Sickle cell disease (SCD) is one of the most common life-threatening monogenic diseases
affecting millions of people worldwide. Allogenic hematopietic stem cell transplantation is …

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 the most common monogenic blood disorder marked by severe
pain, end-organ damage, and early mortality. Treatment options for SCD remain very limited …

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 …

Sickle cell disease (SCD) is a monogenic disease caused by a nucleotide mutation in the β-
globin gene. Current gene therapy studies are mainly focused on lentiviral vector–mediated …

Effective medical management for sickle cell disease (SCD) remains elusive. As a prevalent
and severe monogenic disorder, SCD has been long considered a logical candidate for …

Sickle cell disease (SCD) is the most common serious monogenic disease with 300,000
births annually worldwide. SCD is an autosomal recessive disease resulting from a single …

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 …

Autologous hematopoietic stem cell (HSC)-targeted gene therapy provides a one-time cure
for various genetic diseases including sickle cell disease (SCD) and β-thalassemia. SCD is …

Sickle cell disease (SCD) is a monogenic disorder that affects millions worldwide.
Allogeneic hematopoietic stem cell transplantation is the only available cure. Here, we …