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Novel Gene Therapy Trial for Sickle Cell Disease Launches UCSF Benioff Children’s Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease The trial is the first in the U S to apply non-viral CRISPR-Cas9 gene-editing technology in humans to directly correct the genetic mutation that causes the disease
First CRISPR Therapy Approved for Sickle Cell In the last five years, numerous new disease-modifying drugs have been approved to reduce the symptoms of sickle cell disease, and more gene therapies are being tested in clinical trials, showing good results so far
Gene therapy for sickle cell disease: recent advances, clinical trials . . . Gene therapy, by addressing the root genetic causes, offers a revolutionary alternative This article discusses the molecular mechanisms of SCD and β-thalassemia and highlights advancements in gene therapy, such as gene addition via lentiviral vectors and gene editing with CRISPR Cas9 technology
New sickle cell treatment approved In an historic moment for the sickle cell community, we are delighted that the MHRA (The Medicines and Healthcare products Regulatory Agency) approved the gene therapy known as Casgevy (Exagamglogene autotemcel) for the treatment of sickle cell disorder