MX2019004843A - Terapia genica para esclerosis lateral amiotrofica y otros trastornos de la medula espinal. - Google Patents
Terapia genica para esclerosis lateral amiotrofica y otros trastornos de la medula espinal.Info
- Publication number
- MX2019004843A MX2019004843A MX2019004843A MX2019004843A MX2019004843A MX 2019004843 A MX2019004843 A MX 2019004843A MX 2019004843 A MX2019004843 A MX 2019004843A MX 2019004843 A MX2019004843 A MX 2019004843A MX 2019004843 A MX2019004843 A MX 2019004843A
- Authority
- MX
- Mexico
- Prior art keywords
- spinal cord
- transgene
- subject
- gene therapy
- lateral sclerosis
- Prior art date
Links
- 208000029033 Spinal Cord disease Diseases 0.000 title 1
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 108700019146 Transgenes Proteins 0.000 abstract 5
- 210000004556 brain Anatomy 0.000 abstract 3
- 239000013603 viral vector Substances 0.000 abstract 3
- 239000000203 mixture Substances 0.000 abstract 2
- 230000000508 neurotrophic effect Effects 0.000 abstract 2
- 210000000278 spinal cord Anatomy 0.000 abstract 2
- 108010067390 Viral Proteins Proteins 0.000 abstract 1
- 241000700605 Viruses Species 0.000 abstract 1
- 230000006378 damage Effects 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 208000015181 infectious disease Diseases 0.000 abstract 1
- 230000007659 motor function Effects 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/22—Hormones
- A61K38/30—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
-
- 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
- A61K48/0075—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
- 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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
-
- 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
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- 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
- 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
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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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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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
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Genetics & Genomics (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Gastroenterology & Hepatology (AREA)
- Endocrinology (AREA)
- Zoology (AREA)
- Diabetes (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Psychiatry (AREA)
- Pain & Pain Management (AREA)
- Hospice & Palliative Care (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Abstract
La presente invención proporciona métodos y composiciones para tratar enfermedades o lesiones que afectan la función y control motores en un sujeto. En un aspecto, en la invención se administra un producto de transgenes a la médula espinal de un sujeto mediante la administración al cerebro de un vector viral neurotrófico recombinante que contiene el transgen. El vector viral distribuye el transgen en una región del cerebro que essusceptible de infección por el virus y que expresa el producto del gen viral recombinante codificado. Igualmente se proporcionan composiciones para la distribución de un producto de transgenes a la médula espinal de un sujeto mediante la administración al cerebro del sujeto de un vector viral neurotrófico recombinante que contiene el transgen.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US81141906P | 2006-06-07 | 2006-06-07 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2019004843A true MX2019004843A (es) | 2021-06-15 |
Family
ID=38832369
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2008015530A MX2008015530A (es) | 2006-06-07 | 2007-06-07 | Terapia genica para esclerosis lateral amiotrofica y otros trastornos de la medula espinal. |
| MX2015001403A MX364444B (es) | 2006-06-07 | 2007-06-07 | Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la médula espinal. |
| MX2019004843A MX2019004843A (es) | 2006-06-07 | 2008-12-04 | Terapia genica para esclerosis lateral amiotrofica y otros trastornos de la medula espinal. |
