PT2527457T - Entrega generalizada de genes nos neurónios motores usando injecção periférica de vectores aav - Google Patents
Entrega generalizada de genes nos neurónios motores usando injecção periférica de vectores aavInfo
- Publication number
- PT2527457T PT2527457T PT12180951T PT12180951T PT2527457T PT 2527457 T PT2527457 T PT 2527457T PT 12180951 T PT12180951 T PT 12180951T PT 12180951 T PT12180951 T PT 12180951T PT 2527457 T PT2527457 T PT 2527457T
- Authority
- PT
- Portugal
- Prior art keywords
- gene delivery
- motor neurons
- aav vectors
- peripheral injection
- widespread gene
- Prior art date
Links
Classifications
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- 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
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- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
- A61P17/02—Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
-
- 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
-
- 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
-
- 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
- A61P25/04—Centrally acting analgesics, e.g. opioids
-
- 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
- A61P25/20—Hypnotics; Sedatives
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
-
- 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
- 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0083—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 administration regime
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- 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Immunology (AREA)
- Neurology (AREA)
- Biotechnology (AREA)
- Neurosurgery (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Pain & Pain Management (AREA)
- Transplantation (AREA)
- Hospice & Palliative Care (AREA)
- Anesthesiology (AREA)
- Rheumatology (AREA)
- Psychiatry (AREA)
- Dermatology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP07301435A EP2058401A1 (en) | 2007-10-05 | 2007-10-05 | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
Publications (1)
Publication Number | Publication Date |
---|---|
PT2527457T true PT2527457T (pt) | 2019-05-31 |
Family
ID=39186802
Family Applications (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PT88367768T PT2212424E (pt) | 2007-10-05 | 2008-10-03 | Entrega generalizada de genes nos neurónios motores usando injecção periférica de vectores aav |
PT12180951T PT2527457T (pt) | 2007-10-05 | 2008-10-03 | Entrega generalizada de genes nos neurónios motores usando injecção periférica de vectores aav |
PT191581529T PT3505635T (pt) | 2007-10-05 | 2008-10-03 | Entrega generalizada de genes nos neurónios motores por injeção periférica de vetores aav |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PT88367768T PT2212424E (pt) | 2007-10-05 | 2008-10-03 | Entrega generalizada de genes nos neurónios motores usando injecção periférica de vectores aav |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PT191581529T PT3505635T (pt) | 2007-10-05 | 2008-10-03 | Entrega generalizada de genes nos neurónios motores por injeção periférica de vetores aav |
Country Status (18)
Country | Link |
---|---|
US (3) | US9926574B2 (pt) |
EP (5) | EP2058401A1 (pt) |
JP (2) | JP5620819B2 (pt) |
CN (2) | CN101883859A (pt) |
CA (2) | CA2999607A1 (pt) |
CY (1) | CY1125608T1 (pt) |
DK (2) | DK2212424T3 (pt) |
ES (3) | ES2924892T3 (pt) |
FI (1) | FI2212424T4 (pt) |
FR (2) | FR20C1057I2 (pt) |
HR (2) | HRP20220992T3 (pt) |
HU (2) | HUE059864T2 (pt) |
LT (2) | LT3505635T (pt) |
NO (1) | NO2022048I1 (pt) |
PL (2) | PL3505635T3 (pt) |
PT (3) | PT2212424E (pt) |
SI (2) | SI3505635T1 (pt) |
WO (1) | WO2009043936A1 (pt) |
Families Citing this family (95)
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EP2058401A1 (en) * | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
ES2796085T3 (es) | 2008-01-15 | 2020-11-25 | Univ Leland Stanford Junior | Marcadores de células madre de leucemia mieloide aguda |
US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
US9415121B2 (en) | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
MX356669B (es) | 2009-05-02 | 2018-06-08 | Genzyme Corp | Terapia genica para trastornos neurodegenerativos. |
WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
JP2013533847A (ja) | 2010-04-23 | 2013-08-29 | ユニバーシティ オブ マサチューセッツ | コレステロール関連障害のaavベースの治療 |
US9546369B2 (en) | 2010-04-23 | 2017-01-17 | University Of Massachusetts | Multicistronic expression constructs |
CA3049237C (en) * | 2010-04-23 | 2024-06-11 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
CN103189507A (zh) * | 2010-10-27 | 2013-07-03 | 学校法人自治医科大学 | 用于向神经系统细胞导入基因的腺相关病毒粒子 |
WO2012145624A2 (en) | 2011-04-21 | 2012-10-26 | University Of Massachusetts | Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
US20130039888A1 (en) | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
JP2015523998A (ja) | 2012-06-19 | 2015-08-20 | ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド | 疾患を治療するための組成物および方法 |
CA2877428A1 (en) | 2012-06-21 | 2013-12-27 | Association Institut De Myologie | Widespread gene delivery of gene therapy vectors |
JP6314138B2 (ja) | 2012-08-01 | 2018-04-18 | ネイションワイド チルドレンズ ホスピタル | 組換えアデノ随伴ウイルス9の髄腔内送達 |
CA2885216A1 (en) | 2012-09-17 | 2014-03-20 | The Research Institute At Nationwide Children's Hospital | Compositions and methods for treating amyotrophic lateral sclerosis |
KR20240093939A (ko) * | 2013-05-15 | 2024-06-24 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
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KR102390075B1 (ko) | 2014-03-09 | 2022-04-26 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 오르니틴 트랜스카르바밀라아제(otc) 결핍증의 치료에 유용한 조성물 |
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AU2015364636B9 (en) | 2014-12-16 | 2021-12-02 | Board Of Regents Of The University Of Nebraska | Gene therapy for Juvenile Batten Disease |
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