CN115515646A - 用于治疗代谢性肝病的组合物和方法 - Google Patents

用于治疗代谢性肝病的组合物和方法 Download PDF

Info

Publication number
CN115515646A
CN115515646A CN202180032731.2A CN202180032731A CN115515646A CN 115515646 A CN115515646 A CN 115515646A CN 202180032731 A CN202180032731 A CN 202180032731A CN 115515646 A CN115515646 A CN 115515646A
Authority
CN
China
Prior art keywords
sequence
aav
nucleic acid
seq
acid sequence
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
CN202180032731.2A
Other languages
English (en)
Chinese (zh)
Inventor
J·江
J·唐
X·李
E·M·奥斯特塔
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.)
Poseida Therapeutics Inc
Original Assignee
Poseida Therapeutics 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 Poseida Therapeutics Inc filed Critical Poseida Therapeutics Inc
Publication of CN115515646A publication Critical patent/CN115515646A/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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1018—Carboxy- and carbamoyl transferases (2.1.3)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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
    • 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
    • C12N2800/90—Vectors containing a transposable element
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/40—Vector systems having a special element relevant for transcription being an insulator
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/03—Carboxy- and carbamoyltransferases (2.1.3)
    • C12Y201/03003—Ornithine carbamoyltransferase (2.1.3.3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07—Nucleotidyltransferases (2.7.7)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN202180032731.2A 2020-03-04 2021-03-04 用于治疗代谢性肝病的组合物和方法 Pending CN115515646A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062985047P 2020-03-04 2020-03-04
US62/985047 2020-03-04
US202063121488P 2020-12-04 2020-12-04
US63/121488 2020-12-04
PCT/US2021/020929 WO2021178707A1 (en) 2020-03-04 2021-03-04 Compositions and methods for the treatment of metabolic liver disorders

Publications (1)

Publication Number Publication Date
CN115515646A true CN115515646A (zh) 2022-12-23

Family

ID=75267602

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180032731.2A Pending CN115515646A (zh) 2020-03-04 2021-03-04 用于治疗代谢性肝病的组合物和方法

Country Status (8)

Country Link
US (1) US12540335B2 (https=)
EP (1) EP4114469A1 (https=)
JP (1) JP7817176B2 (https=)
KR (1) KR20220149588A (https=)
CN (1) CN115515646A (https=)
AU (1) AU2021230361A1 (https=)
CA (1) CA3169529A1 (https=)
WO (1) WO2021178707A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022061002A1 (en) * 2020-09-18 2022-03-24 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency
CA3094859A1 (en) * 2020-10-01 2022-04-01 Entos Pharmaceuticals Inc. Proteolipid vesicles formulated with fusion associated small transmembrane proteins
WO2022187671A1 (en) * 2021-03-04 2022-09-09 Poseida Therapeutics, Inc. Compositions and methods for the treatment of hemophilia
GB202205606D0 (en) * 2022-04-14 2022-06-01 Genespire S R L Lentiviral vector
AU2024224841A1 (en) * 2023-02-21 2025-08-21 Poseida Therapeutics, Inc. Aav piggybac transposon polynucleotide compositions and methods of use therefor
KR20250173518A (ko) * 2023-04-05 2025-12-10 포세이다 테라퓨틱스, 인크. 트랜스포사제 폴리뉴클레오티드 및 그의 용도
IL325133A (en) * 2023-06-07 2026-02-01 Res Inst Nationwide Childrens Hospital Gene therapy for lysosomal acid lipase deficiency (LAL-D)
WO2025171163A1 (en) * 2024-02-06 2025-08-14 Duke University Compositions for and methods of treating lysosomal storage diseases

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011024480A1 (ja) * 2009-08-31 2011-03-03 Hukuda Keiichi 血管新生療法用医薬組成物
AU2015234242A1 (en) * 2014-03-21 2016-11-03 Children's Medical Research Institute Stable gene transfer to proliferating cells

