MX2023000994A - Genoterapia con csrp3 (proteina 3 rica en cisteina y glicina). - Google Patents
Genoterapia con csrp3 (proteina 3 rica en cisteina y glicina).Info
- Publication number
- MX2023000994A MX2023000994A MX2023000994A MX2023000994A MX2023000994A MX 2023000994 A MX2023000994 A MX 2023000994A MX 2023000994 A MX2023000994 A MX 2023000994A MX 2023000994 A MX2023000994 A MX 2023000994A MX 2023000994 A MX2023000994 A MX 2023000994A
- Authority
- MX
- Mexico
- Prior art keywords
- csrp3
- cysteine
- gene therapy
- rich protein
- glycine rich
- Prior art date
Links
- 102100031620 Cysteine and glycine-rich protein 3 Human genes 0.000 title abstract 3
- 108010023942 cysteine and glycine-rich protein 3 Proteins 0.000 title abstract 3
- 238000001415 gene therapy Methods 0.000 title abstract 2
- 101150062275 CSRP3 gene Proteins 0.000 title 1
- 210000000234 capsid Anatomy 0.000 abstract 3
- 238000000034 method Methods 0.000 abstract 2
- 239000013598 vector Substances 0.000 abstract 2
- 208000031229 Cardiomyopathies Diseases 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 102000004987 Troponin T Human genes 0.000 abstract 1
- 108090001108 Troponin T Proteins 0.000 abstract 1
- 230000000747 cardiac effect Effects 0.000 abstract 1
- 230000006735 deficit Effects 0.000 abstract 1
- 238000001990 intravenous administration Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 239000013608 rAAV vector Substances 0.000 abstract 1
Classifications
-
- 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
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
- C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
-
- 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
-
- 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
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4716—Muscle proteins, e.g. myosin, actin
-
- 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
- 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
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
-
- 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
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- 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
-
- 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)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Cardiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Heart & Thoracic Surgery (AREA)
- Hospice & Palliative Care (AREA)
- Epidemiology (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)
- Immunology (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063061727P | 2020-08-05 | 2020-08-05 | |
| PCT/US2021/044412 WO2022031756A1 (en) | 2020-08-05 | 2021-08-03 | Csrp3 (cysteine and glycine rich protein 3) gene therapy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2023000994A true MX2023000994A (es) | 2023-03-01 |
Family
ID=80118758
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2023000994A MX2023000994A (es) | 2020-08-05 | 2021-08-03 | Genoterapia con csrp3 (proteina 3 rica en cisteina y glicina). |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230257431A1 (https=) |
| EP (1) | EP4192962A4 (https=) |
| JP (1) | JP2023536618A (https=) |
| KR (1) | KR20230042468A (https=) |
| CN (1) | CN116234916A (https=) |
| AU (1) | AU2021321410A1 (https=) |
| BR (1) | BR112023001336A2 (https=) |
| CA (1) | CA3184983A1 (https=) |
| IL (1) | IL300187A (https=) |
| MX (1) | MX2023000994A (https=) |
| WO (1) | WO2022031756A1 (https=) |
Families Citing this family (8)
| 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. |
| EP4103214B1 (en) | 2020-02-13 | 2026-04-29 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| KR20230043869A (ko) | 2020-08-07 | 2023-03-31 | 스페이스크래프트 세븐, 엘엘씨 | Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법 |
| JP2025508743A (ja) * | 2022-02-18 | 2025-04-10 | ギンコ バイオワークス インコーポレイテッド | 不整脈原性心筋症のための遺伝子治療 |
| US20250205365A1 (en) * | 2022-03-18 | 2025-06-26 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treating rbm20 related cardiomyopathy with a viral vector |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE10004857A1 (de) * | 2000-02-03 | 2001-08-16 | Schering Ag | MLP-Gen, Nukleinsäuren, Polypeptide und deren Verwendung |
| EP2176283B1 (en) | 2007-07-14 | 2016-11-02 | University of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| EP2792742A1 (en) * | 2013-04-17 | 2014-10-22 | Universitätsklinikum Hamburg-Eppendorf (UKE) | Gene-therapy vectors for treating cardiomyopathy |
| ES2739288T3 (es) | 2013-09-13 | 2020-01-30 | California Inst Of Techn | Recuperación selectiva |
