Alternative Strategies for SMA Gene Therapy

Description

This lecture discusses alternative strategies for spinal muscular atrophy (SMA) gene therapy, including the use of antisense oligonucleotides to inhibit exon 8 splicing, enhancing exon 8 inclusion to improve exon 7 levels, and exploring genome editing through homology-directed repair of the SMN2 gene. The potential of non-homologous repair and the limitations of the CRISPR-Cas9 system in SMA gene therapy are also addressed.

In MOOCs (6)

Instructor

deserunt laboris anim esse

Eu id occaecat Lorem esse Lorem pariatur aliqua nisi laboris tempor incididunt anim consequat. Qui veniam aliqua ullamco voluptate sit. Voluptate non nisi qui quis irure proident enim. Velit occaecat ea nulla adipisicing labore nisi ad. Proident cillum nulla sint exercitation velit quis do et nisi.

Official source

https://mediaspace.epfl.ch/media/0_7ex36mbc

About this result

This page is automatically generated and may contain information that is not correct, complete, up-to-date, or relevant to your search query. The same applies to every other page on this website. Please make sure to verify the information with EPFL's official sources.

Ontological neighbourhood

Related lectures (38)