CN114340683A - 用于转基因表达的方法和组合物 - Google Patents
用于转基因表达的方法和组合物 Download PDFInfo
- Publication number
- CN114340683A CN114340683A CN202080043579.3A CN202080043579A CN114340683A CN 114340683 A CN114340683 A CN 114340683A CN 202080043579 A CN202080043579 A CN 202080043579A CN 114340683 A CN114340683 A CN 114340683A
- Authority
- CN
- China
- Prior art keywords
- cell
- raav
- transgene
- polynucleotide
- aav
- 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
Links
Images
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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/69—Boron compounds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
- A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
- A61K31/704—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin attached to a condensed carbocyclic ring system, e.g. sennosides, thiocolchicosides, escin, daunorubicin
-
- 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
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
- A61K38/05—Dipeptides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
- A61K38/06—Tripeptides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
- A61K38/07—Tetrapeptides
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
-
- 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)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Virology (AREA)
- Zoology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Physics & Mathematics (AREA)
- Mycology (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biochemistry (AREA)
- Marine Sciences & Fisheries (AREA)
- Pulmonology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Apparatus Associated With Microorganisms And Enzymes (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962833979P | 2019-04-15 | 2019-04-15 | |
| US62/833,979 | 2019-04-15 | ||
| US201962926317P | 2019-10-25 | 2019-10-25 | |
| US62/926,317 | 2019-10-25 | ||
| US202062967219P | 2020-01-29 | 2020-01-29 | |
| US62/967,219 | 2020-01-29 | ||
| PCT/US2020/028269 WO2020214672A1 (en) | 2019-04-15 | 2020-04-15 | Methods and compositions for transgene expression |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN114340683A true CN114340683A (zh) | 2022-04-12 |
Family
ID=70779846
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202080043579.3A Pending CN114340683A (zh) | 2019-04-15 | 2020-04-15 | 用于转基因表达的方法和组合物 |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20220195461A1 (https=) |
| EP (1) | EP3955970A1 (https=) |
| JP (2) | JP2022529470A (https=) |
| KR (1) | KR20220047538A (https=) |
| CN (1) | CN114340683A (https=) |
| AU (1) | AU2020257182A1 (https=) |
| BR (1) | BR112021020706A2 (https=) |
| CA (1) | CA3137078A1 (https=) |
| IL (1) | IL287262A (https=) |
| MX (1) | MX2021012682A (https=) |
| PH (1) | PH12021552609A1 (https=) |
| SG (1) | SG11202111353QA (https=) |
| WO (1) | WO2020214672A1 (https=) |
Cited By (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN114641318A (zh) * | 2019-04-15 | 2022-06-17 | 衣阿华大学研究基金会 | 用于治疗囊性纤维化的组合物和方法 |
| US11684679B2 (en) | 2016-03-07 | 2023-06-27 | University Of Iowa Research Foundation | AAV-mediated expression using a synthetic promoter and enhancer |
