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.
