A novel gene therapy that accurately “edits” DNA could potentially offer a revolutionary treatment for sickle cell disease, an inherited and often painful condition affecting primarily Black children and adults, according to a new study published in the New England Journal of Medicine. Early results from trials with three severely ill adults are reported to be promising, demonstrating a reduced frequency of pain attacks and other symptoms within 6 to 18 months post-gene therapy treatment. This innovative treatment, requiring a single but intensive application, adds to a growing body of evidence indicating that gene therapy could be a viable curative approach for sickle cell patients.
The gene therapy process involves the removal of a patient’s bone marrow stem cells, which then undergo genetic modification in a lab using the cutting-edge CRISPR-Cas9 technique, allowing precise DNA alterations. Following this, patients receive chemotherapy to obliterate the remaining faulty stem cells, allowing for the injection of the genetically enhanced cells. One such therapy, named exa-cel, which utilizes this CRISPR mechanism, is currently being evaluated for FDA approval. The key advantage of gene therapy over conventional stem cell transplantations is the elimination of donor requirements and hence, reduced risk associated with graft-versus-host disease and transplant rejection. However, significant questions remain regarding gene therapy, particularly concerning its long-term efficacy and safety.
Read more: https://lnkd.in/e95AVaJt