RU2019120721A - Генная терапия для лечения мукополисахаридоза i типа - Google Patents
Генная терапия для лечения мукополисахаридоза i типа Download PDFInfo
- Publication number
- RU2019120721A RU2019120721A RU2019120721A RU2019120721A RU2019120721A RU 2019120721 A RU2019120721 A RU 2019120721A RU 2019120721 A RU2019120721 A RU 2019120721A RU 2019120721 A RU2019120721 A RU 2019120721A RU 2019120721 A RU2019120721 A RU 2019120721A
- Authority
- RU
- Russia
- Prior art keywords
- polynucleotide
- promoter
- ltr
- mps
- hiv
- Prior art date
Links
Classifications
-
- 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
- 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
- A61P19/00—Drugs for skeletal disorders
-
- 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
- A61P25/28—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
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular 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
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—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)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Cardiology (AREA)
- Heart & Thoracic Surgery (AREA)
- Ophthalmology & Optometry (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Physical Education & Sports Medicine (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Diabetes (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662430795P | 2016-12-06 | 2016-12-06 | |
| US62/430,795 | 2016-12-06 | ||
| PCT/US2017/064913 WO2018106807A1 (en) | 2016-12-06 | 2017-12-06 | Gene therapy for mucopolysaccharidosis, type i |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RU2019120721A true RU2019120721A (ru) | 2021-01-11 |
Family
ID=62491308
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2019120721A RU2019120721A (ru) | 2016-12-06 | 2017-12-06 | Генная терапия для лечения мукополисахаридоза i типа |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20220339296A1 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3551750A4 (cg-RX-API-DMAC7.html) |
| JP (1) | JP2019536484A (cg-RX-API-DMAC7.html) |
| KR (1) | KR20190089988A (cg-RX-API-DMAC7.html) |
| CN (1) | CN110214182A (cg-RX-API-DMAC7.html) |
| AU (1) | AU2017370662A1 (cg-RX-API-DMAC7.html) |
| BR (1) | BR112019011635A2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA3046079A1 (cg-RX-API-DMAC7.html) |
| IL (1) | IL267057A (cg-RX-API-DMAC7.html) |
| MA (1) | MA47847A (cg-RX-API-DMAC7.html) |
| MX (1) | MX2019006552A (cg-RX-API-DMAC7.html) |
| RU (1) | RU2019120721A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2018106807A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR102673203B1 (ko) | 2016-04-20 | 2024-06-10 | 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. | Pklr의 유전자 발현 증강을 위한 조성물 및 방법 |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| AU2018352583B2 (en) | 2017-10-16 | 2025-04-17 | Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. | Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency |
| US12539316B2 (en) | 2018-04-11 | 2026-02-03 | Spacecraft Seven, Llc | Compositions and methods for stem cell transplant |
| IL280465B2 (en) * | 2018-07-30 | 2025-03-01 | Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P | Methods for gene modification of hematopoietic cells |
| CN115109790A (zh) * | 2021-03-23 | 2022-09-27 | 北京据德医药科技有限公司 | 一种重组a-L-艾杜糖醛酸普酶及其制备方法 |
| MX2023014228A (es) * | 2021-06-08 | 2024-01-25 | Touchlight Ip Ltd | Vector lentiviral. |
| WO2024201066A1 (en) * | 2023-03-31 | 2024-10-03 | Dawn Therapeutics Limited | Novel vector |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| FR2872170B1 (fr) * | 2004-06-25 | 2006-11-10 | Centre Nat Rech Scient Cnrse | Lentivirus non interactif et non replicatif, preparation et utilisations |
| US20110294114A1 (en) * | 2009-12-04 | 2011-12-01 | Cincinnati Children's Hospital Medical Center | Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells |
| DK2717922T3 (en) * | 2011-06-10 | 2019-01-07 | Bluebird Bio Inc | GENERATIVE VECTORS FOR ADDRENOLEUCODYSTROPHY AND ADDRENOMYELONE EUROPE |
| EP2855684A1 (en) * | 2012-05-25 | 2015-04-08 | Commissariat à l'Énergie Atomique et aux Énergies Alternatives | Vector for the selective silencing of a gene in astrocytes |
| WO2014201252A2 (en) * | 2013-06-13 | 2014-12-18 | Shire Human Genetic Therapies, Inc. | Messenger rna based viral production |
| CN108883136A (zh) * | 2016-02-12 | 2018-11-23 | 蓝鸟生物公司 | Vcn增强子组合物及其使用方法 |
-
2017
- 2017-12-06 MX MX2019006552A patent/MX2019006552A/es unknown
- 2017-12-06 CA CA3046079A patent/CA3046079A1/en not_active Abandoned
- 2017-12-06 AU AU2017370662A patent/AU2017370662A1/en not_active Abandoned
- 2017-12-06 RU RU2019120721A patent/RU2019120721A/ru not_active Application Discontinuation
- 2017-12-06 MA MA047847A patent/MA47847A/fr unknown
- 2017-12-06 EP EP17877511.0A patent/EP3551750A4/en not_active Withdrawn
- 2017-12-06 JP JP2019551256A patent/JP2019536484A/ja active Pending
- 2017-12-06 CN CN201780083351.5A patent/CN110214182A/zh active Pending
- 2017-12-06 US US16/466,130 patent/US20220339296A1/en not_active Abandoned
- 2017-12-06 BR BR112019011635-4A patent/BR112019011635A2/pt not_active IP Right Cessation
- 2017-12-06 WO PCT/US2017/064913 patent/WO2018106807A1/en not_active Ceased
- 2017-12-06 KR KR1020197019344A patent/KR20190089988A/ko not_active Ceased
-
2019
- 2019-06-03 IL IL267057A patent/IL267057A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| US20220339296A1 (en) | 2022-10-27 |
| CA3046079A1 (en) | 2018-06-14 |
| MA47847A (fr) | 2020-01-29 |
| BR112019011635A2 (pt) | 2019-11-12 |
| IL267057A (en) | 2019-08-29 |
| JP2019536484A (ja) | 2019-12-19 |
| EP3551750A4 (en) | 2020-07-22 |
| AU2017370662A1 (en) | 2019-06-27 |
| MX2019006552A (es) | 2019-10-15 |
| CN110214182A (zh) | 2019-09-06 |
| WO2018106807A1 (en) | 2018-06-14 |
| KR20190089988A (ko) | 2019-07-31 |
| EP3551750A1 (en) | 2019-10-16 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| RU2019120663A (ru) | Генная терапия для лечения мукополисахаридоза ii типа | |
| JP2019536484A5 (cg-RX-API-DMAC7.html) | ||
| JP2019517281A5 (cg-RX-API-DMAC7.html) | ||
| JP2020500562A5 (cg-RX-API-DMAC7.html) | ||
| RU2021103425A (ru) | Композиции, повышающие число копий вектора (чкв), и способы их применения | |
| Hirsch et al. | The genome organization of STLV-3 is similar to that of the AIDS virus except for a truncated transmembrane protein | |
| Ailles et al. | HIV-1-derived lentiviral vectors | |
| Durand et al. | The inside out of lentiviral vectors | |
| Fäcke et al. | A large deletion in the matrix domain of the human immunodeficiency virus gag gene redirects virus particle assembly from the plasma membrane to the endoplasmic reticulum | |
| Ramezani et al. | Overview of the HIV‐1 lentiviral vector system | |
| Miyazawa et al. | The genome of feline immunodeficiency virus | |
| Peterlin et al. | Molecular biology of HIV | |
| Yang et al. | Role of the gp120 inner domain β-sandwich in the interaction between the human immunodeficiency virus envelope glycoprotein subunits | |
| Parolin et al. | Use ofcis-andtrans-Acting Viral Regulatory Sequences to Improve Expression of Human Immunodeficiency Virus Vectors in Human Lymphocytes | |
| JP2014519335A5 (cg-RX-API-DMAC7.html) | ||
| Vodicka | Determinants for lentiviral infection of non-dividing cells | |
| Saenz et al. | FIV: from lentivirus to lentivector | |
| Liu et al. | HIV-1-based lentiviral vectors | |
| Pandya et al. | Development of an Rev-independent, minimal simian immunodeficiency virus-derived vector system | |
| Pallansch et al. | Bovine immunodeficiency-like virus encodes factors which trans activate the long terminal repeat | |
| Perez et al. | Suppression of HIV-1 infection in primary CD4 T cells transduced with a self-inactivating lentiviral vector encoding a membrane expressed gp41-derived fusion inhibitor | |
| Trono | Lentiviral vectors | |
| Naldini | In vivo gene delivery by lentiviral vectors | |
| Browning et al. | Sequences within the gag gene of feline immunodeficiency virus (FIV) are important for efficient RNA encapsidation | |
| WO2003080846A1 (fr) | Procede de production de vecteurs lentiviraux a haut titre |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FA93 | Acknowledgement of application withdrawn (no request for examination) |
Effective date: 20201207 |