TW202423956A - 用於治療肌肉失養症之重組ａａｖ載體 - Google Patents

用於治療肌肉失養症之重組ａａｖ載體 Download PDF

Info

Publication number
TW202423956A
TW202423956A TW112136446A TW112136446A TW202423956A TW 202423956 A TW202423956 A TW 202423956A TW 112136446 A TW112136446 A TW 112136446A TW 112136446 A TW112136446 A TW 112136446A TW 202423956 A TW202423956 A TW 202423956A
Authority
TW
Taiwan
Prior art keywords
muscular dystrophy
aav vectors
recombinant aav
raav
present disclosure
Prior art date
Application number
TW112136446A
Other languages
English (en)
Inventor
克拉帕克 路易斯 羅帝諾
伊達 Ｈ 莫勒爾
史黛芬妮 Ｅ 梅森
Original Assignee
美商薩羅塔治療公司
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 美商薩羅塔治療公司 filed Critical 美商薩羅塔治療公司
Publication of TW202423956A publication Critical patent/TW202423956A/zh

Links

Abstract

本發明提供用於表現人類微肌肉萎縮蛋白(micro-dystrophin)基因之基因療法載體，諸如重組腺相關病毒(rAAV)。本發明亦提供使用此等rAAV治療肌肉失養症(諸如杜興氏肌肉失養症)之組合物及方法。本發明亦提供對個體之 <i>DMD</i>基因進行基因分型以確定是否應禁忌rAAV基因療法。
TW112136446A 2022-09-23 2023-09-23 用於治療肌肉失養症之重組ａａｖ載體 TW202423956A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US63/376,840 2022-09-23
US63/487,217 2023-02-27
US63/509,221 2023-06-20
US63/518,055 2023-08-07
WOPCT/US23/74934 2023-09-22

Publications (1)

Publication Number Publication Date
TW202423956A true TW202423956A (zh) 2024-06-16

Family

ID=

Similar Documents

Publication Publication Date Title
PH12021551341A1 (en) Compositions for drg-specific reduction of transgene expression
MX2020008933A (es) Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos.
TN2020000187A1 (en) Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism
WO2019169004A8 (en) Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
NZ763018A (en) Adeno-associated virus variant capsids and methods of use thereof
EP4272728A3 (en) Adeno-associated virus variant capsids and use for inhibiting angiogenesis
WO2001016330A3 (en) Prevention of affections associated with porcine circovirus-2
WO2005033321A3 (en) Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
WO2020223362A8 (en) Compositions useful for treatment of pompe disease
RU2018136611A (ru) Генная терапия для лечения гемофилии a
PH12020500239A1 (en) Factor viii (fviii) gene therapy methods
MX2022009883A (es) Vectores de genoterapia para tratar enfermedades cardíacas.
MX2021010134A (es) Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn.
MX2021013420A (es) Composiciones utiles en el tratamiento de leucodistrofia metacromatica.
WO2020219766A8 (en) Compositions useful in treatment of rett syndrome
WO2021163357A3 (en) Gene therapy vectors for treating heart disease
CA2315277A1 (en) Pharmaceutical compositions containing the long pentraxin ptx3
MX2022011777A (es) Itrs libres de cpg para la terapia genica aav.
WO2023169115A9 (zh) 一种神经系统高亲和性的aav载体及其应用
WO2002063025A8 (en) Muscle-directed gene therapy with aav-1 and aav-6 vectors
TW202423956A (zh) 用於治療肌肉失養症之重組ａａｖ載體
MX2022016332A (es) Administracion de vectores de virus adenoasociados para distrofias musculares.
MX2022004799A (es) Composiciones y metodos para el tratamiento de los trastornos de almacenamiento de glucogeno.
PH12020551319A1 (en) Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies
MX2022002132A (es) Administracion de vectores de virus adenoasociado de alfa-sarcoglicano y el tratamiento de la distrofia muscular.