| US11702672B2 (en) | 2016-02-08 | 2023-07-18 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| US11999965B2 (en) | 2017-01-13 | 2024-06-04 | University Of Iowa Research Foundation | Bocaparvovirus small noncoding RNA and uses thereof |
| US12173305B2 (en) | 2016-05-26 | 2024-12-24 | University Of Iowa Research Foundation | cis and trans requirements for terminal resolution of human bocavirus 1 |
Citations (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2004090145A2 (en) * | 2003-03-31 | 2004-10-21 | University Of Iowa Research Foundation | Compounds and methods to enhance raav transduction |
| WO2011097456A2 (en) * | 2010-02-05 | 2011-08-11 | The University Of North Carolina At Chapel Hill | Compositions and methods for enhanced parvovirus transduction |
| US20150259395A1 (en) * | 2014-03-17 | 2015-09-17 | Avalanche Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| CN105142676A (zh) * | 2013-03-14 | 2015-12-09 | 夏尔人类遗传性治疗公司 | Cftr mrna组合物以及相关方法和用途 |
| CN105431170A (zh) * | 2013-04-08 | 2016-03-23 | 爱荷华大学研究基金会 | 嵌合腺相关病毒/博卡病毒细小病毒载体 |
| CN106928336A (zh) * | 2017-01-20 | 2017-07-07 | 首都医科大学附属北京儿童医院 | 囊性纤维化患者的cftr基因突变形式及其应用 |
| WO2017139381A1 (en) * | 2016-02-08 | 2017-08-17 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| WO2017155973A1 (en) * | 2016-03-07 | 2017-09-14 | University Of Iowa Research Foundation | Aav-mediated expression using a synthetic promoter and enhancer |
| US20180127471A1 (en) * | 2016-10-19 | 2018-05-10 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| WO2018170310A1 (en) * | 2017-03-15 | 2018-09-20 | The University Of North Carolina At Chapel Hill | Polyploid adeno-associated virus vectors and methods of making and using the same |
| CN110650733A (zh) * | 2017-02-28 | 2020-01-03 | 阿德夫拉姆生物技术股份有限公司 | 经过修饰的aav衣壳和其用途 |
| CN114641318A (zh) * | 2019-04-15 | 2022-06-17 | 衣阿华大学研究基金会 | 用于治疗囊性纤维化的组合物和方法 |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| AU707866B2 (en) | 1994-12-06 | 1999-07-22 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant AAV vectors |
| US5656785A (en) | 1995-08-07 | 1997-08-12 | The Charles Stark Draper Laboratory, Inc. | Micromechanical contact load force sensor for sensing magnitude and distribution of loads and tool employing micromechanical contact load force sensor |
| AU5445998A (en) | 1996-11-19 | 1998-06-10 | Surgx Corporation | A transient voltage protection device and method of making same |
| AU741605B2 (en) | 1996-12-18 | 2001-12-06 | Targeted Genetics Corporation | AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
-
2020
- 2020-04-15 EP EP20727413.5A patent/EP3955970A1/en not_active Withdrawn
- 2020-04-15 PH PH1/2021/552609A patent/PH12021552609A1/en unknown
- 2020-04-15 AU AU2020257182A patent/AU2020257182A1/en not_active Abandoned
- 2020-04-15 CA CA3137078A patent/CA3137078A1/en active Pending
- 2020-04-15 KR KR1020217037211A patent/KR20220047538A/ko not_active Ceased
- 2020-04-15 MX MX2021012682A patent/MX2021012682A/es unknown
- 2020-04-15 CN CN202080043579.3A patent/CN114340683A/zh active Pending
- 2020-04-15 BR BR112021020706A patent/BR112021020706A2/pt unknown
- 2020-04-15 SG SG11202111353QA patent/SG11202111353QA/en unknown
- 2020-04-15 US US17/603,840 patent/US20220195461A1/en not_active Abandoned
