and uses thereof
CN111718418B (zh) 2019-03-19 2021-08-27 华东师范大学 一种增强基因编辑的融合蛋白及其应用
MX2021012003A (es) 2019-04-02 2021-11-04 Kenjockety Biotechnology Inc Anticuerpos multiespecificos contra antigeno de cancer de la bomba de eflujo y composiciones, reactivos, kits y metodos relacionados con los mismos.
US20220211741A1 (en) 2019-04-18 2022-07-07 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment and prognosis of cancer
EP3736330A1 (en) 2019-05-08 2020-11-11 European Molecular Biology Laboratory Modified adeno-associated virus (aav) particles for gene therapy
CN112180094A (zh) 2019-07-04 2021-01-05 上海东慈生物科技有限公司 Dkk1抑制剂在预防和/或治疗肿瘤恶病质与糖尿病伴随疾病中的应用
US20230137971A1 (en) 2019-07-11 2023-05-04 Tenaya Therapeutics Inc. Cardiac cell reprogramming with micrornas and other factors
US20220296926A1 (en) 2019-07-31 2022-09-22 Memorial Sloan Kettering Cancer Center Perfusion modulated tumor dose sculpting with single dose radiotherapy
BR112022002406A2 (pt) 2019-08-12 2022-07-19 Purinomia Biotech Inc Métodos e composições para promover e potencializar respostas imunes mediadas por célula t através do alvejamento de adcc de células que expressam cd39
EP4039801A4 (en) 2019-08-16 2023-10-18 Center For Excellence In Brain Science And Intelligence Technology, Chinese Academy Of Sciences USE OF PTBP1 INHIBITORS TO PREVENT AND/OR TREAT NERVOUS SYSTEM DISEASES ASSOCIATED WITH FUNCTIONAL NEURONAL DEATH
WO2021037972A1 (en) 2019-08-27 2021-03-04 Sanofi Compositions and methods for inhibiting pcsk9
JP2022546570A (ja) 2019-09-03 2022-11-04 アルナイラム ファーマシューティカルズ， インコーポレイテッド Ｌｅｃｔ２遺伝子の発現を阻害するための組成物および方法
WO2021067747A1 (en) 2019-10-04 2021-04-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
WO2021074695A1 (en) 2019-10-16 2021-04-22 Avacta Life Sciences Limited PD-L1 INHIBITOR - TGFβ INHIBITOR BISPECIFIC DRUG MOIETIES.
WO2021087325A1 (en) 2019-11-01 2021-05-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
EP4058577A1 (en) 2019-11-13 2022-09-21 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
WO2021113736A1 (en) 2019-12-05 2021-06-10 Massachusetts Institute Of Technology Single-domain antibody to chloramphenicol
US20220356537A1 (en) 2019-12-31 2022-11-10 Roche Molecular Systems, Inc. Quantitative pcr screening of inducible prophage from bacterial isolates
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
TW202140786A (zh) 2020-02-10 2021-11-01 美商艾爾妮蘭製藥公司 用於靜默ｖｅｇｆ－ａ表現之組合物及方法
IL295989A (en) 2020-03-02 2022-10-01 Tenaya Therapeutics Inc Gene vector control using cardiomyocyte expression microRNA
GB202004498D0 (en) 2020-03-27 2020-05-13 Ucl Business Ltd Activity-dependent gene therapy for neurological disorders
MX2022012209A (es) 2020-03-30 2022-10-27 Wuhan Neurophth Biotechnology Ltd Company Vector de expresion del factor 2 relacionado con el factor nuclear humano e2 y aplicacion del vector de expresion.
US20230190785A1 (en) 2020-03-30 2023-06-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
US20230295622A1 (en) 2020-04-06 2023-09-21 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
EP4133079A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
US20230183707A1 (en) 2020-05-21 2023-06-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
WO2021247423A1 (en) 2020-06-04 2021-12-09 Kenjockety Biotechnology, Inc. Abcg2 efflux pump-cancer antigen multi-specific antibodies and compositions, reagents, kits and methods related thereto
GB202101299D0 (en) 2020-06-09 2021-03-17 Avacta Life Sciences Ltd Diagnostic polypetides and methods
GB202010981D0 (en) 2020-07-16 2020-09-02 Ucl Business Ltd Gene therapy for neuromuscular and neuromotor disorders
US20230295615A1 (en) 2020-08-07 2023-09-21 The Jackson Laboratory Targeted Sequence Insertion Compositions and Methods
