Hematopoietic stem cell gene-addition/editing therapy in sickle cell disease
P Germino-Watnick, M Hinds, A Le, R Chu, X Liu… - Cells, 2022 - mdpi.com
Autologous hematopoietic stem cell (HSC)-targeted gene therapy provides a one-time cure
for various genetic diseases including sickle cell disease (SCD) and β-thalassemia. SCD is …
for various genetic diseases including sickle cell disease (SCD) and β-thalassemia. SCD is …
Hematopoietic stem cell-targeted gene-addition and gene-editing strategies for β-hemoglobinopathies
CM Drysdale, T Nassehi, J Gamer, M Yapundich… - Cell Stem Cell, 2021 - cell.com
Sickle cell disease (SCD) is caused by a well-defined point mutation in the β-globin gene
and therefore is an optimal target for hematopoietic stem cell (HSC) gene-addition/editing …
and therefore is an optimal target for hematopoietic stem cell (HSC) gene-addition/editing …
Gene therapy for hemoglobinopathies
MR Lidonnici, S Scaramuzza, G Ferrari - Human Gene Therapy, 2023 - liebertpub.com
β-Thalassemia and sickle cell disease are autosomal recessive disorders of red blood cells
due to mutations in the adult β-globin gene, with a worldwide diffusion. The severe forms of …
due to mutations in the adult β-globin gene, with a worldwide diffusion. The severe forms of …