For over 100 years, clinicians and scientists have been unravelling the consequences of the
A to T substitution in the β-globin gene that produces haemoglobin S, which leads to the …

The discovery of the molecular basis of sickle cell disease was an important landmark in
molecular medicine. The modern tools of molecular and cellular biology have refined our …

The β-haemoglobinopathies are the most prevalent inherited disorders worldwide. Gene
therapy of β-thalassaemia is particularly challenging given the requirement for massive …

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 …

The capacity to efficiently transduce nondividing cells, shuttle large genetic payloads, and
maintain stable long-term transgene expression are attributes that have brought lentiviral …

Abstract CRISPR/Cas9-mediated beta-globin (HBB) gene correction of sickle cell disease
(SCD) patient-derived hematopoietic stem cells (HSCs) in combination with autologous …

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 …

Abstract β-thalassemias (β-thal) are a group of blood disorders caused by mutations in the β-
globin gene (HBB) cluster. β-globin associates with α-globin to form adult hemoglobin (HbA …

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 …

Gene transfer into autologous hematopoietic stem progenitor cells (HSPCs) has the
potential to cure monogenic inherited disorders caused by an altered development and/or …