MX364444B - Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la médula espinal. - Google Patents
Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la médula espinal.Info
- Publication number
- MX364444B MX364444B MX2015001403A MX2015001403A MX364444B MX 364444 B MX364444 B MX 364444B MX 2015001403 A MX2015001403 A MX 2015001403A MX 2015001403 A MX2015001403 A MX 2015001403A MX 364444 B MX364444 B MX 364444B
- 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
-
- 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)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Neurology (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Biotechnology (AREA)
- Genetics & Genomics (AREA)
- Neurosurgery (AREA)
- Biomedical Technology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Diabetes (AREA)
- Endocrinology (AREA)
- Zoology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pain & Pain Management (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 se refiere a 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 es susceptible 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 (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US81141906P | 2006-06-07 | 2006-06-07 | |
| PCT/US2007/013391 WO2007146046A2 (en) | 2006-06-07 | 2007-06-07 | Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX364444B true MX364444B (es) | 2019-04-26 |
Family
ID=38832369
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2015001403A MX364444B (es) | 2006-06-07 | 2007-06-07 | Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la médula espinal. |
| MX2008015530A MX2008015530A (es) | 2006-06-07 | 2007-06-07 | 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. |
| MX2019004843A MX2019004843A (es) | 2006-06-07 | 2008-12-04 | Terapia genica para esclerosis lateral amiotrofica y otros trastornos de la medula espinal. |
Family Applications After (3)
| 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. |
| MX2023001965A MX2023001965A (es) | 2006-06-07 | 2008-12-04 | Terapia genica para esclerosis lateral amiotrofica y otros trastornos de la medula espinal. |
| MX2019004843A MX2019004843A (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 (2) | CA3139726A1 (es) |
| CY (1) | CY1118555T1 (es) |
| ES (2) | ES2596885T3 (es) |
| HU (2) | HUE044182T2 (es) |
| IL (2) | IL195647A (es) |
| LT (2) | LT2489733T (es) |
| MX (4) | MX364444B (es) |
| PL (2) | PL2489733T3 (es) |
| PT (2) | PT2029742T (es) |
| RU (1) | RU2008152821A (es) |
| SI (2) | SI2029742T1 (es) |
| TR (1) | TR201906106T4 (es) |
| WO (1) | WO2007146046A2 (es) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX364444B (es) | 2006-06-07 | 2019-04-26 | Genzyme Corp | Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la médula espinal. |
| ES2596360T3 (es) * | 2008-01-16 | 2017-01-09 | Neurodyn Life Sciences Inc. | Progranulina para su uso en el tratamiento de enfermedad de Parkinson o enfermedad de Alzheimer |
| US9573972B2 (en) | 2008-10-31 | 2017-02-21 | Neurodyn, Inc. | Nurotoxic sterol glycosides |
| RS62779B1 (sr) | 2009-05-02 | 2022-01-31 | Genzyme Corp | Genska terapija neurodegenerativnih poremećaja |
| EP2640407A4 (en) * | 2010-11-16 | 2014-07-09 | Denis G Kay | PROCESS FOR INCREASING THE EXPRESSION AND ACTIVITY OF NEPRILYSIN |
| 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 |
| US9512194B2 (en) | 2012-01-27 | 2016-12-06 | The Board Of Trustees Of The Leland Stanford Junior University | Modified IL-13 polypeptides |
| US20140065110A1 (en) | 2012-08-31 | 2014-03-06 | The Regents Of The University Of California | Genetically modified msc and therapeutic methods |
| US9663564B2 (en) | 2013-03-15 | 2017-05-30 | The Regents Of The University Of California | Vectors and methods to treat ischemia |
| EP2913063B1 (en) | 2012-10-24 | 2019-09-11 | Daiichi Sankyo Company, Limited | Therapeutic agent for use in treating amyotrophic lateral sclerosis |
| WO2014084085A1 (ja) * | 2012-11-28 | 2014-06-05 | 国立大学法人名古屋大学 | 聴覚障害又は小脳性運動失調症の予防・治療剤 |
| CA2911105A1 (en) * | 2013-05-01 | 2014-11-06 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