- 2020-04-15 JP JP2021561893A patent/JP2022529470A/ja active Pending
- 2020-04-15 WO PCT/US2020/028269 patent/WO2020214672A1/en not_active Ceased
-
2021
- 2021-10-14 IL IL287262A patent/IL287262A/en unknown
-
2023
- 2023-07-21 JP JP2023119196A patent/JP2023126658A/ja active Pending
Patent Citations (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2004090145A2 (en) * | 2003-03-31 | 2004-10-21 | University Of Iowa Research Foundation | Compounds and methods to enhance raav transduction |
| WO2011097456A2 (en) * | 2010-02-05 | 2011-08-11 | The University Of North Carolina At Chapel Hill | Compositions and methods for enhanced parvovirus transduction |
| CN105142676A (zh) * | 2013-03-14 | 2015-12-09 | 夏尔人类遗传性治疗公司 | Cftr mrna组合物以及相关方法和用途 |
| CN105431170A (zh) * | 2013-04-08 | 2016-03-23 | 爱荷华大学研究基金会 | 嵌合腺相关病毒/博卡病毒细小病毒载体 |
| US20150259395A1 (en) * | 2014-03-17 | 2015-09-17 | Avalanche Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| CN106414474A (zh) * | 2014-03-17 | 2017-02-15 | 阿德夫拉姆生物技术股份有限公司 | 用于视锥细胞中增强的基因表达的组合物和方法 |
| WO2017139381A1 (en) * | 2016-02-08 | 2017-08-17 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| WO2017155973A1 (en) * | 2016-03-07 | 2017-09-14 | University Of Iowa Research Foundation | Aav-mediated expression using a synthetic promoter and enhancer |
| US20180127471A1 (en) * | 2016-10-19 | 2018-05-10 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| CN106928336A (zh) * | 2017-01-20 | 2017-07-07 | 首都医科大学附属北京儿童医院 | 囊性纤维化患者的cftr基因突变形式及其应用 |
| CN110650733A (zh) * | 2017-02-28 | 2020-01-03 | 阿德夫拉姆生物技术股份有限公司 | 经过修饰的aav衣壳和其用途 |
| US20210130413A1 (en) * | 2017-02-28 | 2021-05-06 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| WO2018170310A1 (en) * | 2017-03-15 | 2018-09-20 | The University Of North Carolina At Chapel Hill | Polyploid adeno-associated virus vectors and methods of making and using the same |
| CN114641318A (zh) * | 2019-04-15 | 2022-06-17 | 衣阿华大学研究基金会 | 用于治疗囊性纤维化的组合物和方法 |
Non-Patent Citations (9)
| Title |
|---|
| ASHLEY L. COONEY等: "Cystic Fibrosis Gene Therapy: Looking Back, Looking Forward", GENES, vol. 9, no. 11, 7 November 2018 (2018-11-07), pages 8 - 23 * |
| BRADLEY A. HAMILT ON等: "Polarized AAVR expression determines infectivity by AAV gene therapy vectors", GENE THERAPY, vol. 26, 8 April 2019 (2019-04-08), pages 240 - 249, XP036815850, DOI: 10.1038/s41434-019-0078-3 * |
| KATHERINE J. D. A. EXCOFFON等: "Directed evolution of adeno-associated virus to an infectious respiratory virus", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. 106, no. 10, 10 March 2009 (2009-03-10), pages 3865 - 3870, XP093064067, DOI: 10.1073/pnas.0813365106 * |
| LINS等: "Delivery of a novel AAV, AV.Tl65-CFTR, to human bronchial epithelial cells from patients with cystic fibrosis augments functional recovery of chloride conductance", PEDIATRIC PULMONOLOGY, vol. 54, no. 2, 1 October 2019 (2019-10-01), pages 218, XP009521879, DOI: 10.1002/ppul.22495 * |
| 卢超;王启钊;许瑞安;: "重组腺相关病毒载体的整合性研究进展", 生命科学, no. 05, 15 May 2010 (2010-05-15), pages 459 - 465 * |
| 宫晓丽;王乐;王玮;付夏;张婷;王晓民;: "阿霉素在小鼠离体培养脑片中增强Ⅱ型腺相关病毒的转导", 首都医科大学学报, no. 06, 14 December 2015 (2015-12-14), pages 902 - 907 * |
