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 …

Sickle cell disease (SCD) is a monogenic disorder that afflicts approximately 100,000
Americans and millions of people worldwide. It is characterized by hemolytic anemia, vaso …

Neurocognitive impairment is common in sickle cell disease (SCD) and is associated with
significant functional limitations. In a cross‐sectional analysis, we examined the association …

Hydroxyurea is FDA‐approved and now increasingly used for children with sickle cell
anemia (SCA), but dosing strategies, pharmacokinetic (PK) profiles, and treatment …

CRISPR/Cas systems (clustered regularly interspaced short palindromic repeats) have
emerged as powerful tools to manipulate the genome for both research and therapeutic …

Clustered regularly interspaced short palindromic repeats (CRISPR)‐driven genome editing
has rapidly transformed preclinical biomedical research by eliminating the underlying …

Sickle cell disease (SCD) is one of the most common inherited hemoglobinopathy disorders
that affects millions of people worldwide. Reactivation of HBG (HBG1, HBG2) gene …

Fetal hemoglobin (HbF) can blunt the pathophysiology, temper the clinical course, and offer
prospects for curative therapy of sickle cell disease. This review focuses on (1) HbF …

Sickle cell disease (SCD) afflicts millions of people worldwide but is referred to as an orphan
disease in the United States. Over the past several decades, there has been an increasing …

Background: Sickle cell disease (SCD) complications lead to poor health-related quality of
life (HRQOL) and increased healthcare utilization in this population, which could be …