The landscape of sickle cell disease (SCD) treatment continues to evolve rapidly, with new
disease-modifying therapies in development and potentially curative options on the horizon …

Gene therapy as a potential cure for sickle cell disease (SCD) has long been pursued, given
that this hemoglobin (Hb) disorder results from a single point mutation. Advances in genomic …

Sickle cell disease (SCD) is potentially curable after allogeneic hematopoietic stem cell
transplantation (HSCT) or autologous HSCT after ex vivo genetic modification. Autologous …

Sickle cell disease afflicts millions of people worldwide and approximately 100,000
Americans. Complications are myriad and arise as a result of complex pathological …

Sickle cell disease (SCD) is a common genetic blood disorder associated with acute and
chronic pain, progressive multiorgan damage, and early mortality. Recent advances in …

Sickle cell disease (SCD) is a monogenic red cell disorder affecting more than 300 000
annual births worldwide and leading to significant organ toxicity and premature mortality …

Recent advances in managing Sickle Cell Disease (SCD) significantly improved patient
survival and quality of life. Disease-modifying drug therapies such as hydroxyurea, L …

Background Gene therapy is an emerging treatment for sickle cell disease that works by
replacing a defective gene with a healthy gene, allowing the body to produce normal red …

A growing number of gene therapy‐and gene editing‐based treatments for patients with
sickle cell disease (SCD) are entering clinical trials. These treatments, designed to target the …

Sickle cell disease (SCD) results from a single base pair change in the sixth codon of the β-
globin chain of hemoglobin, which promotes aggregation of deoxyhemoglobin, increasing …