CN112153973A - 用于干细胞移植的组合物和方法 - Google Patents

用于干细胞移植的组合物和方法 Download PDF

Info

Publication number
CN112153973A
CN112153973A CN201980018070.0A CN201980018070A CN112153973A CN 112153973 A CN112153973 A CN 112153973A CN 201980018070 A CN201980018070 A CN 201980018070A CN 112153973 A CN112153973 A CN 112153973A
Authority
CN
China
Prior art keywords
cells
cell population
enriched cell
stringency
gene
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN201980018070.0A
Other languages
English (en)
Chinese (zh)
Inventor
B·彼尔德
J·塞维拉纳瓦罗
G·萨
K·帕特尔
R·普拉布哈卡
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus
Rocket Pharmaceuticals Inc
Original Assignee
Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus
Rocket Pharmaceuticals Inc
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 Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus, Rocket Pharmaceuticals Inc filed Critical Fundacion Para La Investigacion Biomedica Del Hospital Infantil Universitario Nino Jesus
Publication of CN112153973A publication Critical patent/CN112153973A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2510/00—Genetically modified cells
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Hematology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (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)
CN201980018070.0A 2018-04-11 2019-04-11 用于干细胞移植的组合物和方法 Pending CN112153973A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862656292P 2018-04-11 2018-04-11
US62/656,292 2018-04-11
PCT/US2019/027083 WO2019200167A1 (en) 2018-04-11 2019-04-11 Compositions and methods for stem cell transplant

Publications (1)

Publication Number Publication Date
CN112153973A true CN112153973A (zh) 2020-12-29

Family

ID=68162990

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201980018070.0A Pending CN112153973A (zh) 2018-04-11 2019-04-11 用于干细胞移植的组合物和方法

Country Status (11)

Country Link
US (1) US12539316B2 (https=)
JP (2) JP7576754B2 (https=)
KR (1) KR20210003738A (https=)
CN (1) CN112153973A (https=)
AU (2) AU2019252662A1 (https=)
BR (1) BR112020020841A2 (https=)
CA (1) CA3093708A1 (https=)
IL (2) IL307996A (https=)
MX (1) MX2020010718A (https=)
SG (1) SG11202008400SA (https=)
WO (1) WO2019200167A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE055862T2 (hu) 2016-04-20 2021-12-28 Centro De Investig Energeticas Készítmények és eljárások PKLR fokozott génexpressziójára
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
WO2019200167A1 (en) 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for stem cell transplant
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
CN119264211A (zh) 2018-08-27 2025-01-07 瑞泽恩制药公司 拉曼光谱在下游纯化中的应用
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
JP2024502820A (ja) * 2020-12-31 2024-01-23 ブイオーアール バイオファーマ インコーポレーテッド Ｃｄ３４遺伝子改変のための組成物及び方法
US20250221931A1 (en) * 2022-03-25 2025-07-10 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104419683A (zh) * 2013-09-10 2015-03-18 中国科学院生物物理研究所 制备范可尼贫血症患者自体诱导多能干细胞的方法及其应用
WO2016118780A1 (en) * 2015-01-21 2016-07-28 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
WO2017218948A2 (en) * 2016-06-17 2017-12-21 Fred Hutchinson Cancer Research Center Strategies to assess and/or produce cell populations with predictive engraftment potential

