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© 2020 The Authors The AAV-based gene therapies used in DMD have the major limitation of maintaining the therapeutic effect over time. Forand et al. demonstrated an undeniable advantage of the PPMO and AAV combination on the striated muscles, notably on heart and diaphragm structure and function with an unrivalled extent of survival.

Original publication

DOI

10.1016/j.omtm.2020.03.011

Type

Journal article

Journal

Molecular Therapy - Methods and Clinical Development

Publication Date

12/06/2020

Volume

17

Pages

695 - 708