RU2468820C2 - Способы и композиции для лечения заболевания - Google Patents

Способы и композиции для лечения заболевания Download PDF

Info

Publication number
RU2468820C2
RU2468820C2 RU2009103208/15A RU2009103208A RU2468820C2 RU 2468820 C2 RU2468820 C2 RU 2468820C2 RU 2009103208/15 A RU2009103208/15 A RU 2009103208/15A RU 2009103208 A RU2009103208 A RU 2009103208A RU 2468820 C2 RU2468820 C2 RU 2468820C2
Authority
RU
Russia
Prior art keywords
cells
polynucleotide
specified
promoter
subject
Prior art date
Application number
RU2009103208/15A
Other languages
English (en)
Russian (ru)
Other versions
RU2009103208A (ru
Inventor
Томас Д. РИД
Роберт П. БИЧ
Original Assignee
Интрексон Корпорейшн
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 Интрексон Корпорейшн filed Critical Интрексон Корпорейшн
Publication of RU2009103208A publication Critical patent/RU2009103208A/ru
Application granted granted Critical
Publication of RU2468820C2 publication Critical patent/RU2468820C2/ru

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/711Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/0081Purging biological preparations of unwanted cells
    • C12N5/0093Purging against cancer cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45Transferases (2)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • A61P35/02Antineoplastic agents specific for leukemia
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0647Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/70Enzymes
    • C12N2501/72Transferases [EC 2.]
    • C12N2501/724Glycosyltransferases (EC 2.4.)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00Genetically modified cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y204/00Glycosyltransferases (2.4)
    • C12Y204/02Pentosyltransferases (2.4.2)
    • C12Y204/02036NAD(+)--diphthamide ADP-ribosyltransferase (2.4.2.36)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Oncology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Developmental Biology & Embryology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
RU2009103208/15A 2006-07-26 2007-07-26 Способы и композиции для лечения заболевания RU2468820C2 (ru)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US82038106P 2006-07-26 2006-07-26
US60/820,381 2006-07-26
US88909507P 2007-02-09 2007-02-09
US60/889,095 2007-02-09
PCT/US2007/016747 WO2008073154A2 (en) 2006-07-26 2007-07-26 Methods and compositions for treating disease

Publications (2)

Publication Number Publication Date
RU2009103208A RU2009103208A (ru) 2010-09-10
RU2468820C2 true RU2468820C2 (ru) 2012-12-10

Family

ID=39512236

Family Applications (1)

Application Number Title Priority Date Filing Date
RU2009103208/15A RU2468820C2 (ru) 2006-07-26 2007-07-26 Способы и композиции для лечения заболевания

Country Status (16)

Country Link
US (3) US20100003226A1 (enExample)
EP (1) EP2043662B1 (enExample)
JP (1) JP2009544711A (enExample)
KR (1) KR20090035011A (enExample)
AU (1) AU2007332980A1 (enExample)
CA (1) CA2658836C (enExample)
DK (1) DK2043662T3 (enExample)
ES (1) ES2553332T3 (enExample)
HU (1) HUE027142T2 (enExample)
IL (1) IL196638A (enExample)
MX (1) MX2009000966A (enExample)
NZ (1) NZ575075A (enExample)
PL (1) PL2043662T3 (enExample)
PT (1) PT2043662E (enExample)
RU (1) RU2468820C2 (enExample)
WO (1) WO2008073154A2 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013109944A1 (en) * 2012-01-18 2013-07-25 The Trustees Of The University Of Pennsylvania Methods for assessing risk for cancer using biomarkers
EA029382B1 (ru) 2012-05-09 2018-03-30 Кантекс Фармасьютикалз, Инк. Лечение миелосупрессии
EP3258941A4 (en) 2015-02-17 2018-09-26 Cantex Pharmaceuticals, Inc. Treatment of cancers and hematopoietic stem cell disorders privileged by cxcl12-cxcr4 interaction
WO2016133907A1 (en) * 2015-02-17 2016-08-25 Cantex Pharmaceuticals, Inc. Adoptive cell transfer methods
KR20180069081A (ko) 2015-11-11 2018-06-22 인트렉손 코포레이션 심장 이상 및 기타 병리 이상의 치료를 위한 복수의 생물학적으로 활성화된 폴리펩티드를 단일 벡터로부터 발현하기 위한 조성물 및 방법
JP7051060B2 (ja) * 2017-05-09 2022-04-11 学校法人慶應義塾 脳腫瘍治療用細胞製剤