Family Cites Families (84)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2779192B2 (ja) 1987-09-17 1998-07-23 マサチユセツツ・インスチチユート・オブ・テクノロジー ヒト赤血球特異的転写エンハンサー
US5278056A (en) 1988-02-05 1994-01-11 The Trustees Of Columbia University In The City Of New York Retroviral packaging cell lines and process of using same
DE69434860T2 (de) 1993-02-22 2007-03-15 The Rockefeller University Herstellung von helfer-freien retroviren mit hohem titer mittels transienter transfektion
FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
AU687117B2 (en) 1993-10-25 1998-02-19 Canji, Inc. Recombinant adenoviral vector and methods of use
US5484720A (en) 1994-09-08 1996-01-16 Genentech, Inc. Methods for calcium phosphate transfection
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US6027721A (en) 1996-05-20 2000-02-22 Cytotherapeutics, Inc. Device and method for encapsulated gene therapy
US5952190A (en) 1996-10-04 1999-09-14 Fanconi Anemia Research Fund, Inc. cDNA for fanconi anemia complementation group A
US6136597A (en) 1997-09-18 2000-10-24 The Salk Institute For Biological Studies RNA export element
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US20020065236A1 (en) 1998-09-09 2002-05-30 Yew Nelson S. CpG reduced plasmids and viral vectors
WO2001088088A2 (en) 2000-05-18 2001-11-22 Hyseq, Inc. Novel nucleic acids and polypeptides
US7994278B1 (en) 1999-08-06 2011-08-09 Nobel Biosciences Llc Biologically active polypeptides derived from a novel early stage pregnancy factor designated maternin (MA)
HK1048302A1 (zh) 1999-08-13 2003-03-28 大正制药株式会社 前列腺素衍生物
HK1050210B (en) 1999-10-12 2007-08-24 Institut Pasteur Lentiviral triplex dna, and vectors and recombinant cells containing lentiviral triplex dna
US20020013294A1 (en) 2000-03-31 2002-01-31 Delong Mitchell Anthony Cosmetic and pharmaceutical compositions and methods using 2-decarboxy-2-phosphinico derivatives
US6627442B1 (en) 2000-08-31 2003-09-30 Virxsys Corporation Methods for stable transduction of cells with hiv-derived viral vectors
GB0024550D0 (https=) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
US7575924B2 (en) 2000-11-13 2009-08-18 Research Development Foundation Methods and compositions relating to improved lentiviral vectors and their applications
ATE527347T1 (de) 2001-08-02 2011-10-15 Inst Clayton De La Rech Verfahren und zusammensetzungen im zusammenhang mit verbesserten lentivirusvektor- produktionssystemen
RU2305708C2 (ru) 2001-10-02 2007-09-10 Энститю Клейтон Де Ля Решерш Рекомбинантный лентивирусный вектор, клетка-хозяин, трансдуцированная лентивирусным вектором, способ ее трансдукции и применение
AU2003223661A1 (en) 2002-04-15 2003-11-03 Rigel Pharmaceuticals, Inc. Methods of assaying for cell cycle modulators
CA2483915A1 (en) 2002-05-02 2003-11-13 University Of Rochester Vectors having both isoforms of b-hexosaminidase
ES2298745T3 (es) 2003-04-24 2008-05-16 Fondazione Centro San Raffaele Del Monte Tabor Promotores bidireccionales de sintesis y utilizacion de los mismos.
US20070048285A1 (en) 2005-08-24 2007-03-01 Christopher Baum Self-inactivating retroviral vector
BE1016793A4 (fr) 2005-10-04 2007-06-05 Artelis Procede de culture de cellules et dispositif permettant sa mise en oeuvre.
US8324367B2 (en) 2006-11-03 2012-12-04 Medtronic, Inc. Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
WO2008136670A2 (en) 2007-05-02 2008-11-13 Erasmus University Medical Center Rotterdam Improved methods and means for lentiviral gene delivery
EP2333091B1 (en) 2007-05-29 2017-06-21 Nature Technology Corporation Vectors and methods for genetic immunization
ES2660180T3 (es) 2007-12-07 2018-03-21 Miltenyi Biotec Gmbh Sistemas y métodos para procesamiento de células
DE102008063606A1 (de) 2008-12-18 2010-06-24 Eufets Ag Ein-Vektor Tet-System und dessen Verwendung
WO2010126766A1 (en) 2009-04-30 2010-11-04 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Inducible interleukin-12
DE102009021592A1 (de) 2009-05-15 2010-11-18 Medizinische Hochschule Hannover ASLV-Vektorsystem
WO2010138750A2 (en) 2009-05-27 2010-12-02 The Salk Institute For Biological Studies Generation of genetically corrected disease-free induced pluripotent stem cells
JP5996429B2 (ja) 2009-09-03 2016-09-21 ジェネンテック， インコーポレイテッド 関節リウマチの治療、診断及びモニターするための方法
WO2011043719A1 (en) 2009-10-05 2011-04-14 Ya-Fang Mei Replicating viral vectors for gene therapy
KR20140045411A (ko) 2011-05-16 2014-04-16 젠자임 코포레이션 Ｃｘｃｒ４ 길항제의 용도
EP2820138B1 (en) 2012-02-29 2020-10-28 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Retroviral transduction using poloxamers
AU2013293270B2 (en) 2012-07-25 2018-08-16 Massachusetts Institute Of Technology Inducible DNA binding proteins and genome perturbation tools and applications thereof
