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Developing Gene Therapy Strategies to Treat Alpha Thalassemia

Kyle Cromer, PhD

Tuesday, August 23, 2022, 4:00-5:00 pm (San Francisco)  Check your time zone

Image of Kyle Cromer, PhD

In this session Dr. Cromer will:

  • Summarize a three-pronged approach to develop gene therapies to cure alpha thalassemia major (ATM) ex vivo
  • Describe one approach in depth − CRISPR/AAV6-mediated genome editing correction strategy
  • Discuss future directions that include the potential adaptation of successful ex vivo strategies for in utero and postnatal in vivo delivery