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MX2020003042A - Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. - Google Patents

Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.

Info

Publication number
MX2020003042A
MX2020003042A MX2020003042A MX2020003042A MX2020003042A MX 2020003042 A MX2020003042 A MX 2020003042A MX 2020003042 A MX2020003042 A MX 2020003042A MX 2020003042 A MX2020003042 A MX 2020003042A MX 2020003042 A MX2020003042 A MX 2020003042A
Authority
MX
Mexico
Prior art keywords
viral genome
capsid
friedreich
ataxia
rescue
Prior art date
Application number
MX2020003042A
Other languages
English (en)
Inventor
Dinah Wen-Yee Sah
Yanqun Shu
Jinzhao Hou
Martin Goulet
Holger Patzke
Hélène Puccio
Original Assignee
Voyager 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 Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of MX2020003042A publication Critical patent/MX2020003042A/es

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Abstract

En el presente documento se proporciona una particula de virus adenoasociados (AAV) que comprende una capside y un genoma viral, en donde dicha capside suministra la particula de AAV a un sistema nervioso, y en donde dicho genoma viral comprende una secuencia de polinucleotidos que codifica Frataxina y uno o mas sitios de union de microARN. Ademas, en este documento se proporciona un metodo para tratar, mejorar y/o prevenir un trastorno en un sujeto derivado de una perdida o perdida parcial de proteina Frataxina en el sujeto, en donde el método comprende: administrar al sujeto una cantidad terapeuticamente efectiva de composición que comprende una particula de AAV que comprende una capside y un genoma viral, en donde dicha capside administra el genoma viral a un sistema nervioso, y en donde dicho genoma viral comprende una secuencia de polinucleotidos que codifica Frataxina y uno o mas sitios de union a microARN, como se describe en el presente documento.
MX2020003042A 2017-09-29 2018-09-28 Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. MX2020003042A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762565840P 2017-09-29 2017-09-29
US201862663835P 2018-04-27 2018-04-27
PCT/US2018/053312 WO2019067840A1 (en) 2017-09-29 2018-09-28 RESTRICTING CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE FROM FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION

Publications (1)

Publication Number Publication Date
MX2020003042A true MX2020003042A (es) 2020-11-18

Family

ID=65903225

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MX2020003042A MX2020003042A (es) 2017-09-29 2018-09-28 Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.

Country Status (6)

Country Link
US (2) US20200263199A1 (es)
EP (1) EP3687582A4 (es)
AU (1) AU2018338728B2 (es)
CA (1) CA3075656A1 (es)
MX (1) MX2020003042A (es)
WO (1) WO2019067840A1 (es)

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US20210230632A1 (en) * 2018-05-15 2021-07-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
EP3883954A4 (en) * 2018-11-21 2022-08-10 Stridebio, Inc. Recombinant viral vectors and nucleic acids for producing the same
KR20220116459A (ko) * 2019-11-22 2022-08-23 더 칠드런스 호스피탈 오브 필라델피아 아데노(adeno)-관련 바이러스 벡터 변이체
WO2024220722A1 (en) * 2023-04-18 2024-10-24 Cornell University Compositions and methods for tissue- specific expression of gene therapies
WO2026017965A1 (en) * 2024-07-19 2026-01-22 The University Court Of The University Of Edinburgh Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions

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