Family Cites Families (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3773919A (en) 1969-10-23 1973-11-20 Du Pont Polylactide-drug mixtures
US4309989A (en) 1976-02-09 1982-01-12 The Curators Of The University Of Missouri Topical application of medication by ultrasound with coupling agent
FR2374910A1 (fr) 1976-10-23 1978-07-21 Choay Sa Preparation a base d'heparine, comprenant des liposomes, procede pour l'obtenir et medicaments contenant de telles preparations
US4554101A (en) 1981-01-09 1985-11-19 New York Blood Center, Inc. Identification and preparation of epitopes on antigens and allergens on the basis of hydrophilicity
US4656134A (en) 1982-01-11 1987-04-07 Board Of Trustees Of Leland Stanford Jr. University Gene amplification in eukaryotic cells
US5168062A (en) 1985-01-30 1992-12-01 University Of Iowa Research Foundation Transfer vectors and microorganisms containing human cytomegalovirus immediate-early promoter-regulatory DNA sequence
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4965188A (en) 1986-08-22 1990-10-23 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences using a thermostable enzyme
US4766067A (en) 1985-05-31 1988-08-23 President And Fellows Of Harvard College Gene amplification
GB8601597D0 (en) 1986-01-23 1986-02-26 Wilson R H Nucleotide sequences
US4800159A (en) 1986-02-07 1989-01-24 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences
US4767402A (en) 1986-07-08 1988-08-30 Massachusetts Institute Of Technology Ultrasound enhancement of transdermal drug delivery
EP0545913B1 (en) 1986-08-18 1999-02-24 Emisphere Technologies, Inc. Delivery systems for pharmacological agents
US4889818A (en) 1986-08-22 1989-12-26 Cetus Corporation Purified thermostable enzyme
US4921794A (en) 1987-01-14 1990-05-01 President And Fellows Of Harvard College T7 DNA polymerase
US4795699A (en) 1987-01-14 1989-01-03 President And Fellows Of Harvard College T7 DNA polymerase
US5130238A (en) 1988-06-24 1992-07-14 Cangene Corporation Enhanced nucleic acid amplification process
US5091310A (en) 1988-09-23 1992-02-25 Cetus Corporation Structure-independent dna amplification by the polymerase chain reaction
US5066584A (en) 1988-09-23 1991-11-19 Cetus Corporation Methods for generating single stranded dna by the polymerase chain reaction
US5142033A (en) 1988-09-23 1992-08-25 Hoffmann-La Roche Inc. Structure-independent DNA amplification by the polymerase chain reaction
US4994370A (en) 1989-01-03 1991-02-19 The United States Of America As Represented By The Department Of Health And Human Services DNA amplification technique
US5266491A (en) 1989-03-14 1993-11-30 Mochida Pharmaceutical Co., Ltd. DNA fragment and expression plasmid containing the DNA fragment
US5580575A (en) 1989-12-22 1996-12-03 Imarx Pharmaceutical Corp. Therapeutic drug delivery systems
US5580734A (en) 1990-07-13 1996-12-03 Transkaryotic Therapies, Inc. Method of producing a physical map contigous DNA sequences
US5733761A (en) 1991-11-05 1998-03-31 Transkaryotic Therapies, Inc. Protein production and protein delivery
US5641670A (en) 1991-11-05 1997-06-24 Transkaryotic Therapies, Inc. Protein production and protein delivery
US5643252A (en) 1992-10-28 1997-07-01 Venisect, Inc. Laser perforator
US5849695A (en) 1993-01-13 1998-12-15 The Regents Of The University Of California Parathyroid hormone analogues useful for treatment of osteoporosis and disorders of calcium meatabolism in mammals
CZ206195A3 (en) 1993-02-12 1996-04-17 Univ Leland Stanford Junior Controlled transcription of target genes and other biological materials
US5514670A (en) 1993-08-13 1996-05-07 Pharmos Corporation Submicron emulsions for delivery of peptides
US5814599A (en) 1995-08-04 1998-09-29 Massachusetts Insitiute Of Technology Transdermal delivery of encapsulated drugs
US5730723A (en) 1995-10-10 1998-03-24 Visionary Medical Products Corporation, Inc. Gas pressured needle-less injection device and method
US6218185B1 (en) 1996-04-19 2001-04-17 The United States Of America As Represented By The Secretary Of Agriculture Piggybac transposon-based genetic transformation system for insects
US5827729A (en) 1996-04-23 1998-10-27 Advanced Tissue Sciences Diffusion gradient bioreactor and extracorporeal liver device using a three-dimensional liver tissue
US5879681A (en) 1997-02-07 1999-03-09 Emisphere Technolgies Inc. Compounds and compositions for delivering active agents
IL120943A (en) 1997-05-29 2004-03-28 Univ Ben Gurion A system for administering drugs through the skin
US6309663B1 (en) 1999-08-17 2001-10-30 Lipocine Inc. Triglyceride-free compositions and methods for enhanced absorption of hydrophilic therapeutic agents
US6962810B2 (en) 2000-10-31 2005-11-08 University Of Notre Dame Du Lac Methods and compositions for transposition using minimal segments of the eukaryotic transformation vector piggyBac
EP2160461B1 (en) 2007-07-04 2012-08-08 Max-Delbrück-Centrum für Molekulare Medizin (MDC) Hyperactive variants of the transposase protein of the transposon system sleeping beauty
WO2010099296A1 (en) 2009-02-26 2010-09-02 Transposagen Biopharmaceuticals, Inc. Hyperactive piggybac transposases
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2012050919A2 (en) * 2010-09-28 2012-04-19 The University Of Notre Dame Chimeric spider silk and uses thereof
WO2012074758A1 (en) * 2010-11-16 2012-06-07 Transposagen Bioharmaceuticals, Inc. Hyperactive piggybac transposases
AU2013281328B2 (en) * 2012-06-27 2017-11-23 MeiraGTx Gene Regulation Limited Combination for treating an inflammatory disorder
WO2014143884A2 (en) 2013-03-15 2014-09-18 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Synthetic methylmalonyl-coa mutase transgene for the treatment of mut class methylmalonic acidemia (mma)
RS60902B1 (sr) 2014-03-09 2020-11-30 Univ Pennsylvania Kompozicije korisne u lečenju nedostatka ornitinske transkarbamilaze (otc)
US10927384B2 (en) 2014-04-09 2021-02-23 Dna Twopointo Inc. DNA vectors, transposons and transposases for eukaryotic genome modification
KR20180034467A (ko) 2015-08-03 2018-04-04 마이오도파 리미티드 L-dopa의 전신 합성 및 조절
US20170112108A1 (en) * 2015-10-22 2017-04-27 Hera Testing Laboratories, Inc. Genetically modified non-human mammals having modified liver cells and/or tissue and methods of making same
WO2017099838A1 (en) 2015-12-11 2017-06-15 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Gene therapy for combined methylmalonic acidemia/aciduria and hyperhomocysteinemia/homocystinuria, cobalamin c type, and deficiency of mmachc
EP3680280A4 (en) 2017-09-05 2021-06-02 Toray Industries, Inc. FIBER REINFORCED THERMOPLASTIC RESIN MOLDINGS
KR102849343B1 (ko) * 2018-03-07 2025-08-25 포세이다 테라퓨틱스, 인크. Cartyrin 조성물 및 사용 방법
EP3847197A1 (en) 2018-09-05 2021-07-14 Poseida Therapeutics, Inc. Allogeneic cell compositions and methods of use

