Executive summary All over the world, people with sickle cell disease (an inherited
condition) have premature deaths and preventable severe chronic complications, which …

Sickle cell disease (SCD), a distinctive and often overlooked illness in the 21st century, is a
congenital blood disorder characterized by considerable phenotypic diversity. It comprises a …

The African continent is poised to have a pivotal role in the global population landscape,
with the United Nations projecting a population of 2.5 billion (more than 25% of the global …

Somatic gene therapy will be one of the most exciting practices of genetic medicine in Africa
and is primed to offer a “new life” for persons living with sickle cell disease (SCD). Recently …

Sickle cell disease (SCD) is an inherited, progressively debilitating blood disorder.
Emerging gene therapies (GTx) may lead to a complete remission, the benefits of such can …

Sickle cell disease (SCD) is a prevalent, life-threatening condition attributable to a heritable
mutation in β-hemoglobin. Therapeutic induction of fetal hemoglobin (HbF) can ameliorate …

In recent years, a growing number of clinical trials have been initiated to evaluate gene
therapy approaches for the treatment of patients with transfusion-dependent β-thalassemia …

Targeted delivery and cell-type-specific expression of gene-editing proteins in various cell
types in vivo represent major challenges for all viral and non-viral delivery platforms …

Children with sickle cell anemia (SCA) living in Nigeria are at an increased risk of
malnutrition, which contributes to increased morbidity and mortality. However, evidence …

Sickle cell disease is a hereditary multiorgan disease that is considered rare in the EU. In
2017, the Rare Diseases Plan was implemented within the EU and 24 European Reference …