| 张凤兰;文朝阳;丁卫;: "腺相关病毒基因治疗载体的改良与应用", 首都医科大学学报, no. 04, 21 August 2009 (2009-08-21), pages 565 - 573 * |
| 朱甫祥;刘泽隆;缪静;屈慧鸽;迟晓艳;: "基于蛋白质剪接的BHK细胞Ser~(660)前断裂的CFTR基因转移", 中国生物化学与分子生物学报, no. 05, 20 May 2010 (2010-05-20), pages 429 - 435 * |
| 殷子斐;王丽娜;王园;凌晨;: "提高重组型腺相关病毒转导效率的研究现状", 生物技术通报, no. 09, 15 September 2015 (2015-09-15), pages 49 - 59 * |
Cited By (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US11702672B2 (en) | 2016-02-08 | 2023-07-18 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| US11684679B2 (en) | 2016-03-07 | 2023-06-27 | University Of Iowa Research Foundation | AAV-mediated expression using a synthetic promoter and enhancer |
| US12173305B2 (en) | 2016-05-26 | 2024-12-24 | University Of Iowa Research Foundation | cis and trans requirements for terminal resolution of human bocavirus 1 |
| US11999965B2 (en) | 2017-01-13 | 2024-06-04 | University Of Iowa Research Foundation | Bocaparvovirus small noncoding RNA and uses thereof |
| CN114641318A (zh) * | 2019-04-15 | 2022-06-17 | 衣阿华大学研究基金会 | 用于治疗囊性纤维化的组合物和方法 |
Also Published As
| Publication number | Publication date |
|---|---|
| MX2021012682A (es) | 2022-03-25 |
| KR20220047538A (ko) | 2022-04-18 |
| IL287262A (en) | 2021-12-01 |
| AU2020257182A1 (en) | 2021-12-09 |
| PH12021552609A1 (en) | 2022-06-27 |
| BR112021020706A2 (pt) | 2022-03-15 |
| JP2023126658A (ja) | 2023-09-07 |
| JP2022529470A (ja) | 2022-06-22 |
| SG11202111353QA (en) | 2021-11-29 |
| WO2020214672A1 (en) | 2020-10-22 |
| CA3137078A1 (en) | 2020-10-22 |
| US20220195461A1 (en) | 2022-06-23 |
| EP3955970A1 (en) | 2022-02-23 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7731801B2 (ja) | 嚢胞性線維症の治療のための組換えアデノ随伴ウイルス | |
| CN114340683A (zh) | 用于转基因表达的方法和组合物 | |
| CN105431170B (zh) | 嵌合腺相关病毒/博卡病毒细小病毒载体 | |
| US8137962B2 (en) | Compositions for treating cystic fibrosis | |
| CN116096431A (zh) | 肽修饰的aav衣壳 | |
| EP2660325A2 (en) | AAV vectors and corresponding nucleotide sequences and methods | |
| JP2016525356A (ja) | 変異aav、及び、細胞、臓器並びに組織への遺伝子導入のための組成物、方法並びに使用法 | |
| US20230242941A1 (en) | Methods and compositions for administering recombinant viral vectors | |
| CN115960921B (zh) | 一组肝靶向新型腺相关病毒的获得及其应用 | |
| Kochergin-Nikitsky et al. | Tissue and cell-type-specific transduction using rAAV vectors in lung diseases | |
| JP2025527658A (ja) | 組換えアデノ随伴ウイルス及びその使用 | |
| US20240115738A1 (en) | Methods and compositions for treatment of cystic fibrosis | |
| EA050636B1 (ru) | Композиции и способы лечения муковисцидоза | |
| US11345927B2 (en) | Compositions for reducing sarcolipin expression and preventing and treating muscular dystrophy and cardiomyopathy and methods of use | |
| WO2025259575A9 (en) | Methods and compositions for treatment of duchenne muscular dystrophy | |
| WO2025039622A1 (zh) | 经修饰的腺相关病毒载体及其在治疗中枢神经系统疾病中的用途 | |
| AU2024305531A1 (en) | Materials and methods for the treatment of neurofibromin 1 mutations and diseases resulting therefrom | |
| HK1217916B (zh) | 嵌合腺相關病毒/博卡病毒細小病毒載體 |
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 | ||
| WD01 | Invention patent application deemed withdrawn after publication | ||
| WD01 | Invention patent application deemed withdrawn after publication |
Application publication date: 20220412 |