EP2931871B1 (en) 2012-12-11 2026-03-04 Pall Technology UK limited Recipient for cell cultivation
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
WO2014100073A2 (en) 2012-12-21 2014-06-26 Merck Sharp & Dohme Corp. Expression vectors for recombinant protein production in mammalian cells
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
GB201318347D0 (en) 2013-10-16 2013-11-27 Ucl Business Plc Retroviral vectors
WO2015073922A2 (en) 2013-11-15 2015-05-21 Northwestern University Inhibition of oxidative stress in atrial fibrillation
US20170037431A1 (en) 2014-05-01 2017-02-09 University Of Washington In vivo Gene Engineering with Adenoviral Vectors
US20170175143A1 (en) * 2014-05-20 2017-06-22 Regents Of The University Of Minnesota Method for editing a genetic sequence
WO2015188191A1 (en) 2014-06-06 2015-12-10 Wong Wilson W Dna recombinase circuits for logical control of gene expression
GB201417042D0 (en) 2014-09-29 2014-11-12 Fkd Therapies Oy Method
MA41346A (fr) 2015-01-12 2017-11-21 Juno Therapeutics Inc Eléments régulateurs post-transcriptionnels d'hépatite modifiée
MA41506A (fr) 2015-02-13 2017-12-19 Takeda Vaccines Inc Procédés de production de virus pour produire des vaccins
KR20240119152A (ko) 2015-03-10 2024-08-06 더 트러스티스 오브 컬럼비아 유니버시티 인 더 시티 오브 뉴욕 재조합 glut1 아데노-관련 바이러스 벡터 작제물 및 glut1 발현을 회복하기 위한 관련된 방법
WO2016183593A2 (en) 2015-05-14 2016-11-17 The Regents Of The University Of California Prenatal therapy
SG11201803934YA (en) 2015-11-11 2018-06-28 Intrexon Corp Compositions and methods for expression of multiple biologically active polypeptides from a single vector for treatment of cardiac conditions and other pathologies
PT3405215T (pt) 2016-01-19 2022-09-20 Univ California Métodos para o tratamento da doença de danon e outros transtornos da autofagia
CA3011939A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
IL261002B2 (en) 2016-02-12 2024-07-01 Bluebird Bio Inc Vcn enhancer compositions and methods of using the same
CN116144500A (zh) 2016-04-14 2023-05-23 崔泽尔有限公司 具有恒流泵/管道系统的固定床生物反应器
HUE055862T2 (hu) * 2016-04-20 2021-12-28 Centro De Investig Energeticas Készítmények és eljárások PKLR fokozott génexpressziójára
CA3058735A1 (en) 2016-06-13 2017-12-21 Bluebird Bio, Inc. Gene therapy of neuronal ceroid lipofuscinoses
KR20240151726A (ko) 2016-09-08 2024-10-18 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 판코니 빈혈 환자를 위한 유전자 치료
JP2020500562A (ja) 2016-12-06 2020-01-16 ブルーバード バイオ， インコーポレイテッド ムコ多糖症ｉｉ型のための遺伝子治療
BR112019011635A2 (pt) 2016-12-06 2019-11-12 Bluebird Bio Inc terapia genética para mucopolissacaridose, tipo i
US20180326022A1 (en) 2017-04-21 2018-11-15 The Trustees Of The University Of Pennsylvania Compositions and methods for improving heart function and treating heart failure
WO2018201065A1 (en) 2017-04-27 2018-11-01 Fred Hutchinson Cancer Research Center Therapeutic formulations containing cd34+ stem cells derived from negative selection
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
WO2019200167A1 (en) 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for stem cell transplant
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
KR102918544B1 (ko) 2018-07-12 2026-01-27 로켓 파마슈티컬스, 리미티드 다논병을 치료하기 위한 유전자 요법 벡터
KR20210046004A (ko) 2018-07-30 2021-04-27 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. 조혈 세포의 유전자 변형 방법
BR112021002765A2 (pt) 2018-08-16 2021-07-20 Spacecraft Seven, Llc métodos de produção para vetores virais
BR112021015751A2 (pt) 2019-02-12 2022-02-08 Spacecraft Seven Llc Vetores de terapia genética para tratamento da doença de danon
SG11202112347RA (en) 2019-05-23 2021-12-30 Spacecraft Seven Llc Gene therapy vectors for infantile malignant osteopetrosis
CA3177341A1 (en) 2020-05-20 2021-11-25 Chester Bittencort SACRAMENTO Engineered parkin and uses thereof
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
CN116113700A (zh) 2020-08-05 2023-05-12 太空飞船七有限责任公司 用于glut1表达的腺相关病毒载体及其用途
KR20230042468A (ko) 2020-08-05 2023-03-28 스페이스크래프트 세븐, 엘엘씨 Csrp3 (시스테인 및 글리신 풍부 단백질 3) 유전자 요법
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
CN116887868A (zh) 2020-12-07 2023-10-13 太空飞船七有限责任公司 达农病的治疗
JP2024546103A (ja) 2021-12-08 2024-12-17 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ａａｖベクターを用いたジャンクトフィリン－２（ｊｐｈ２）遺伝子治療
EP4444893A4 (en) 2021-12-10 2026-02-25 Spacecraft Seven Llc GENE THERAPY FOR TROPONIN C (TNNC1) USING AN AAV VECTOR
IL314602A (en) 2022-02-08 2024-09-01 Spacecraft Seven Llc Adeno-associated viral vector for GLUT1 expression and uses thereof
KR20250012749A (ko) 2022-04-22 2025-01-24 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 b-세포 림프종 2-관련 안타노겐 3(bag3) 유전자 요법

