DNAJB6 isoform specific knockdown: Therapeutic potential for limb girdle muscular dystrophy D1

DNAJB6 isoform specific knockdown: Therapeutic potential for limb girdle muscular dystrophy D1

Dominant missense mutations in DNAJB6, a co-chaperone of HSP70, cause limb girdle muscular dystrophy (LGMD) D1. No treatments are currently available. Two isoforms exist, DNAJB6a and DNAJB6b, each with distinct localizations in muscle. Mutations reside in both isoforms, yet evidence suggests that DN...

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Bibliographic Details
Main Authors: Andrew R. Findlay, May M. Paing, Jil A. Daw, Meade Haller, Rocio Bengoechea, Sara K. Pittman, Shan Li, Feng Wang, Timothy M. Miller, Heather L. True, Tsui-Fen Chou, Conrad C. Weihl
Format: Article
Language:English
Published: Elsevier 2023-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
limb girdle muscular dystrophy D1 (LGMDD1)
DNAJB6
isoform specific knockdown
morpholino
proteomics
Online Access:http://www.sciencedirect.com/science/article/pii/S216225312300135X

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http://www.sciencedirect.com/science/article/pii/S216225312300135X

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