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MX2020003965A - Terapias genicas para los trastornos lisosomales. - Google Patents

Terapias genicas para los trastornos lisosomales.

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Publication number
MX2020003965A
MX2020003965A MX2020003965A MX2020003965A MX2020003965A MX 2020003965 A MX2020003965 A MX 2020003965A MX 2020003965 A MX2020003965 A MX 2020003965A MX 2020003965 A MX2020003965 A MX 2020003965A MX 2020003965 A MX2020003965 A MX 2020003965A
Authority
MX
Mexico
Prior art keywords
disease
gene therapies
lysosomal disorders
expression constructs
disclosure provides
Prior art date
Application number
MX2020003965A
Other languages
English (en)
Inventor
Asa Abeliovich
Laura Heckman
Herve Rhinn
Original Assignee
Prevail Therapeutics Inc
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 Prevail Therapeutics Inc filed Critical Prevail Therapeutics Inc
Publication of MX2020003965A publication Critical patent/MX2020003965A/es

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Abstract

La divulgación se refiere, en algunos aspectos, a composiciones y métodos para el tratamiento de las enfermedades asociadas con una función lisosomal aberrante, por ejemplo, la enfermedad de Parkinson (PD) y la enfermedad de Gaucher. En algunas modalidades, la divulgación proporciona las construcciones de expresión que comprenden un transgén que codifica la beta-glucocerebrosidasa (GBA) o una porción de la misma, solo o en combinación con uno o más genes asociados con la PD. En algunas modalidades, la divulgación proporciona métodos para el tratamiento de la enfermedad de Parkinson por medio de la administración de las construcciones de expresión a un sujeto que lo necesite.
MX2020003965A 2017-10-03 2018-10-03 Terapias genicas para los trastornos lisosomales. MX2020003965A (es)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201762567296P 2017-10-03 2017-10-03
US201762567301P 2017-10-03 2017-10-03
US201762567310P 2017-10-03 2017-10-03
US201762567319P 2017-10-03 2017-10-03
US201762567311P 2017-10-03 2017-10-03
PCT/US2018/054227 WO2019070894A1 (en) 2017-10-03 2018-10-03 GENE THERAPIES FOR LYSOSOMIAL DISORDERS

Publications (1)

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MX2020003965A true MX2020003965A (es) 2020-10-05

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