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104419683A (zh) * 2013-09-10 2015-03-18 中国科学院生物物理研究所 制备范可尼贫血症患者自体诱导多能干细胞的方法及其应用
WO2016118780A1 (en) * 2015-01-21 2016-07-28 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
WO2017218948A2 (en) * 2016-06-17 2017-12-21 Fred Hutchinson Cancer Research Center Strategies to assess and/or produce cell populations with predictive engraftment potential

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
MEGAN D HOBAN ET AL: "CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells", 《MOLECULAR THERAPY》, pages 1561 *
PAULA R´IO ET AL: "Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD341 cells from Fanconi anemia patients", 《BLOOD》, pages 1535 - 1542 *

Also Published As

Publication number Publication date
IL307996A (en) 2023-12-01
WO2019200167A1 (en) 2019-10-17
JP2021521180A (ja) 2021-08-26
US12539316B2 (en) 2026-02-03
EP3773636A1 (en) 2021-02-17
BR112020020841A2 (pt) 2021-01-19
KR20210003738A (ko) 2021-01-12
JP2025004171A (ja) 2025-01-14
SG11202008400SA (en) 2020-09-29
AU2026200325A1 (en) 2026-04-09
MX2020010718A (es) 2021-01-20
JP7576754B2 (ja) 2024-11-01
AU2019252662A1 (en) 2020-09-24
IL277878A (en) 2020-11-30
US20210154237A1 (en) 2021-05-27
RU2020129103A (ru) 2022-03-03
CA3093708A1 (en) 2019-10-17

Similar Documents

Publication Publication Date Title
JP2025004171A (ja) 幹細胞移植のための組成物および方法
AU2021273525B2 (en) Gene therapy for patients with Fanconi anemia
JP7601916B2 (ja) 蝸牛および前庭細胞に核酸を送達するための物質および方法
JP7817979B2 (ja) ピルビン酸キナーゼ欠損症を治療するためのｐｋｌｒ送達用レンチウイルスベクター
RU2718536C2 (ru) Способы получения и применения популяций мультипотентных и плюрипотентных клеток, устойчивых к заболеваниям и способных к дифференцировке
KR102794870B1 (ko) 핵산을 와우 및 전정 세포에 전달하기 위한 물질 및 방법
CN115323000B (zh) 使用细胞分裂基因座控制细胞增殖的工具和方法
KR20200107949A (ko) 조작된 dna 결합 단백질
AU2016343979A1 (en) Delivery of central nervous system targeting polynucleotides
US11129890B1 (en) Non-integrating HIV-1 comprising mutant RT/IN proteins and the SARS-CoV-2 spike protein
CN112888777A (zh) 用于造血细胞的基因修饰的方法
AU2018345536B2 (en) Treating diabetes with genetically modified beta cells
RU2809803C2 (ru) Композиции и способы для трансплантации стволовых клеток
RU2801241C2 (ru) Генная терапия пациентов с анемией фанкони
NL2027815B1 (en) Genomic integration
RU2777934C2 (ru) Лентивирусные векторы для доставки pklr для лечения дефицита пируваткиназы
WO2024238764A2 (en) Bioinformatically-identified control elements for regulated expression of a lentiviral vector to treat x-linked lymphoproliferative disease
HK40082332B (zh) 使用细胞分裂基因座控制细胞增殖的工具和方法
JP2024516459A (ja) 遺伝子治療のための新規プロモータ配列

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination