CL2017002028A1 - Mejora del suministro de partículas virales al cuerpo estriado y al córtex. - Google Patents

Mejora del suministro de partículas virales al cuerpo estriado y al córtex.

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Publication number
CL2017002028A1
CL2017002028A1 CL2017002028A CL2017002028A CL2017002028A1 CL 2017002028 A1 CL2017002028 A1 CL 2017002028A1 CL 2017002028 A CL2017002028 A CL 2017002028A CL 2017002028 A CL2017002028 A CL 2017002028A CL 2017002028 A1 CL2017002028 A1 CL 2017002028A1
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CL
Chile
Prior art keywords
supply
viral particles
striatum
cortex
improvement
Prior art date
Application number
CL2017002028A
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English (en)
Inventor
Lamya Shihabuddin
Lisa M Stanek
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Genzyme Corp
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Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of CL2017002028A1 publication Critical patent/CL2017002028A1/es

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    • A—HUMAN NECESSITIES
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    • 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
    • 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
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00—Drugs for disorders of the nervous system
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    • A61P25/00—Drugs for disorders of the nervous system
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • A61P25/16—Anti-Parkinson drugs
    • 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
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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
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    • C12N2750/00011—Details
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Neurosurgery (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Abstract

<p>LA INVENCIÓN PROPORCIONA NUEVOS MÉTODOS PARA SUMINISTRAR PARTÍCULAS VIRALES ADENO-ASOCIADAS RECOMBINANTES (rAAV) AL SISTEMA NERVIOSO CENTRAL DE UN MAMÍFERO (P. EJ. UN SER HUMANO). EN ASPECTOS, LOS MÉTODOS COMPRENDEN ADMINISTRAR PARTÍCULAS DE rAAV QUE CONTIENEN UN ÁCIDO NUCLÉICO HETERÓLOGO AL CUERPO ESTRIADO Y CAUSAR LA EXPRESIÓN DEL ÁCIDO NUCLÉICO HETERÓLOGO EN AL MENOS EL I CÓRTEX CEREBRAL Y EL CUERPO ESTRIADO DEL MAMÍFERO.</p>
CL2017002028A 2015-02-10 2017-08-08 Mejora del suministro de partículas virales al cuerpo estriado y al córtex. CL2017002028A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562114544P 2015-02-10 2015-02-10
US201562220997P 2015-09-19 2015-09-19

Publications (1)

Publication Number Publication Date
CL2017002028A1 true CL2017002028A1 (es) 2018-04-13

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CL2017002028A CL2017002028A1 (es) 2015-02-10 2017-08-08 Mejora del suministro de partículas virales al cuerpo estriado y al córtex.

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US (2) US20190111157A1 (es)
EP (3) EP3256594B1 (es)
JP (3) JP6928558B2 (es)
KR (2) KR20240010539A (es)
CN (2) CN107530447A (es)
AU (2) AU2016219398A1 (es)
BR (1) BR112017017037A2 (es)
CA (1) CA2976082A1 (es)
CL (1) CL2017002028A1 (es)
CR (1) CR20170407A (es)
DK (1) DK3256594T3 (es)
EA (1) EA201791803A1 (es)
ES (1) ES2904504T3 (es)
HK (1) HK1246340A1 (es)
HU (1) HUE057272T2 (es)
IL (3) IL253894B (es)
LT (1) LT3256594T (es)
MX (2) MX2017010370A (es)
PH (1) PH12017501436A1 (es)
PL (1) PL3256594T3 (es)
PT (1) PT3256594T (es)
RS (1) RS62811B1 (es)
SG (3) SG11201706445SA (es)
SI (1) SI3256594T1 (es)
TN (1) TN2017000353A1 (es)
TW (1) TWI730952B (es)
WO (1) WO2016130591A2 (es)

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CN107530447A (zh) 2018-01-02
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US20220395586A1 (en) 2022-12-15
EP3256594B1 (en) 2021-10-20
SG11201706445SA (en) 2017-09-28
SI3256594T1 (sl) 2022-07-29
CA2976082A1 (en) 2016-08-18
IL285060B2 (en) 2023-02-01
US20190111157A1 (en) 2019-04-18
WO2016130591A2 (en) 2016-08-18
JP6928558B2 (ja) 2021-09-01
MX2021004294A (es) 2021-06-04
PH12017501436A1 (en) 2018-01-15
WO2016130591A3 (en) 2016-11-10
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EP3256594A2 (en) 2017-12-20
TW201642909A (zh) 2016-12-16
TN2017000353A1 (en) 2019-01-16
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AU2016219398A1 (en) 2017-09-28
EP4023758A1 (en) 2022-07-06
IL253894B (en) 2021-08-31
IL296466A (en) 2022-11-01
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DK3256594T3 (da) 2022-01-24
IL285060A (en) 2021-08-31
CN116019934A (zh) 2023-04-28
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SG10201907399RA (en) 2019-09-27
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MX2017010370A (es) 2017-12-14
CR20170407A (es) 2017-11-14
HUE057272T2 (hu) 2022-04-28
JP2023058630A (ja) 2023-04-25
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JP2021178861A (ja) 2021-11-18
TWI730952B (zh) 2021-06-21
BR112017017037A2 (pt) 2018-04-10
KR20240010539A (ko) 2024-01-23
IL253894A0 (en) 2017-10-31
PL3256594T3 (pl) 2022-02-14
AU2022204583A1 (en) 2022-07-21
LT3256594T (lt) 2022-02-10
PT3256594T (pt) 2022-01-18
RS62811B1 (sr) 2022-02-28
EP4219728A3 (en) 2023-08-23
EA201791803A1 (ru) 2017-11-30

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