| MX2016001026A (es) * | 2013-07-26 | 2016-08-03 | Univ Iowa Res Found | Metodos y composiciones para tratar enfermedades del cerebro. |
| KR101548989B1 (ko) | 2013-07-30 | 2015-09-01 | 광주과학기술원 | ＳｉｒＴ１을 이용한 관절 질환의 예방 또는 치료용 물질의 스크리닝 방법 |
| US9119832B2 (en) | 2014-02-05 | 2015-09-01 | The Regents Of The University Of California | Methods of treating mild brain injury |
| DE102014207498A1 (de) * | 2014-04-17 | 2015-10-22 | Universitätsklinikum Hamburg-Eppendorf | Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark |
| CA2966620A1 (en) | 2014-11-05 | 2016-05-12 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| MX2017014966A (es) * | 2015-05-28 | 2018-08-15 | Univ Cornell | Liberación de c1ei mediada por virus adenoasociado como terapia para el angioedema. |
| CL2015003024A1 (es) | 2015-10-09 | 2016-05-13 | Univ Chile | Método de tratamiento genético utilizando el virus aav-xbp1s/gfp, y su uso en la prevención y tratamiento de la esclerosis lateral amiotrofica. |
| EP3377073A4 (en) * | 2015-11-16 | 2019-06-26 | Ohio State Innovation Foundation | METHOD AND COMPOSITIONS FOR TREATING DISORDERS AND DISEASES WITH THE SURVIVAL MOTOR NEURON (SMN) PROTEIN |
| CA3016314A1 (en) * | 2016-03-02 | 2017-09-08 | Julianne REIDERS | Therapy for frontotemporal dementia |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| MX2019010275A (es) | 2017-02-28 | 2019-10-15 | Univ Pennsylvania | Novedoso vector del subtipo f del virus adenoasociado (aav) y usos de este. |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CN111492061A (zh) | 2017-10-03 | 2020-08-04 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| MX2020004005A (es) | 2017-10-03 | 2020-10-05 | Prevail Therapeutics Inc | Terapias genicas para los trastornos lisosomales. |
| EP3692075A4 (en) | 2017-10-03 | 2022-02-09 | Prevail Therapeutics, Inc. | GENE THERAPIES FOR LYSOSOMAL DISEASES |
| US20200390861A1 (en) * | 2017-10-10 | 2020-12-17 | Medicenna Therapeutics, Inc. | Il-4-fusion formulations for treatment of central nervous system (cns) tumors |
| RU2731514C2 (ru) * | 2018-12-21 | 2020-09-03 | Селл энд Джин Терапи Лтд | Генотерапевтический ДНК-вектор на основе генотерапевтического ДНК-вектора VTvaf17, несущий целевой ген, выбранный из группы генов SHH, CTNNB1, NOG, WNT7A для повышения уровня экспрессии этих целевых генов, способ его получения и применения, штамм Escherichia coli SCS110-AF/VTvaf17-SHH, или Escherichia coli SCS110-AF/VTvaf17-CTNNB1, или Escherichia coli SCS110-AF/VTvaf17-NOG, или Escherichia coli SCS110-AF/VTvaf17-WNT7A, несущий генотерапевтический ДНК-вектор, способ его получения, способ производства в промышленных масштабах генотерапевтического ДНК-вектора |
| JP2022523913A (ja) * | 2019-02-01 | 2022-04-27 | アブロバイオ，インコーポレーテッド | 神経認知障害を処置するための組成物及び方法 |
| JP2022523766A (ja) * | 2019-02-22 | 2022-04-26 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Ｇｒｎ関連成人発症性神経変性の治療のための組換えアデノ随伴ウイルス |
| CN114174324A (zh) | 2019-04-10 | 2022-03-11 | 普利维尔治疗公司 | 用于溶酶体病症的基因疗法 |
| CA3136646A1 (en) * | 2019-04-12 | 2020-10-15 | Encoded Therapeutics, Inc. | Compositions and methods for administration of therapeutics |
| CN113660958B (zh) * | 2019-10-12 | 2023-10-31 | 深圳大学 | 使Sirt7基因表达的作用剂及其用途 |
| KR20220130678A (ko) | 2019-12-23 | 2022-09-27 | 데날리 테라퓨틱스 인크. | 프로그라눌린 변이체들 |
| CN115323002A (zh) * | 2022-07-28 | 2022-11-11 | 深圳先进技术研究院 | 一种基因递送系统在从脑部逆行递送基因到脊髓神经元中的应用 |
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| US5672344A (en) | 1987-12-30 | 1997-09-30 | The Regents Of The University Of Michigan | Viral-mediated gene transfer system |
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| MX364444B (es) | 2006-06-07 | 2019-04-26 | Genzyme Corp | Terapia génica para esclerosis lateral amiotrófica y otros trastornos de la médula espinal. |
| RS58911B1 (sr) | 2011-11-01 | 2019-08-30 | Resverlogix Corp | Oralne formulacije sa trenutnim otpuštanjem za supstituisane hinazolinone |
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-
2007
- 2007-06-07 MX MX2015001403A patent/MX364444B/es unknown
- 2007-06-07 PT PT77958353T patent/PT2029742T/pt unknown
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