AR063113A1 - Terapia genica para esclerosis lateral amiotrofica y otros trastornos del cordon espinal - Google Patents

Terapia genica para esclerosis lateral amiotrofica y otros trastornos del cordon espinal

Info

Publication number
AR063113A1
AR063113A1 ARP070104379A ARP070104379A AR063113A1 AR 063113 A1 AR063113 A1 AR 063113A1 AR P070104379 A ARP070104379 A AR P070104379A AR P070104379 A ARP070104379 A AR P070104379A AR 063113 A1 AR063113 A1 AR 063113A1
Authority
AR
Argentina
Prior art keywords
spinal cord
disorders
gene therapy
subject
lateral sclerosis
Prior art date
Application number
ARP070104379A
Other languages
English (en)
Inventor
Catherine E Riordan
Samuel C Wadsworth
Original Assignee
Genzyme Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of AR063113A1 publication Critical patent/AR063113A1/es

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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
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14145—Special targeting system for 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
    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
    • 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
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • 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
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La invencion comprende métodos y composiciones para tratar lesiones o trastornos que afectan tanto el control como la funcion motora en un sujeto. En un aspecto, la presente es un producto transgénico suministrado en el cordon espinal de un sujeto mediante la administracion en el cordon espinal de un vector viral recombinante que contiene el transgen. El vector viral suministra el transgen que expresa el producto de gen recombinante viral codificado. El producto de gen viral comprende HIF1- alfa. También se contemplan composiciones para el suministro de un producto transgénico al cordon espinal de un sujeto.
ARP070104379A 2006-10-03 2007-10-03 Terapia genica para esclerosis lateral amiotrofica y otros trastornos del cordon espinal AR063113A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US82797706P 2006-10-03 2006-10-03

Publications (1)

Publication Number Publication Date
AR063113A1 true AR063113A1 (es) 2008-12-30

Family

ID=39269037

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP070104379A AR063113A1 (es) 2006-10-03 2007-10-03 Terapia genica para esclerosis lateral amiotrofica y otros trastornos del cordon espinal

Country Status (13)

Country Link
US (2) US9890394B2 (es)
EP (4) EP3632472A1 (es)
CN (3) CN105770910A (es)
AR (1) AR063113A1 (es)
CY (1) CY1118477T1 (es)
DK (2) DK2497500T3 (es)
ES (3) ES2607060T3 (es)
HU (2) HUE046709T2 (es)
LT (2) LT3146982T (es)
PL (2) PL2066791T3 (es)
PT (3) PT2066791E (es)
SI (3) SI2066791T1 (es)
WO (1) WO2008042420A2 (es)

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WO2009090933A1 (ja) * 2008-01-16 2009-07-23 Japan Science And Technology Agency 低酸素状態にある細胞で増殖するウイルスまたは遺伝子を発現するウイルスベクター
RU2603740C2 (ru) 2009-05-02 2016-11-27 Джензим Корпорейшн Генная терапия нейродегенеративных нарушений
WO2013025963A2 (en) 2011-08-17 2013-02-21 President And Fellows Of Harvard College Conversion of somatic cells into functional spinal motor neurons, and methods and uses thereof
SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
WO2016122791A1 (en) * 2015-01-30 2016-08-04 The Regents Of The University Of California Spinal subpial gene delivery system
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.
CN108884022B (zh) 2016-03-28 2022-01-28 加利福尼亚大学董事会 用于治疗神经元的过度兴奋的方法和组合物
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
MX2019005874A (es) * 2016-11-17 2020-02-07 Nationwide Childrens Hospital Inc Administracion intratecal de virus recombinante adeno-asociado que codifica la proteina 2 que une metilo-cpg.
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
US11603542B2 (en) 2017-05-05 2023-03-14 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
CN111094327A (zh) * 2017-08-16 2020-05-01 Lgv1有限公司 Bpifb4蛋白的用于神经元疾病和损伤的vtft同种型
WO2019051202A1 (en) 2017-09-08 2019-03-14 The Regents Of The University Of California METHOD AND COMPOSITION FOR TREATING NEUROPATHIC PAIN
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
AU2018352236A1 (en) 2017-10-16 2020-04-23 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
WO2020146718A1 (en) 2019-01-10 2020-07-16 The Regents Of The University Of California Subpial delivery system and methods of use
USD963164S1 (en) 2020-01-09 2022-09-06 The Regents Of The University Of California Surgical needle
EP4370679A2 (en) * 2021-07-16 2024-05-22 President and Fellows of Harvard College Enhancers driving expression in motor neurons

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Also Published As

Publication number Publication date
US20090286857A1 (en) 2009-11-19
CN101528922A (zh) 2009-09-09
US20180230490A1 (en) 2018-08-16
DK2066791T3 (da) 2012-10-15
PT2066791E (pt) 2012-12-05
EP2497500B1 (en) 2016-09-28
EP2066791A2 (en) 2009-06-10
LT3146982T (lt) 2019-11-11
ES2607060T3 (es) 2017-03-29
ES2757723T3 (es) 2020-04-30
EP2066791A4 (en) 2010-11-03
LT2497500T (lt) 2016-12-27
US9890394B2 (en) 2018-02-13
PL2497500T3 (pl) 2017-07-31
EP3146982B1 (en) 2019-08-21
CY1118477T1 (el) 2017-07-12
PT3146982T (pt) 2019-10-31
DK2497500T3 (da) 2017-01-02
EP3146982A1 (en) 2017-03-29
EP2066791B1 (en) 2012-09-12
EP2497500A1 (en) 2012-09-12
ES2391789T3 (es) 2012-11-29
SI2497500T1 (sl) 2017-01-31
CN108853517A (zh) 2018-11-23
WO2008042420A2 (en) 2008-04-10
EP3632472A1 (en) 2020-04-08
SI3146982T1 (sl) 2019-11-29
PL2066791T3 (pl) 2013-02-28
HUE046709T2 (hu) 2020-03-30
SI2066791T1 (sl) 2013-01-31
HUE031182T2 (en) 2017-06-28
PT2497500T (pt) 2016-11-23
WO2008042420A3 (en) 2008-07-03
US10920245B2 (en) 2021-02-16
CN105770910A (zh) 2016-07-20

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