CN114761569A - 阿尔茨海默氏病的基因疗法 - Google Patents
阿尔茨海默氏病的基因疗法 Download PDFInfo
- Publication number
- CN114761569A CN114761569A CN202080073402.8A CN202080073402A CN114761569A CN 114761569 A CN114761569 A CN 114761569A CN 202080073402 A CN202080073402 A CN 202080073402A CN 114761569 A CN114761569 A CN 114761569A
- Authority
- CN
- China
- Prior art keywords
- vector
- composition
- sequence
- sequences
- nucleic acid
- 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
- 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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- 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
- C07K14/775—Apolipopeptides
-
- 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
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- 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
-
- 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
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
- A01K2267/0312—Animal model for Alzheimer's disease
-
- 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
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
- C12N2310/141—MicroRNAs, miRNAs
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/31—Combination 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)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Toxicology (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Virology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962915988P | 2019-10-16 | 2019-10-16 | |
| US62/915,988 | 2019-10-16 | ||
| PCT/US2020/056051 WO2021076941A1 (en) | 2019-10-16 | 2020-10-16 | Gene therapy for alzheimer's disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN114761569A true CN114761569A (zh) | 2022-07-15 |
Family
ID=73288693
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202080073402.8A Pending CN114761569A (zh) | 2019-10-16 | 2020-10-16 | 阿尔茨海默氏病的基因疗法 |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20230405148A1 (https=) |
| EP (1) | EP4045653A1 (https=) |
| JP (2) | JP2023500793A (https=) |
| KR (1) | KR20220082050A (https=) |
| CN (1) | CN114761569A (https=) |
| AU (2) | AU2020367437B2 (https=) |
| BR (1) | BR112022007010A2 (https=) |
| CA (1) | CA3157864A1 (https=) |
| CO (1) | CO2022006367A2 (https=) |
| IL (1) | IL292148A (https=) |
| MX (1) | MX2022004524A (https=) |
| WO (1) | WO2021076941A1 (https=) |
| ZA (1) | ZA202205344B (https=) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12611467B2 (en) | 2020-11-25 | 2026-04-28 | Cornell University | APOE gene therapy |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP7616995B2 (ja) * | 2018-11-28 | 2025-01-17 | プリベイル セラピューティクス,インコーポレーテッド | 神経変性疾患のための遺伝子治療 |
| EP4508213A1 (en) * | 2022-04-12 | 2025-02-19 | uniQure biopharma B.V. | Nucleic acid regulation of apoe |
| EP4551711A1 (en) * | 2022-07-08 | 2025-05-14 | Cornell University | Methods and pharmaceutical compositions for the treatment and the prevention of alzheimers disease |
| WO2025213111A2 (en) * | 2024-04-05 | 2025-10-09 | Cornell University | Aav expression of mirna to suppress human apoe mrna |
Citations (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2005116204A1 (ja) * | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Rna干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法 |
| CN101652063A (zh) * | 2007-02-06 | 2010-02-17 | T·J·杨 | 使用基因疗法治疗和预防神经变性疾病 |
| WO2013181618A2 (en) * | 2012-05-31 | 2013-12-05 | The Trustees Of Columbia University In The City Of New York | Methods to treat alzheimer's disease using apoe inhibitors |
| WO2015077473A1 (en) * | 2013-11-20 | 2015-05-28 | University Of Iowa Research Foundation | Methods and compositions for treating amyloid deposits |
| WO2018045347A1 (en) * | 2016-09-02 | 2018-03-08 | Spark Therapeutics, Inc. | Methods and vectors for treating cns disorders |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US4863457A (en) | 1986-11-24 | 1989-09-05 | Lee David A | Drug delivery device |
| US5378475A (en) | 1991-02-21 | 1995-01-03 | University Of Kentucky Research Foundation | Sustained release drug delivery devices |
