Guiding Patients With Sickle Cell Disease Through the Transformative Era of Gene Therapy

The Rarest of All Diseases Are Becoming Treatable

For a decade after its discovery, CRISPR gene editing was stuck on the cusp of transforming medicine. Then, in 2023, scientists started using it on sickle-cell disease, and Victoria Gray, a  patient who lived with constant pain—like lightning inside her body, she has said—got the first-ever FDA-approved CRISPR gene-editing treatment. Her symptoms vanished; so did virtually everyone else’s in the clinical trial she was a part of. This year, the t…

Guiding Patients With Sickle Cell Disease Through the Transformative Era of Gene Therapy

Experts discuss the complexities of gene therapy for sickle cell disease, emphasizing patient support, psychosocial factors, and long-term care considerations.