MX2021012184A - Terapias genicas para trastornos lisosomales. - Google Patents

Terapias genicas para trastornos lisosomales.

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Publication number
MX2021012184A
MX2021012184A MX2021012184A MX2021012184A MX2021012184A MX 2021012184 A MX2021012184 A MX 2021012184A MX 2021012184 A MX2021012184 A MX 2021012184A MX 2021012184 A MX2021012184 A MX 2021012184A MX 2021012184 A MX2021012184 A MX 2021012184A
Authority
MX
Mexico
Prior art keywords
disease
parkinson
gene therapies
expression constructs
lysosomal disorders
Prior art date
Application number
MX2021012184A
Other languages
English (en)
Inventor
Asa Abeliovich
Laura Heckman
Herve Rhinn
Franz Hefti
Original Assignee
Prevail Therapeutics Inc
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Filing date
Publication date
Application filed by Prevail Therapeutics Inc filed Critical Prevail Therapeutics Inc
Publication of MX2021012184A publication Critical patent/MX2021012184A/es

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    • 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
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Abstract

La descripción se refiere a composiciones y métodos para el tratamiento de enfermedades asociadas con una función lisosomal anómala, tales como la enfermedad de Parkinson y la enfermedad de Gaucher. La descripción proporciona construcciones de expresión que comprenden un transgén que codifica beta-glucocerebrosidasa, un ARN inhibidor que se dirige a alfa-sinucleína, o una combinación de los anteriores. La descripción proporciona, además, métodos para tratar la enfermedad de Gaucher, la enfermedad de Parkinson u otras sinucleinopatías mediante la administración de dichas construcciones de expresión a un sujeto que lo necesita.
MX2021012184A 2019-04-10 2020-04-10 Terapias genicas para trastornos lisosomales. MX2021012184A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962831840P 2019-04-10 2019-04-10
US202062990246P 2020-03-16 2020-03-16
PCT/US2020/027788 WO2020210713A1 (en) 2019-04-10 2020-04-10 Gene therapies for lysosomal disorders

Publications (1)

Publication Number Publication Date
MX2021012184A true MX2021012184A (es) 2022-01-24

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Application Number Title Priority Date Filing Date
MX2021012184A MX2021012184A (es) 2019-04-10 2020-04-10 Terapias genicas para trastornos lisosomales.

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US (2) US11903985B2 (es)
EP (1) EP3953378A1 (es)
JP (1) JP2022526021A (es)
KR (1) KR20220006527A (es)
CN (1) CN114026115A (es)
AU (1) AU2020270984A1 (es)
BR (1) BR112021019880A2 (es)
CA (1) CA3136117A1 (es)
IL (1) IL286901A (es)
MX (1) MX2021012184A (es)
WO (1) WO2020210713A1 (es)

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US20200338148A1 (en) 2020-10-29
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