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999042593A1 (en) * 1998-02-19 1999-08-26 St. Jude Children's Research Hospital Compositions and methods for sensitizing and inhibiting growth of human tumor cells
US20020150557A1 (en) * 1998-10-15 2002-10-17 Muralidhara Ramachandra Selectively replicating viral vectors
US20030224521A1 (en) * 2000-08-14 2003-12-04 The Gov. Of The Usa Secretary Of The Department Of Health And Human Services Enhanced homologous recombination mediated by lambda recombination proteins
US20050220765A1 (en) * 2002-01-14 2005-10-06 Van Beusechem Victor W Viruses with enhanced lytic potency

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5830686A (en) * 1994-01-13 1998-11-03 Calydon Tissue-specific enhancer active in prostate
EP1188448A3 (en) * 1994-03-15 2002-04-17 Prizm Pharmaceuticals, Inc. Heparin-binding growth factors for gene therapy and anterior eye disorders
WO1996036362A1 (en) * 1995-05-16 1996-11-21 Prizm Pharmaceuticals, Inc. Compositions containing nucleic acids and ligands for therapeutic treatment
WO1997049434A2 (en) * 1996-06-24 1997-12-31 Prizm Pharmaceuticals, Inc. Heparinized medical devices containing heparin-binding growth factor conjugates
DE60043889D1 (en) * 1999-06-07 2010-04-08 Tet Systems Holding Gmbh & Co Tet repressor basierte transkriptionale regulatorische proteine
JP5031967B2 (ja) 2000-03-22 2012-09-26 イントレキソン コーポレーション 新規エクジソン受容体ベースの誘導性遺伝子発現系
US20040033600A1 (en) 2001-03-21 2004-02-19 Palli Subba Reddy Ecdysone receptor-based inducible gene expression system
US8105825B2 (en) 2000-10-03 2012-01-31 Intrexon Corporation Multiple inducible gene regulation system
CA2435503C (en) * 2001-01-19 2011-02-22 Ludwig Institute For Cancer Research Flt4(vegfr-3) as a target for tumor imaging and anti-tumor therapy
ES2422303T3 (es) 2001-02-20 2013-09-10 Intrexon Corp Receptores mutantes por sustitución novedosos y su uso en un sistema de expresión génica inducible basado en receptores nucleares
CA2438119C (en) 2001-02-20 2014-12-16 Rheogene Holdings, Inc. Chimeric retinoid x receptors and their use in a novel ecdysone receptor-based inducible gene expression system
AU2002248500B2 (en) 2001-02-20 2007-12-13 Intrexon Corporation Novel substitution mutant receptors and their use in a nuclear receptor-based inducible gene expression system
ES2385598T3 (es) 2001-02-20 2012-07-27 Intrexon Corporation Sistema novedoso de expresión génica inducible basado en el receptor de ecdisona/receptor X retinoide de invertebrado
US6828102B2 (en) * 2001-11-20 2004-12-07 Albany Medical College Plasmids and methods for monitoring endonuclease digestion efficiency
EP1529110B1 (en) * 2002-08-01 2011-10-05 Evolva Ltd. Methods of mixing large numbers of heterologous genes
US7785871B2 (en) 2002-10-09 2010-08-31 Intrexon Corporation DNA cloning vector plasmids and methods for their use
EP1631319A4 (en) * 2003-05-30 2007-10-31 Cleveland Clinic Foundation IN VIVO PRODUCTION OF A CLOSTRIDIUM NEUROTOXIN LIGHT CHAIN PEPTIDE
CA2436196A1 (en) * 2003-07-25 2005-01-25 Oncolytics Biotech Inc. Oncolytic virus for purging cellular compositions of cells of lymphoid malignancies
US7935510B2 (en) 2004-04-30 2011-05-03 Intrexon Corporation Mutant receptors and their use in a nuclear receptor-based inducible gene expression system
AU2005248371B2 (en) 2004-05-18 2011-12-15 Intrexon Corporation Methods for dynamic vector assembly of DNA cloning vector plasmids

