Lecture

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

Lorem voluptate

Adipisicing anim excepteur cillum minim deserunt in et. Officia cupidatat cupidatat commodo consequat velit tempor non. Ad aliquip ex tempor duis.

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

Biology

Genetics: Molecular genetics

Cell biology: Cancer

Health sciences

Immunology: Immune system

Biological engineering

Biomedical engineering: Genetic engineering

Related lectures (38)

Graph Chatbot

Chat with Graph Search

Ask any question about EPFL courses, lectures, exercises, research, news, etc. or try the example questions below.

DISCLAIMER: The Graph Chatbot is not programmed to provide explicit or categorical answers to your questions. Rather, it transforms your questions into API requests that are distributed across the various IT services officially administered by EPFL. Its purpose is solely to collect and recommend relevant references to content that you can explore to help you answer your questions.