학술논문

scAAV9.U7-ACCA treatment of DMD exon 2 duplication leads to significant dystrophin expression and evidence of clinical benefit, particularly following treatment as an infant
Document Type
Journal
Source
NEUROMUSCULAR DISORDERS; OCT 2023, 33 pS217-pS217, 1p. Supplement: SUPP1
Subject
Language
English
ISSN
18732364