Systemic deletion of DMD exon 51 rescues clinically severe Duchenne muscular dystrophy in a pig model lacking DMD exon 52

Stirm, M; Shashikadze, B; Blutke, A; Kemter, E; Lange, A; Stöckl, JB; Jaudas, F; Laane, L; Kurome, M; Kessler, B; Zakhartchenko, V; Bähr, A; Klymiuk, N; Nagashima, H; Walter, MC; Wurst, W; Kupatt, C; Fröhlich, T; Wolf, E

Wolf, E (通讯作者),Ludwig Maximilians Univ Munchen, Gene Ctr, Mol Anim Breeding & Biotechnol, D-81377 Munich, Germany.;Wolf, E (通讯作者),Ludwig Maximilians Univ Munchen, Dept Vet Sci, D-81377 Munich, Germany.;Wolf, E (通讯作者),Ludwig Maximilians Univ Munchen, Dept Vet Sci, Ctr Innovat Med Models, D-85764 Oberschleissheim, Germany.;Wolf, E (通讯作者),Ludwig Maximilians Univ Munchen, Gene Ctr, Lab Funct Genome Anal, D-81377 Munich, Germany.;Wolf, E (通讯作者),Ludwig Maximilians Univ Munchen, Interfac Ctr Endocrine & Cardiovasc Dis Network M, D-81377 Munich, Germany.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2023; 120 (29):

Abstract

Duchenne muscular dystrophy (DMD) is a fatal X-linked disease caused by mutations in the DMD gene, leading to complete absence of dystrophin and progr......

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