| MX2023001965A MX2023001965A (es) | 2006-06-07 | 2008-12-04 | Terapia genica para esclerosis lateral amiotrofica y otros trastornos de la medula espinal. |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2008015530A MX2008015530A (es) | 2006-06-07 | 2007-06-07 | Terapia genica para esclerosis lateral amiotrofica y otros trastornos de la medula espinal. |
| MX2015001403A MX364444B (es) | 2006-06-07 | 2007-06-07 | Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la médula espinal. |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2023001965A MX2023001965A (es) | 2006-06-07 | 2008-12-04 | Terapia genica para esclerosis lateral amiotrofica y otros trastornos de la medula espinal. |
Country Status (19)
| Country | Link |
|---|---|
| US (4) | US9034836B2 (es) |
| EP (3) | EP3540054A3 (es) |
| JP (6) | JP2009539847A (es) |
| CN (1) | CN101460621B (es) |
| AR (2) | AR061282A1 (es) |
| BR (1) | BRPI0711965A2 (es) |
| CA (1) | CA2654292C (es) |
| CY (1) | CY1118555T1 (es) |
| ES (2) | ES2596885T3 (es) |
| HU (2) | HUE044182T2 (es) |
| IL (2) | IL195647A (es) |
| LT (2) | LT2029742T (es) |
| MX (4) | MX2008015530A (es) |
| PL (2) | PL2489733T3 (es) |
| PT (2) | PT2489733T (es) |
| RU (1) | RU2008152821A (es) |
| SI (2) | SI2029742T1 (es) |
| TR (1) | TR201906106T4 (es) |
| WO (1) | WO2007146046A2 (es) |
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| BRPI0711965A2 (pt) | 2006-06-07 | 2012-01-24 | Genzyme Corp | terapia de gene para esclerose lateral amiotrófica e outras enfermidades da medula espinal |
| EP3009143B1 (en) * | 2008-01-16 | 2018-10-03 | Neurodyn Life Sciences Inc. | Progranulin for use in treating parkinson's disease or alzheimer's disease |
| CN102272321A (zh) | 2008-10-31 | 2011-12-07 | 神经动力公司 | 神经毒性甾醇糖苷 |
| CN102459611B (zh) | 2009-05-02 | 2016-11-09 | 建新公司 | 神经退行性疾病的基因治疗 |
| CN103491974A (zh) * | 2010-11-16 | 2014-01-01 | 神经机能生命科学公司 | 用于提高中性溶酶的表达与活性的方法 |
| CN103083667A (zh) * | 2011-11-01 | 2013-05-08 | 北京清美联创干细胞科技有限公司 | 嗅鞘细胞增殖试剂的连续给药方案 |
| EP2850093B1 (en) | 2012-01-27 | 2019-01-09 | The Board of Trustees of the Leland Stanford Junior University | Therapeutic il-13 polypeptides |
| US9738696B2 (en) | 2012-08-09 | 2017-08-22 | The Board Of Trustees Of The Leland Stanford Junior University | Superkines and synthekines: repurposed cytokines with new and enhanced signaling activities |
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| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| AU2018227440B2 (en) | 2017-02-28 | 2024-06-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clade f vector and uses therefor |
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| AU2003215297A1 (en) * | 2002-02-15 | 2003-09-09 | Cornell Research Foundation, Inc. | Enhancing neurotrophin-induced neurogenesis by endogenous neural progenitor cells by concurrent overexpression of brain derived neurotrophic factor and an inhibitor of a pro-gliogenic bone morphogenetic protein |
| US20060246036A1 (en) * | 2002-09-06 | 2006-11-02 | The General Hospital Corporation | Delivery of therapeutics to the brain and spinal cord |
| JPWO2006035741A1 (ja) * | 2004-09-29 | 2008-05-15 | 伸弥 山中 | Ｅｓ細胞特異的発現遺伝子及びその利用 |
| JP5103615B2 (ja) * | 2004-11-18 | 2012-12-19 | 国立大学法人 東京医科歯科大学 | 新規タンパク質及びそれを利用したポリグルタミン病等の神経変性疾患の予防・治療薬 |
| MX2007013734A (es) * | 2005-05-02 | 2008-03-14 | Genzyme Corp | Terapia genica para trastornos de la medula espinal. |
| PL3520823T3 (pl) | 2005-05-02 | 2021-11-22 | Genzyme Corporation | Terapia genowa zaburzeń neurometabolicznych |
| BRPI0711965A2 (pt) | 2006-06-07 | 2012-01-24 | Genzyme Corp | terapia de gene para esclerose lateral amiotrófica e outras enfermidades da medula espinal |
| SI2773354T1 (sl) | 2011-11-01 | 2019-08-30 | Resverlogix Corp. | Peroralne formulacije s takojšnjim sproščanjem za substituirane kinazolinone |
| WO2013190059A1 (en) | 2012-06-21 | 2013-12-27 | Association Institut De Myologie | Widespread gene delivery of gene therapy vectors |
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