The basics of genetic approaches for NMDs – using DMD as a paradigm - Annemieke Aartsma-Rus

This webinar was be delivered by Annemieke Aartsma-Rus, who is a Professor of Translational Genetics, Department of Human Genetics, Leiden University Medical Center in the Netherlands.

Annemeike briefly introduces the basic concepts of different genetic therapies (gene addition, genome editing, splicing modulation and transcript readthrough). Duchenne is used as a paradigm since all these approaches are in development. However, the webinar also takes into account challenges and considerations for applying these approaches broader to other NMDs.