| AU4198793A (en) | 1992-07-24 | 1994-01-27 | Takeda Chemical Industries Ltd. | Microparticle preparation and production thereof |
| FR2695563B1 (fr) | 1992-09-11 | 1994-12-02 | Pasteur Institut | Microparticules portant des antigènes et leur utilisation pour l'induction de réponses humorales ou cellulaires. |
| ATE163230T1 (de) | 1993-03-09 | 1998-02-15 | Epic Therapeutics Inc | Makromolekulare mikropartikel und verfahren zu ihrer herstellung |
| US6090925A (en) | 1993-03-09 | 2000-07-18 | Epic Therapeutics, Inc. | Macromolecular microparticles and methods of production and use |
| US5981719A (en) | 1993-03-09 | 1999-11-09 | Epic Therapeutics, Inc. | Macromolecular microparticles and methods of production and use |
| US5603960A (en) | 1993-05-25 | 1997-02-18 | O'hagan; Derek T. | Preparation of microparticles and method of immunization |
| US5464758A (en) | 1993-06-14 | 1995-11-07 | Gossen; Manfred | Tight control of gene expression in eucaryotic cells by tetracycline-responsive promoters |
| US5814618A (en) | 1993-06-14 | 1998-09-29 | Basf Aktiengesellschaft | Methods for regulating gene expression |
| US6913767B1 (en) | 1993-10-25 | 2005-07-05 | Genentech, Inc. | Compositions for microencapsulation of antigens for use as vaccines |
| US5443505A (en) | 1993-11-15 | 1995-08-22 | Oculex Pharmaceuticals, Inc. | Biocompatible ocular implants |
| US6342390B1 (en) | 1994-11-23 | 2002-01-29 | The United States Of America As Represented By The Secretary Of Health And Human Services | Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy |
| CA2267930A1 (en) | 1996-10-09 | 1998-04-16 | Nobuyuki Takechi | A method for producing a microparticle |
| US6210707B1 (en) | 1996-11-12 | 2001-04-03 | The Regents Of The University Of California | Methods of forming protein-linked lipidic microparticles, and compositions thereof |
| US6884435B1 (en) | 1997-01-30 | 2005-04-26 | Chiron Corporation | Microparticles with adsorbent surfaces, methods of making same, and uses thereof |
| GB9810236D0 (en) | 1998-05-13 | 1998-07-08 | Microbiological Res Authority | Improvements relating to encapsulation of bioactive agents |
| US6406719B1 (en) | 1998-05-13 | 2002-06-18 | Microbiological Research Authority | Encapsulation of bioactive agents |
| US6264987B1 (en) | 2000-05-19 | 2001-07-24 | Alkermes Controlled Therapeutics Inc. Ii | Method for preparing microparticles having a selected polymer molecular weight |
| FR2814642B1 (fr) | 2000-10-03 | 2005-07-01 | Ass Pour Le Dev De La Rech En | Souris transgenique pour la recombinaison ciblee mediee par la cre-er modifiee |
| US6723551B2 (en) | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| WO2007041190A2 (en) | 2005-09-30 | 2007-04-12 | The University Of Iowa Research Foundation | Polymer-based delivery system for immunotherapy of cancer |
| CA2643048A1 (en) * | 2006-02-21 | 2007-08-30 | Oklahoma Medical Research Foundation | Treatment of alzheimer's disease with inhibitors of apoe binding to apoe receptor |
| US9572894B2 (en) | 2010-10-18 | 2017-02-21 | The University Of Iowa Research Foundation | Biodegradable particulate formulations |
| WO2012115806A1 (en) | 2011-02-24 | 2012-08-30 | University Of Iowa Research Foundation | New biodegradable polymers with sulfenamide bonds for drug delivery applications |
| JP6469000B2 (ja) * | 2012-05-18 | 2019-02-13 | ユニバーシティー オブ アイオワ リサーチ ファウンデーション | アミロイドの沈着を処置するための方法および組成物 |
| US11071791B2 (en) * | 2018-01-26 | 2021-07-27 | Wisconsin Alumni Research Foundation | Vector for gene silencing and replacement and methods of use thereof |
| JP7616995B2 (ja) * | 2018-11-28 | 2025-01-17 | プリベイル セラピューティクス,インコーポレーテッド | 神経変性疾患のための遺伝子治療 |
-
2020
- 2020-10-16 EP EP20804387.7A patent/EP4045653A1/en active Pending
- 2020-10-16 BR BR112022007010A patent/BR112022007010A2/pt unknown
- 2020-10-16 KR KR1020227016247A patent/KR20220082050A/ko active Pending
- 2020-10-16 US US17/769,255 patent/US20230405148A1/en active Pending
- 2020-10-16 WO PCT/US2020/056051 patent/WO2021076941A1/en not_active Ceased
- 2020-10-16 JP JP2022522814A patent/JP2023500793A/ja active Pending
