CL2022003099A1 - Terapia génica con vectores duales de disferlina - Google Patents
Terapia génica con vectores duales de disferlinaInfo
- Publication number
- CL2022003099A1 CL2022003099A1 CL2022003099A CL2022003099A CL2022003099A1 CL 2022003099 A1 CL2022003099 A1 CL 2022003099A1 CL 2022003099 A CL2022003099 A CL 2022003099A CL 2022003099 A CL2022003099 A CL 2022003099A CL 2022003099 A1 CL2022003099 A1 CL 2022003099A1
- Authority
- CL
- Chile
- Prior art keywords
- dual
- vectors
- dysferlin
- gene therapy
- recombinant polynucleotides
- Prior art date
Links
- 230000009977 dual effect Effects 0.000 title abstract 3
- 239000013598 vector Substances 0.000 title abstract 3
- 102000004168 Dysferlin Human genes 0.000 title 1
- 108090000620 Dysferlin Proteins 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 102000040430 polynucleotide Human genes 0.000 abstract 3
- 108091033319 polynucleotide Proteins 0.000 abstract 3
- 239000002157 polynucleotide Substances 0.000 abstract 3
- 239000000203 mixture Substances 0.000 abstract 2
- 239000013612 plasmid Substances 0.000 abstract 2
- 239000013603 viral vector Substances 0.000 abstract 2
- 101001016184 Homo sapiens Dysferlin Proteins 0.000 abstract 1
- 208000022583 Qualitative or quantitative defects of dysferlin Diseases 0.000 abstract 1
- 239000012634 fragment Substances 0.000 abstract 1
- 102000056610 human DYSF Human genes 0.000 abstract 1
Classifications
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- 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
- C07K14/4707—Muscular dystrophy
-
- 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
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- A01K2217/075—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
-
- 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
-
- 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
- C12N2800/40—Systems of functionally co-operating vectors
-
- 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
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Dermatology (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)
- Medicinal Preparation (AREA)
Abstract
En el presente documento se describen polinucleótidos recombinantes que codifican fragmentos de una proteína disferlina humana. Además, se describen adicionalmente plásmidos, vectores virales, sistemas de vectores duales, células y composiciones que comprenden dichos polinucleótidos recombinantes. Dichos polinucleótidos recombinantes, plásmidos, vectores virales, sistemas de vectores duales, células y composiciones pueden usarse para tratar disferlinopatías.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063024338P | 2020-05-13 | 2020-05-13 |
Publications (1)
Publication Number | Publication Date |
---|---|
CL2022003099A1 true CL2022003099A1 (es) | 2023-10-13 |
Family
ID=78524926
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CL2022003099A CL2022003099A1 (es) | 2020-05-13 | 2022-11-08 | Terapia génica con vectores duales de disferlina |
Country Status (16)
Country | Link |
---|---|
US (1) | US20230279065A1 (es) |
EP (1) | EP4138999A4 (es) |
JP (2) | JP2023519762A (es) |
KR (1) | KR20230009444A (es) |
CN (1) | CN115605266A (es) |
AR (1) | AR123826A1 (es) |
AU (2) | AU2021270526B2 (es) |
BR (1) | BR112022020387A2 (es) |
CA (1) | CA3172664A1 (es) |
CL (1) | CL2022003099A1 (es) |
CO (1) | CO2022016968A2 (es) |
IL (1) | IL297656A (es) |
MX (1) | MX2022014066A (es) |
NZ (1) | NZ793468A (es) |
TW (1) | TW202208407A (es) |
WO (1) | WO2021231577A1 (es) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2024020574A2 (en) * | 2022-07-21 | 2024-01-25 | New York University | Auf1 gene therapy for limb girdle muscular dystrophy |
Family Cites Families (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2000011157A1 (en) * | 1998-08-25 | 2000-03-02 | The General Hospital Corporation | Dysferlin, a gene mutated in distal myopathy and limb girdle muscular dystrophy |
US20030110526A1 (en) * | 1998-08-25 | 2003-06-12 | Robert H. Brown | Dysferlin mutations |
RU2019100990A (ru) * | 2016-06-17 | 2020-07-17 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Укороченный фисферлин для лечения фисферлинопатии |
EP3833780A4 (en) * | 2018-08-10 | 2022-05-11 | Unm Rainforest Innovations | METHODS FOR DETECTING HEREDITARY MYOPATHIES IN HORSES |
-
2021
- 2021-05-12 JP JP2022567513A patent/JP2023519762A/ja active Pending
- 2021-05-12 WO PCT/US2021/031998 patent/WO2021231577A1/en active Application Filing
- 2021-05-12 US US17/924,820 patent/US20230279065A1/en active Pending
- 2021-05-12 IL IL297656A patent/IL297656A/en unknown
- 2021-05-12 AU AU2021270526A patent/AU2021270526B2/en active Active
- 2021-05-12 NZ NZ793468A patent/NZ793468A/en unknown
- 2021-05-12 BR BR112022020387A patent/BR112022020387A2/pt unknown
- 2021-05-12 TW TW110117182A patent/TW202208407A/zh unknown
- 2021-05-12 MX MX2022014066A patent/MX2022014066A/es unknown
- 2021-05-12 KR KR1020227042999A patent/KR20230009444A/ko not_active IP Right Cessation
- 2021-05-12 CN CN202180033036.8A patent/CN115605266A/zh active Pending
- 2021-05-12 EP EP21803851.1A patent/EP4138999A4/en active Pending
- 2021-05-12 CA CA3172664A patent/CA3172664A1/en active Pending
- 2021-05-12 AR ARP210101301A patent/AR123826A1/es unknown
-
2022
- 2022-11-08 CL CL2022003099A patent/CL2022003099A1/es unknown
- 2022-11-26 CO CONC2022/0016968A patent/CO2022016968A2/es unknown
-
2023
- 2023-07-18 AU AU2023206111A patent/AU2023206111A1/en active Pending
-
2024
- 2024-02-02 JP JP2024014797A patent/JP2024032967A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
AU2021270526A1 (en) | 2022-11-24 |
NZ793468A (en) | 2023-07-28 |
JP2023519762A (ja) | 2023-05-12 |
TW202208407A (zh) | 2022-03-01 |
BR112022020387A2 (pt) | 2022-11-29 |
EP4138999A4 (en) | 2024-01-10 |
CA3172664A1 (en) | 2021-11-18 |
US20230279065A1 (en) | 2023-09-07 |
CN115605266A (zh) | 2023-01-13 |
JP2024032967A (ja) | 2024-03-12 |
KR20230009444A (ko) | 2023-01-17 |
WO2021231577A1 (en) | 2021-11-18 |
CO2022016968A2 (es) | 2022-12-09 |
AU2023206111A1 (en) | 2023-08-10 |
EP4138999A1 (en) | 2023-03-01 |
IL297656A (en) | 2022-12-01 |
AR123826A1 (es) | 2023-01-18 |
MX2022014066A (es) | 2022-12-07 |
AU2021270526B2 (en) | 2023-04-20 |
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