TR201906106T4 - Amiyotrofik lateral skleroz ve diğer omurilik hastalıkları için gen tedavisi. - Google Patents

Amiyotrofik lateral skleroz ve diğer omurilik hastalıkları için gen tedavisi. Download PDF

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Publication number
TR201906106T4
TR201906106T4 TR2019/06106T TR201906106T TR201906106T4 TR 201906106 T4 TR201906106 T4 TR 201906106T4 TR 2019/06106 T TR2019/06106 T TR 2019/06106T TR 201906106 T TR201906106 T TR 201906106T TR 201906106 T4 TR201906106 T4 TR 201906106T4
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spinal cord
transgene
subject
gene therapy
brain
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TR2019/06106T
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Dodge James
Shihabuddin Lamya
R O'riordan Catherine
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Genzyme Corp
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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
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    • A—HUMAN NECESSITIES
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    • 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
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    • 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
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    • 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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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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    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
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Abstract

Bu açıklama, bir denekteki motor fonksiyonu ve kontrolü etkileyen hastalıkların ya da hasarların tedavisi için yöntemleri ve bileşimleri sağlar. Buluşun bir yönüyle, bir transgen ürünü; beyne, transgeni içeren bir rekombinant nörotrofik viral vektörün tatbik edilmesi ile bir deneğin omuriliğine taşınır. Viral vektör, virüs tarafından enfeksiyona duyarlı olan ve kodlanmış bir rekombinant viral gen ürününü ifade eden beynin bir bölgesine transgeni dağıtır. Ayrıca burada; deneğin beynine, transgeni içeren bir rekombinant nörotrofik viral vektörünün tatbik edilmesi ile bir deneğin omuriliğine bir transgen ürününün dağıtılması için bileşimleri de sağlanır.
TR2019/06106T 2006-06-07 2007-06-07 Amiyotrofik lateral skleroz ve diğer omurilik hastalıkları için gen tedavisi. TR201906106T4 (tr)

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EP (3) EP3540054A3 (tr)
JP (6) JP2009539847A (tr)
CN (1) CN101460621B (tr)
AR (2) AR061282A1 (tr)
BR (1) BRPI0711965A2 (tr)
CA (2) CA3139726C (tr)
CY (1) CY1118555T1 (tr)
ES (2) ES2596885T3 (tr)
HU (2) HUE044182T2 (tr)
IL (2) IL195647A (tr)
LT (2) LT2489733T (tr)
MX (4) MX364444B (tr)
PL (2) PL2029742T3 (tr)
PT (2) PT2489733T (tr)
RU (1) RU2008152821A (tr)
SI (2) SI2489733T1 (tr)
TR (1) TR201906106T4 (tr)
WO (1) WO2007146046A2 (tr)

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EP2029742A4 (en) 2009-09-30
JP2017078071A (ja) 2017-04-27
MX2023001965A (es) 2023-02-23
EP2029742A2 (en) 2009-03-04
WO2007146046A3 (en) 2008-05-22
US9034836B2 (en) 2015-05-19
PT2029742T (pt) 2016-09-09
ES2725552T3 (es) 2019-09-24
PT2489733T (pt) 2019-05-27
BRPI0711965A2 (pt) 2012-01-24
JP2018118995A (ja) 2018-08-02
US20230364200A1 (en) 2023-11-16
CA2654292C (en) 2022-01-11
CY1118555T1 (el) 2017-07-12
RU2008152821A (ru) 2010-07-20
WO2007146046A2 (en) 2007-12-21
JP6606215B2 (ja) 2019-11-13
ES2596885T3 (es) 2017-01-12
CA3139726C (en) 2026-01-20
JP2015120719A (ja) 2015-07-02
CA3139726A1 (en) 2007-12-21
EP2489733A2 (en) 2012-08-22
JP2014012697A (ja) 2014-01-23
SI2029742T1 (sl) 2016-11-30
JP2009539847A (ja) 2009-11-19
CA2654292A1 (en) 2007-12-21
LT2489733T (lt) 2019-05-27
US20100267812A1 (en) 2010-10-21
CN101460621B (zh) 2016-01-27
HUE044182T2 (hu) 2019-10-28
US20200101138A1 (en) 2020-04-02
HUE031156T2 (en) 2017-06-28
US11554161B2 (en) 2023-01-17
US20160067312A1 (en) 2016-03-10
MX2019004843A (es) 2021-06-15
MX364444B (es) 2019-04-26
EP3540054A3 (en) 2019-10-09
AR061282A1 (es) 2008-08-20
EP2489733A3 (en) 2012-11-14
IL195647A0 (en) 2009-09-01
LT2029742T (lt) 2016-11-10
IL241890A0 (en) 2015-11-30
EP2489733B1 (en) 2019-02-13
IL195647A (en) 2015-10-29
AR109171A2 (es) 2018-11-07
SI2489733T1 (sl) 2019-06-28
EP2029742B1 (en) 2016-07-20
EP3540054A2 (en) 2019-09-18
PL2029742T3 (pl) 2017-08-31
MX2008015530A (es) 2009-02-06
CN101460621A (zh) 2009-06-17
JP6320489B2 (ja) 2018-05-09
JP2020079234A (ja) 2020-05-28
PL2489733T3 (pl) 2019-08-30

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