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011024480A1 (ja) * 2009-08-31 2011-03-03 Hukuda Keiichi 血管新生療法用医薬組成物
AU2015234242A1 (en) * 2014-03-21 2016-11-03 Children's Medical Research Institute Stable gene transfer to proliferating cells

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
于海艳;徐俭;: "转基因方法治疗炎症性肠病", 国际消化病杂志, no. 02, 25 April 2006 (2006-04-25) *

Also Published As

Publication number Publication date
JP7817176B2 (ja) 2026-02-18
JP2023515692A (ja) 2023-04-13
WO2021178707A1 (en) 2021-09-10
AU2021230361A1 (en) 2022-09-08
CA3169529A1 (en) 2021-09-10
US12540335B2 (en) 2026-02-03
US20230104091A1 (en) 2023-04-06
EP4114469A1 (en) 2023-01-11
KR20220149588A (ko) 2022-11-08

Similar Documents

Publication Publication Date Title
CN115515646A (zh) 用于治疗代谢性肝病的组合物和方法
AU2019346655B2 (en) Frataxin expression constructs having engineered promoters and methods of use thereof
KR102907773B1 (ko) 인간 대상체에서의 비-노화-관련 청각 손상의 치료를 위한 조성물 및 방법
AU2024278250A1 (en) Compositions and methods of treating Huntington's Disease
KR102049161B1 (ko) 치료적 단백질을 조건부로 발현하는 벡터，상기 벡터를 포함하는 숙주 세포 및 이의 용도
KR20220022107A (ko) 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터
AU2016343979A1 (en) Delivery of central nervous system targeting polynucleotides
KR20210092755A (ko) 신경원성 세로이드 리포푸신증에 대한 유전자 요법
CN111733174B (zh) 一种分离的核酸分子及其用途
KR102628872B1 (ko) 세포의 증식을 제어하기 위해 세포 분열 좌위를 사용하기 위한 도구 및 방법
CN115516100A (zh) 靶向肌肉疾病中肌肉的优化基因疗法
KR20210151785A (ko) 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
TW202221125A (zh) 用於治療與葡萄糖神經醯胺酶β缺陷相關之神經病症的組合物及方法
CN119390787A (zh) 用于aav衣壳的使用转录依赖性定向进化的方法
KR20210049833A (ko) Mma의 치료를 위한 비-붕괴적 유전자 요법
AU2018352855B2 (en) Adeno-associated virus compositions for restoring HBB gene function and methods of use thereof
KR20230148824A (ko) 핵산을 전달하기 위한 조성물 및 방법
CN116157514A (zh) 新颖omni-59、61、67、76、79、80、81和82crispr核酸酶
CN113874512A (zh) 诱导毛细胞分化的组合物和方法
CN113260704B (zh) CYP4V2和RdCVF在制备药物中的用途
CN112639108A (zh) 治疗非综合征性感觉神经性听力损失的方法
CN114402075A (zh) 乌谢尔综合征(ush2a)的基因疗法
CN111902164A (zh) 用于恢复pah基因功能的腺相关病毒组合物及其使用方法
CN115768901A (zh) 腺病毒的大负载整合
CN111296364B (zh) 一种基因改造的小鼠动物模型基因改造方法及其应用

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