Targeting Duchenne muscular dystrophy by skipping DMD exon 45 with base editors

• Autori principali: Michael Gapinske (Autore), Jackson Winter (Autore), Devyani Swami (Autore), Lauren Gapinske (Autore), Wendy S. Woods (Autore), Shraddha Shirguppe (Autore), Angelo Miskalis (Autore), Anna Busza (Autore), Dana Joulani (Autore), Collin J. Kao (Autore), Kurt Kostan (Autore), Anne Bigot (Autore), Rashid Bashir (Autore), Pablo Perez-Pinera (Autore)
• Natura: Libro
• Pubblicazione: Elsevier, 2023-09-01T00:00:00Z.
• Soggetti: article
• Accesso online: Connect to this object online.

Duchenne muscular dystrophy is an X-linked monogenic disease caused by mutations in the dystrophin gene (DMD) characterized by progressive muscle weakness, leading to loss of ambulation and decreased life expectancy. Since the current standard of care for Duchenne muscular dystrophy is to merely tre...