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999042593A1 (en) * 1998-02-19 1999-08-26 St. Jude Children's Research Hospital Compositions and methods for sensitizing and inhibiting growth of human tumor cells
US20020150557A1 (en) * 1998-10-15 2002-10-17 Muralidhara Ramachandra Selectively replicating viral vectors
US20030224521A1 (en) * 2000-08-14 2003-12-04 The Gov. Of The Usa Secretary Of The Department Of Health And Human Services Enhanced homologous recombination mediated by lambda recombination proteins
US20050220765A1 (en) * 2002-01-14 2005-10-06 Van Beusechem Victor W Viruses with enhanced lytic potency

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
MITANI K. et al. Adenovirus as an integrating vector. Curr Gene Ther. 2002 May; 2(2): 135-144. *

Also Published As

Publication number Publication date
CA2658836A1 (en) 2008-06-19
US20170191027A1 (en) 2017-07-06
US20100003226A1 (en) 2010-01-07
IL196638A (en) 2013-11-28
US20150132265A1 (en) 2015-05-14
KR20090035011A (ko) 2009-04-08
RU2009103208A (ru) 2010-09-10
EP2043662A4 (en) 2010-04-14
WO2008073154A2 (en) 2008-06-19
ES2553332T3 (es) 2015-12-07
CA2658836C (en) 2017-11-28
JP2009544711A (ja) 2009-12-17
NZ575075A (en) 2011-10-28
IL196638A0 (en) 2009-11-18
PL2043662T3 (pl) 2016-03-31
MX2009000966A (es) 2009-03-05
AU2007332980A1 (en) 2008-06-19
HK1129596A1 (en) 2009-12-04
PT2043662E (pt) 2015-11-25
HUE027142T2 (en) 2016-08-29
WO2008073154A3 (en) 2008-12-04
EP2043662A2 (en) 2009-04-08
EP2043662B1 (en) 2015-09-09
DK2043662T3 (en) 2015-12-14

Similar Documents

Publication Publication Date Title
US9816074B2 (en) Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
JP6873917B2 (ja) ヌクレアーゼ介在性遺伝子発現調節
AU2022200835A1 (en) Methods And Compositions For Nuclease-Mediated Genome Engineering And Correction In Hematopoietic Stem Cells
US20170191027A1 (en) Methods and Compositions For Treating Disease
JP7558814B2 (ja) 遺伝子治療
US20200316116A1 (en) Method for the treatment of beta-thalassemia
Matsui et al. Caspase 9-induced apoptosis enables efficient fetal cell ablation and disease modeling
KR101658135B1 (ko) 혈액 응고인자 ⅷ 유전자를 타겟으로 하는 엔도뉴클레아제 및 이의 용도
HK1129596B (en) Methods and compositions for treating disease
CN101516383A (zh) 治疗疾病的方法和组合物
CN112218945A (zh) 纯合细胞的制作方法
CA3237598A1 (en) Nucleoside modified mrna and uses thereof
Shin et al. Establishment and characterization of nine human brain tumor cell lines
WO2025088508A1 (en) Means and methods for genetic modification of uba1
Ma et al. Real-time bioluminescence imaging of polycythemia vera development in mice
JP2004166590A (ja) 酸化プリンヌクレオシド三リン酸分解酵素阻害剤のスクリーニング方法
Cui et al. The Polycomb Protein Bmi1 is a Key Effector of the H3. 3 K27m Oncohistone

Legal Events

Date Code Title Description
TK4A Correction to the publication in the bulletin (patent)

Free format text: AMENDMENT TO CHAPTER -FG4A- IN JOURNAL: 34-2012 FOR TAG: (57)

MM4A The patent is invalid due to non-payment of fees

Effective date: 20200727