Pilot Feasibility Study of BCL11A as Gene Therapy Target in Sickle Cell Disease

December 7-10, 2019; Orlando, Florida
Preliminary results from pilot feasibility study suggest that a gene therapy construct targeting BCL11A is safe, effectively induces HbF expression, and improves sickling signs and symptoms when delivered to patients with severe SCD.
Format: Microsoft PowerPoint (.ppt)
File Size: 192 KB
Released: December 13, 2019

Acknowledgements

Provided by the Annenberg Center for Health Sciences at Eisenhower in partnership with Clinical Care Options.
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Annenberg Center for Health Sciences at Eisenhower
39000 Bob Hope Dr
Dinah Shore Bldg.
Rancho Mirage, CA 92270

Melissa Velasquez, Accreditation Specialist
(760) 773-4506
(760) 773-4550 (Fax)
ce@annenberg.net
http://www.annenberg.net/

Supported by educational grants from
AstraZeneca
Celgene Corporation
Dova Pharmaceuticals, Inc.
Karyopharm Therapeutics
Loxo Oncology, Inc. a wholly owned subsidiary of Eli Lilly and Company
Pharmacyclics LLC, an AbbVie Company
Janssen Biotech, Inc., administered by Janssen Scientific Affairs, LLC

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