| LT3137497T (lt) | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| KR102423442B1 (ko) | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| JP7676111B2 (ja) * | 2017-03-17 | 2025-05-14 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 |
| RU2770922C2 (ru) * | 2017-09-20 | 2022-04-25 | 4Д Молекьюлар Терапьютикс Инк. | Капсиды вариантов аденоассоциированных вирусов и методы их применения |
| US20210363193A1 (en) * | 2018-04-27 | 2021-11-25 | Universität Heidelberg | Modified aav capsid polypeptides for treatment of muscular diseases |
| CA3100280A1 (en) * | 2018-06-08 | 2019-12-12 | University Of Florida Research Foundation, Incorporated | Aav cardiac gene therapy for cardiomyopathy |
| EP3947700A4 (en) * | 2019-04-01 | 2023-01-04 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
-
2021
- 2021-08-03 KR KR1020237003436A patent/KR20230042468A/ko active Pending
- 2021-08-03 MX MX2023000994A patent/MX2023000994A/es unknown
- 2021-08-03 CA CA3184983A patent/CA3184983A1/en active Pending
- 2021-08-03 BR BR112023001336A patent/BR112023001336A2/pt unknown
- 2021-08-03 WO PCT/US2021/044412 patent/WO2022031756A1/en not_active Ceased
- 2021-08-03 JP JP2023507388A patent/JP2023536618A/ja active Pending
- 2021-08-03 US US18/019,396 patent/US20230257431A1/en active Pending
- 2021-08-03 IL IL300187A patent/IL300187A/en unknown
- 2021-08-03 AU AU2021321410A patent/AU2021321410A1/en active Pending
- 2021-08-03 EP EP21852527.7A patent/EP4192962A4/en active Pending
- 2021-08-03 CN CN202180057650.8A patent/CN116234916A/zh active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CA3184983A1 (en) | 2022-02-10 |
| JP2023536618A (ja) | 2023-08-28 |
| EP4192962A1 (en) | 2023-06-14 |
| WO2022031756A1 (en) | 2022-02-10 |
| AU2021321410A1 (en) | 2023-04-06 |
| KR20230042468A (ko) | 2023-03-28 |
| CN116234916A (zh) | 2023-06-06 |
| EP4192962A4 (en) | 2025-04-23 |
| BR112023001336A2 (pt) | 2023-02-14 |
| US20230257431A1 (en) | 2023-08-17 |
| IL300187A (en) | 2023-03-01 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| MX2023000994A (es) | Genoterapia con csrp3 (proteina 3 rica en cisteina y glicina). | |
| BR112023001852A2 (pt) | Terapia gênica com placofilina-2 (pkp2) usando vetor de aav | |
| CL2024003182A1 (es) | Terapia génica con el atanógeno 3 asociado a linfoma de células b 2 (bag3) utilizando | |
| WO2023108029A3 (en) | Junctophilin-2 (jph2) gene therapy using aav vector | |
| CL2021002236A1 (es) | Inserción de vectores de virus adeno-asociados de b-sarcoglicano y el tratamiento de distrofia muscular | |
| BR112022021134A2 (pt) | Vírus adeno-associado com capsídeo projetado | |
| BR112021020054A2 (pt) | Capsídeos de vírus adeno-associado (aav) e composições contendo os mesmos | |
| CL2020001360A1 (es) | Cápsides variantes de virus adenoasociados y su uso para inhibir la angiogénesis. | |
| PE20201264A1 (es) | Variantes de capsides de virus adenoasociados y metodos de uso de estas | |
| CL2018003196A1 (es) | Cápsides variantes de virus adenoasociados y métodos de uso de estas. | |
| BR112021019821A2 (pt) | Sorotipo de vírus adenoassociado híbrido recombinante com modificação peptídica entre aav9 e aavrh74 com tropismo hepático reduzido e transdução muscular aumentada | |
| MX2023000995A (es) | Vector de genoterapia para eef1a2 y usos del mismo. | |
| BR112023001418A2 (pt) | Vetor viral adeno-associado para expressão de glut1 e seus usos | |
| EA202290001A1 (ru) | Векторы aav с промотором миелинового белка ноль и их применение для лечения заболеваний, связанных со шванновскими клетками, таких как болезнь шарко-мари-тута | |
| AR122289A1 (es) | Terapia genética cardíaca con aav para la cardiomiopatía en humanos | |
| AR123358A1 (es) | Virus adenoasociado recombinante para el tratamiento de la neurodegeneración de inicio adulto asociada a la grn | |
| CL2023001876A1 (es) | Proteínas de la cápside viral con especificidad para las células del tejido cardiaco | |
| CO2025001825A2 (es) | Composición para su uso en el tratamiento de la enfermedad de fabry | |
| EA202091194A1 (ru) | Вирусные векторы, содержащие области, кодирующие rdh12, и способы лечения дистрофий сетчатки | |
| CL2025002970A1 (es) | Composiciones y métodos para el tratamiento de trastornos relacionados con la deficiencia de cdkl5 | |
| AR129033A1 (es) | Cápsides para terapia con el gen de placofilina-2 | |
| MX2020013313A (es) | Terapia genica cardiaca con aav para cardiomiopatia. | |
| CO2022009604A2 (es) | Vectores de virus adenoasociados para el tratamiento del síndrome de hunter | |
| CO2023004031A2 (es) | Administración sistémica del vector de virus adenoasociado que expresa el sarcoglucano g y el tratamiento de la distrofia muscular | |
| CL2025001989A1 (es) | Terapia génica con plakofilina-2 (pkp2) utilizando vectores aav |