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Deepa Manwani, ASH 2019: Gene Therapy for Beta-Hemoglobinopathies

Published Online: December 18th 2019

At the 61st ASH Annual Meeting & Exposition, Deepa Manwani discusses her presentation: Moving From Science Fiction to Clinical Reality: Gene Therapy for Beta-Hemoglobinopathies.

Questions

1. What is the rationale for gene therapy for beta-hemoglobinopathies and sickle cell disease?

2. What have been the most important recent advances in gene therapies for beta-hemoglobinopathies and sickle cell disease? (1:10)

3. What are the major challenges of translating clinical trial findings into real world use? (2:30)

4. What are the limitations of emerging strategies and how can outcomes be further improved? (3:21)

5. The high cost of gene therapy is a major barrier to its widespread application. How can this be overcome? (4:52)

 

Deepa Manwani has no conflicts of interest to declare in relation to this video.

Filmed at the 61st ASH Annual Meeting & Exposition 2019, Orlando, FL, USA.

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