CRISPR and Vertex’s Potential Cure for Sickle Cell Disease and More Glimmers of Hope

ASH 2020: CRISPR and Vertex’s Potential Cure for Sickle Cell Disease and More Glimmers of Hope
Published: Dec. 7, 2020
BioSpace
CRISPR Therapeutics and Vertex Pharmaceuticals presented new data on 10 patients treated with CTX001, which demonstrated a consistent and sustained response, in transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD). CTX001 is an investigational CRISPR/Cas9-based gene-editing therapy.

Read the source article at pharmalive.com

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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