- 2020-10-16 CN CN202080073402.8A patent/CN114761569A/zh active Pending
- 2020-10-16 AU AU2020367437A patent/AU2020367437B2/en active Active
- 2020-10-16 CA CA3157864A patent/CA3157864A1/en active Pending
- 2020-10-16 MX MX2022004524A patent/MX2022004524A/es unknown
-
2022
- 2022-04-11 IL IL292148A patent/IL292148A/en unknown
- 2022-05-13 ZA ZA2022/05344A patent/ZA202205344B/en unknown
- 2022-05-16 CO CONC2022/0006367A patent/CO2022006367A2/es unknown
-
2025
- 2025-02-06 AU AU2025200812A patent/AU2025200812A1/en active Pending
- 2025-12-05 JP JP2025233417A patent/JP2026041903A/ja active Pending
Patent Citations (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2005116204A1 (ja) * | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Rna干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法 |
| CN101652063A (zh) * | 2007-02-06 | 2010-02-17 | T·J·杨 | 使用基因疗法治疗和预防神经变性疾病 |
| WO2013181618A2 (en) * | 2012-05-31 | 2013-12-05 | The Trustees Of Columbia University In The City Of New York | Methods to treat alzheimer's disease using apoe inhibitors |
| WO2015077473A1 (en) * | 2013-11-20 | 2015-05-28 | University Of Iowa Research Foundation | Methods and compositions for treating amyloid deposits |
| WO2018045347A1 (en) * | 2016-09-02 | 2018-03-08 | Spark Therapeutics, Inc. | Methods and vectors for treating cns disorders |
| CN110121356A (zh) * | 2016-09-02 | 2019-08-13 | 星火治疗股份有限公司 | 治疗cns疾病的方法和载体 |
Non-Patent Citations (3)
| Title |
|---|
| DANIEL K FOWLER 等: "Improved knockdown from artificial microRNAs in an enhanced miR-155 backbone: a designer\'s guide to potent multi-target RNAi", NUCLEIC ACIDS RES, vol. 44, no. 5, 17 November 2015 (2015-11-17), pages 1 * |
| DANIEL K FOWLER 等: "Improved knockdown from artificial microRNAs in an enhanced miR-155 backbone: a designer\'s guide to potent multi-target RNAi", NUCLEIC ACIDS RESEARCH, vol. 44, no. 5, 17 November 2015 (2015-11-17), pages 1 - 16 * |
| YU-WEN ALVIN HUANG 等: "ApoE2, ApoE3, and ApoE4 Differentially Stimulate APP Transcription and Ab Secretion", CELL PRESS, vol. 168, 26 January 2017 (2017-01-26), pages 427 - 441 * |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12611467B2 (en) | 2020-11-25 | 2026-04-28 | Cornell University | APOE gene therapy |
Also Published As
| Publication number | Publication date |
|---|---|
| US20230405148A1 (en) | 2023-12-21 |
| CA3157864A1 (en) | 2021-04-22 |
| JP2026041903A (ja) | 2026-03-10 |
| JP2023500793A (ja) | 2023-01-11 |
| WO2021076941A1 (en) | 2021-04-22 |
| ZA202205344B (en) | 2023-03-29 |
| AU2020367437B2 (en) | 2024-11-07 |
| BR112022007010A2 (pt) | 2022-07-12 |
| AU2025200812A1 (en) | 2025-02-27 |
| MX2022004524A (es) | 2022-07-21 |
| EP4045653A1 (en) | 2022-08-24 |
| WO2021076941A9 (en) | 2021-10-07 |
| KR20220082050A (ko) | 2022-06-16 |
| IL292148A (en) | 2022-06-01 |
| AU2020367437A1 (en) | 2022-06-02 |
| CO2022006367A2 (es) | 2022-08-09 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7504967B2 (ja) | MeCP2発現カセット | |
| AU2020367437B2 (en) | Gene therapy for Alzheimer's disease | |
| JP2019519221A (ja) | 加齢関連疾患及び症状の遺伝子治療法 | |
| US20210324387A1 (en) | Aav vectors for treatment of dominant retinitis pigmentosa | |
| KR20220107243A (ko) | Apoe 유전자 요법 | |
| US20230089490A1 (en) | Raav-mediated in vivo delivery of suppressor trnas | |
| JP2025170347A (ja) | 遺伝子治療発現カセットからの発現レベルを調節するための方法 | |
| CN115960921A (zh) | 一组肝靶向新型腺相关病毒的获得及其应用 | |
| AU2020212026A1 (en) | Highly efficient transduction and lateral spread in the retina by a novel AAV virus enhanced by rational design | |
| CN121013909A (zh) | 视网膜细胞中的基因表达的启动子及包含其的载体系统 | |
| HK40073494A (en) | Gene therapy for alzheimer's disease | |
| US20230414787A1 (en) | Gene knock-out for treatment of glaucoma | |
| WO2025213111A2 (en) | Aav expression of mirna to suppress human apoe mrna | |
| HK40088240A (en) | Aav vectors for treatment of dominant retinitis pigmentosa |
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 | ||
| REG | Reference to a national code |
Ref country code: HK Ref legal event code: DE Ref document number: 40073494 Country of ref document: HK |