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

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Official source

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

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Ontological neighbourhood

Biology

Genetics: Molecular genetics

Cell biology: Cancer

Health sciences

Immunology: Immune system

Biological engineering

Biomedical engineering: Genetic engineering

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