Lecture

Preclinical and Clinical Trials in SMA Gene Therapy

Description

This lecture covers preclinical and clinical trials in spinal muscular atrophy (SMA) gene therapy, discussing the demonstration of potency in mice, efficiency in patients, phase 1 trial results, further trials by Novartis, adverse effects, cellular targeting, and future perspectives in genome engineering and therapeutic strategies.

In MOOCs (6)

Instructor

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Official source

https://mediaspace.epfl.ch/media/0_hmp4s4i9

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Ontological neighbourhood

Biology

Genetics: Molecular genetics

Molecular biology: Topics in molecular biology

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