MX2021004294A - Suministro mejorado de particulas virales al cuerpo estriado y al cortex. - Google Patents

Suministro mejorado de particulas virales al cuerpo estriado y al cortex.

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Publication number
MX2021004294A
MX2021004294A MX2021004294A MX2021004294A MX2021004294A MX 2021004294 A MX2021004294 A MX 2021004294A MX 2021004294 A MX2021004294 A MX 2021004294A MX 2021004294 A MX2021004294 A MX 2021004294A MX 2021004294 A MX2021004294 A MX 2021004294A
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MX
Mexico
Prior art keywords
striatum
cortex
viral particles
enhanced delivery
mammal
Prior art date
Application number
MX2021004294A
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English (en)
Inventor
Lisa M Stanek
Lamya Shihabuddin
Original Assignee
Genzyme Corp
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Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of MX2021004294A publication Critical patent/MX2021004294A/es

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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
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    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
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    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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Abstract

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 nucleico heterólogo al cuerpo estriado y causar la expresión del ácido nucleico heterólogo al menos en el córtex cerebral y en el cuerpo estriado del mamífero.
MX2021004294A 2015-02-10 2017-08-10 Suministro mejorado de particulas virales al cuerpo estriado y al cortex. MX2021004294A (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)

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MX2021004294A true MX2021004294A (es) 2021-06-04

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MX2017010370A MX2017010370A (es) 2015-02-10 2016-02-09 Suministro mejorado de particulas virales al cuerpo estriado y al cortex.
MX2021004294A MX2021004294A (es) 2015-02-10 2017-08-10 Suministro mejorado de particulas virales al cuerpo estriado y al cortex.

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MX2017010370A MX2017010370A (es) 2015-02-10 2016-02-09 Suministro mejorado de particulas virales al cuerpo estriado y al cortex.

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US (2) US20190111157A1 (es)
EP (3) EP4023758A1 (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) IL296466A (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) SG10201907399RA (es)
SI (1) SI3256594T1 (es)
TN (1) TN2017000353A1 (es)
TW (1) TWI730952B (es)
WO (1) WO2016130591A2 (es)

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EP4219728A3 (en) 2023-08-23
RS62811B1 (sr) 2022-02-28
CN116019934A (zh) 2023-04-28
TN2017000353A1 (en) 2019-01-16
SG11201706445SA (en) 2017-09-28
CR20170407A (es) 2017-11-14
KR20240010539A (ko) 2024-01-23
JP2021178861A (ja) 2021-11-18
PT3256594T (pt) 2022-01-18
SG10201907399RA (en) 2019-09-27
NZ735290A (en) 2023-10-27
TW201642909A (zh) 2016-12-16
KR102625784B1 (ko) 2024-01-15
HUE057272T2 (hu) 2022-04-28
US20190111157A1 (en) 2019-04-18
WO2016130591A2 (en) 2016-08-18
LT3256594T (lt) 2022-02-10
CN107530447A (zh) 2018-01-02
ES2904504T3 (es) 2022-04-05
SG10201912926VA (en) 2020-02-27
WO2016130591A3 (en) 2016-11-10
AU2016219398A1 (en) 2017-09-28
US20220395586A1 (en) 2022-12-15
PL3256594T3 (pl) 2022-02-14
EP4219728A2 (en) 2023-08-02
TWI730952B (zh) 2021-06-21
DK3256594T3 (da) 2022-01-24
JP2023058630A (ja) 2023-04-25
IL296466A (en) 2022-11-01
IL253894B (en) 2021-08-31
AU2022204583A1 (en) 2022-07-21
PH12017501436A1 (en) 2018-01-15
JP2018506585A (ja) 2018-03-08
HK1246340A1 (zh) 2018-09-07
IL285060B2 (en) 2023-02-01
CL2017002028A1 (es) 2018-04-13
EP4023758A1 (en) 2022-07-06
SI3256594T1 (sl) 2022-07-29
EP3256594B1 (en) 2021-10-20
EP3256594A2 (en) 2017-12-20
IL253894A0 (en) 2017-10-31
JP6928558B2 (ja) 2021-09-01
EA201791803A1 (ru) 2017-11-30
IL285060B (en) 2022-10-01
IL285060A (en) 2021-08-31
CA2976082A1 (en) 2016-08-18
KR20170110718A (ko) 2017-10-11
BR112017017037A2 (pt) 2018-04-10
MX2017010370A (es) 2017-12-14

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