Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing.

ABSTRACT: Hereditary anemia has various manifestations, such as sickle cell disease (SCD), Fanconi anemia, glucose-6-phosphate dehydrogenase deficiency (G6PDD), and thalassemia. The available management strategies for these disorders are still unsatisfactory and do not eliminate the main causes. As genetic aberrations are the main causes of all forms of hereditary anemia, the optimal approach involves repairing the defective gene, possibly through the transplantation of normal hematopoietic stem cells (HSCs) from a normal matching donor or through gene therapy approaches (either in vivo or ex vivo) to correct the patient's HSCs. To clearly illustrate the importance of cell and gene therapy in hereditary anemia, this paper provides a review of the genetic aberration, epidemiology, clinical features,

SUBMITTER: Anurogo D 

PROVIDER: S-EPMC8230702 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature