Advances in Gene Therapy – 2024 Update (Accredited)-Advances in Gene Therapy 2024 Updates

Video Transcription

Video Summary

In summary, the webinar focused on gene therapy updates for rare diseases. The speakers discussed advancements in gene therapy for conditions like sickle cell disease, Duchenne muscular dystrophy, X-linked adrenoleukodystrophy, and metachromatic leukodystrophy. They highlighted the use of AAV and lentiviral vectors for delivering therapeutic genes and the potential for genome editing with CRISPR-Cas9. The session also addressed ethical and economic considerations such as value-based agreements and outcomes-based payment models to manage the high cost of gene therapies. Overall, the speakers emphasized the transformative potential of gene therapy for rare diseases and the need for ongoing research and innovation in this field.

Keywords

gene therapy

rare diseases

advancements

sickle cell disease

Duchenne muscular dystrophy

X-linked adrenoleukodystrophy

metachromatic leukodystrophy

AAV vectors

lentiviral vectors

CRISPR-Cas9