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
EP4229200A1 (en) 2020-10-16 2023-08-23 Sanofi Novel rna compositions and methods for inhibiting angptl3
CN116490195A (zh) 2020-10-16 2023-07-25 赛诺菲 用于抑制脂蛋白(a)的RNA组合物和方法
CA3198823A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
KR20230107619A (ko) 2020-11-11 2023-07-17 유럽피안 몰레큘러 바이올로지 래보러토리 유전자 치료를 위한 변형된 바이러스 입자
WO2022119873A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
WO2022187289A1 (en) 2021-03-01 2022-09-09 Exuma Biotech Corp. Methods and compositions for the delivery of retroviral particles
JP2024516400A (ja) 2021-04-30 2024-04-15 カリヴィル イムノセラピューティクス， インコーポレイテッド 修飾されたｍｈｃ発現のための腫瘍溶解性ウイルス
WO2022234003A1 (en) 2021-05-07 2022-11-10 Avacta Life Sciences Limited Cd33 binding polypeptides with stefin a protein
EP4101928A1 (en) 2021-06-11 2022-12-14 Bayer AG Type v rna programmable endonuclease systems
BR112023023768A2 (pt) 2021-06-11 2024-02-27 Bayer Ag Sistemas de endonucleases programáveis por rna tipo v
EP4363580A1 (en) 2021-06-30 2024-05-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
EP4144841A1 (en) 2021-09-07 2023-03-08 Bayer AG Novel small rna programmable endonuclease systems with impoved pam specificity and uses thereof
WO2023057946A1 (en) 2021-10-07 2023-04-13 Avacta Life Sciences Limited Serum half-life extended pd-l1 binding polypeptides
TW202334196A (zh) 2021-10-07 2023-09-01 英商阿法克塔生命科學有限公司 Pd-l1結合多肽
WO2023102388A1 (en) 2021-11-30 2023-06-08 Sanofi Pasteur Inc. Human metapneumovirus viral vector-based vaccines
WO2023114658A1 (en) 2021-12-13 2023-06-22 Kenjockety Biotechnology, Inc. Anti-abcb1 antibodies
WO2023118068A1 (en) 2021-12-23 2023-06-29 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
WO2023131682A1 (en) 2022-01-06 2023-07-13 Ucl Business Ltd Endogenous gene regulation to treat neurological disorders and diseases
GB202201744D0 (en) 2022-02-10 2022-03-30 Ucl Business Ltd Treatment of acquired focal epilepsy
WO2023159220A1 (en) 2022-02-18 2023-08-24 Kenjockety Biotechnology, Inc. Anti-cd47 antibodies
WO2023168305A1 (en) 2022-03-01 2023-09-07 Exuma Biotech Corp. Viral particles with membrane-bound hyaluronidase
WO2023222829A1 (en) 2022-05-17 2023-11-23 Centre Hospitalier Universitaire Vaudois Designed biosensors for enhanced t cell therapy
WO2023237587A1 (en) 2022-06-10 2023-12-14 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
EP4299733A1 (en) 2022-06-30 2024-01-03 Inari Agriculture Technology, Inc. Compositions, systems, and methods for genome editing
EP4299739A1 (en) 2022-06-30 2024-01-03 Inari Agriculture Technology, Inc. Compositions, systems, and methods for genome editing
WO2024005864A1 (en) 2022-06-30 2024-01-04 Inari Agriculture Technology, Inc. Compositions, systems, and methods for genome editing
WO2024005863A1 (en) 2022-06-30 2024-01-04 Inari Agriculture Technology, Inc. Compositions, systems, and methods for genome editing
WO2024044659A1 (en) 2022-08-24 2024-02-29 Tectonic Therapeutic, Inc. Constitutively active g protein-coupled receptor compositions and methods of use thereof
WO2024068996A1 (en) 2022-09-30 2024-04-04 Centre Hospitalier Universitaire Vaudois (C.H.U.V.) Anti-sars-cov-2 antibodies and use thereof in the treatment of sars-cov-2 infection
WO2024098061A2 (en) 2022-11-04 2024-05-10 Genkardia Inc. Oligonucleotide-based therapeutics targeting cyclin d2 for the treatment of heart failure
WO2024105633A1 (en) 2022-11-18 2024-05-23 Kyoto Prefectural Public University Corporation Compositions for mitophagy induction and uses thereof
WO2024108217A1 (en) 2022-11-18 2024-05-23 Genkardia Inc. Methods and compositions for preventing, treating, or reversing cardiac diastolic dysfunction

