Micro-dystrophin gene constructs for repairing heart and muscle function in rats: the smaller is enough?

Affiliations

¹ Ludwig Boltzmann Institute for Cardiovascular Research at the Center for Biomedical Research, Medical University of Vienna, Vienna, Austria.
² Ludwig Boltzmann Institute for Cardiovascular Research at the Center for Biomedical Research, Medical University of Vienna, Vienna, Austria. attila.kiss@meduniwien.ac.at.

Comment

Petra Lujza Szabo et al. Gene Ther. 2022 Sep.

. 2022 Sep;29(9):477-478.

doi: 10.1038/s41434-022-00337-2. Epub 2022 Apr 22.

¹ Ludwig Boltzmann Institute for Cardiovascular Research at the Center for Biomedical Research, Medical University of Vienna, Vienna, Austria.
² Ludwig Boltzmann Institute for Cardiovascular Research at the Center for Biomedical Research, Medical University of Vienna, Vienna, Austria. attila.kiss@meduniwien.ac.at.

No abstract available

Comment on

Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMD^mdx rat model.
Bourdon A, François V, Zhang L, Lafoux A, Fraysse B, Toumaniantz G, Larcher T, Girard T, Ledevin M, Lebreton C, Hivonnait A, Creismeas A, Allais M, Marie B, Guguin J, Blouin V, Remy S, Anegon I, Huchet C, Malerba A, Kao B, Le Heron A, Moullier P, Dickson G, Popplewell L, Adjali O, Montanaro F, Le Guiner C. Bourdon A, et al. Gene Ther. 2022 Sep;29(9):520-535. doi: 10.1038/s41434-022-00317-6. Epub 2022 Feb 1. Gene Ther. 2022. PMID: 35105949

1. Duan D, Goemans N, Takeda S, Mercuri E, Aartsma-Rus A. Duchenne muscular dystrophy. Nat Rev Dis Primers. 2021;7:13. - DOI
1. Muntoni F. Cardiomyopathy in muscular dystrophies. Curr Opin Neurol. 2003;16:577–83. - DOI
1. Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol. 2010;9:77–93. - DOI
1. Goemans N, Buyse G. Current treatment and management of dystrophinopathies. Curr Treat Options Neurol. 2014;16:287. - DOI
1. Chamberlain JR, Chamberlain JS. Progress toward gene therapy for Duchenne muscular dystrophy. Mol Ther. 2017;25:1125–31. - DOI