Researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center and the University of Massachusetts Medical School have developed a strategy to treat two of the most common inherited blood diseases, sickle cell disease and beta thalassemia, applying CRISPR-Cas9 gene editing to patients' own blood stem cells.
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The research will focus on adapting gene therapy for use in low-resource areas with high rates of the disease.
A patient with a severe form of sickle cell disease is symptom-free after gene therapy knocks down BCL11A and restores fetal hemoglobin production.
Alessandra Biffi, MD, led a study that shows the efficacy of a new gene therapy transplantation technique that could have future applications for treating neurodegenerative diseases.
David Williams, MD, discusses Dana-Farber/Boston Children's new gene therapy clinical trial for sickle cell disease in a live webchat.