|
|||||
|
|
Molecular Therapies for Myotonic Dystrophy Type 1: From Small Drugs to Gene Editing |
|
| Authors: | Mariapaola Izzo Jonathan Battistini Claudia Provenzano Fabio Martelli Beatrice Cardinali Germana Falcone |
| Affiliation: | 1.Institute of Biochemistry and Cell Biology, National Research Council, Monterotondo, 00015 Rome, Italy; (M.I.); (J.B.); (C.P.);2.Molecular Cardiology Laboratory, IRCCS Policlinico San Donato, San Donato Milanese, 20097 Milan, Italy; |
| Abstract: | |
| Keywords: | myotonic dystrophy trinucleotide-expansion disease DM1 mice antisense oligonucleotides molecular therapy gene editing |