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) * 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
WO1988008450A1 (en) * 1987-05-01 1988-11-03 Birdwell Finlayson Gene therapy for metabolite disorders
AU7906691A (en) * 1990-05-23 1991-12-10 United States of America, as represented by the Secretary, U.S. Department of Commerce, The Adeno-associated virus (aav)-based eucaryotic vectors
US5173414A (en) * 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors

Also Published As

Publication number Publication date
CA2098483A1 (en) 1993-05-09
CA2098483C (en) 2000-03-14
JPH06504680A (ja) 1994-06-02
DK0566732T3 (da) 2003-07-21
US5252479A (en) 1993-10-12
PT101040A (pt) 1994-05-31
AU657829B2 (en) 1995-03-23
AU3134093A (en) 1993-06-07
DE69232983T2 (de) 2003-11-13
PT101040B (pt) 2003-06-30
DE69232983D1 (de) 2003-05-08
WO1993009239A1 (en) 1993-05-13
JP2003174896A (ja) 2003-06-24
ATE236263T1 (de) 2003-04-15
EP0566732A1 (en) 1993-10-27
ES2192555T3 (es) 2003-10-16
EP0566732B1 (en) 2003-04-02
IL103677A0 (en) 1993-04-04

Similar Documents

Publication Publication Date Title
KR930703453A (ko) 유전자 치료법을 위한 안전한 벡터
Flotte et al. Expression of the cystic fibrosis transmembrane conductance regulator from a novel adeno-associated virus promoter.
CN111278972B (zh) 用于细胞遗传修饰的非整合型dna载体
Verma et al. Gene therapy-promises, problems and prospects
ES2204922T3 (es) Terapia genica anticancerosa por modulacion de la respuesta inmunitaria y/o inflamatoria.
US5780447A (en) Recombinant adeno-associated viral vectors
RU2004113452A (ru) Способы и композиции, относящиеся к лентивирусным векторам с ограниченной экспрессией и к их применениям
CN107532168A (zh) 治疗血红蛋白病的材料和方法
JP2003504061A (ja) プラスミド配列の転写の間における異常型ｒｎａの形成を防止するための方法および組成物
JP2004500858A (ja) 二本鎖パルボウイルスベクター
EP1204739A2 (en) Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
JP2013509168A (ja) 心臓特異的核酸調節因子ならびにこの方法および使用
CN112725342B (zh) 一种启动子pCALM2及其应用
KR19980071443A (ko) 구조 유전자의 세포 주기 조절된 발현을 위한 핵산 작제물
ES2215222T3 (es) Modificacion genetica de celulas cepa de repoblacion hematopoyeticas de primates.
CN111235158A (zh) 用于表达重组人β-珠蛋白的病毒载体及其应用
Sultana et al. In vitro synthesis of modified RNA for cardiac gene therapy
Watt et al. A review of gene transfer techniques
Li et al. Targeted correction of the point mutations of β-thalassemia and targeted mutagenesis of the nucleotide associated with HPFH by RNA/DNA oligonucleotides: potential for β-thalassemia gene therapy
Jane et al. Vector development: a major obstacle in human gene therapy
Linden et al. AAVant-garde gene therapy
WO2022251097A1 (en) Engineered guide rnas and polynucleotides
Samara et al. Molecular biology and therapy of disease
Villar et al. Molecular intensive care medicine
Nienhuis et al. Gene transfer into hematopoietic cells

Legal Events

Date Code Title Description
A201 Request for examination
E902 Notification of reason for refusal
E601 Decision to refuse application