In utero Therapy for the Treatment of Sickle Cell Disease: Taking Advantage of the Fetal Immune System.

ABSTRACT: Sickle Cell Disease (SCD) is an autosomal recessive disorder resulting from a β-globin gene missense mutation and is among the most prevalent severe monogenic disorders worldwide. Haematopoietic stem cell transplantation remains the only curative option for the disease, as most management options focus solely on symptom control. Progress in prenatal diagnosis and fetal therapeutic intervention raises the possibility of in utero treatment. SCD can be diagnosed prenatally in high-risk patients using chorionic villus sampling. Among the possible prenatal treatments, in utero stem cell transplantation (IUSCT) shows the most promise. IUSCT is a non-myeloablative, non-immunosuppressive alternative conferring various unique advantages and may also offer safer postnatal management. F

SUBMITTER: Cortabarria ASV 

PROVIDER: S-EPMC7862553 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature