Gene Therapy for Hemoglobinopathies.
| Autor: | Lidonnici MR; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), San Raffaele Scientific Institute, Milan, Italy; and., Scaramuzza S; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), San Raffaele Scientific Institute, Milan, Italy; and., Ferrari G; San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), San Raffaele Scientific Institute, Milan, Italy; and.; University Vita-Salute San Raffaele, Milan, Italy. |
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| Jazyk: | angličtina |
| Zdroj: | Human gene therapy [Hum Gene Ther] 2023 Sep; Vol. 34 (17-18), pp. 793-807. |
| DOI: | 10.1089/hum.2023.138 |
| Abstrakt: | 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 hemoglobinopathies are fatal if untreated, and allogeneic bone marrow transplantation can be offered to a limited proportion of patients. The unmet clinical need and the disease incidence have promoted the development of new genetic therapies based on the engineering of autologous hematopoietic stem cells. Here, the steps of ex vivo gene therapy development are reviewed along with results from clinical trials and recent new approaches employing cutting edge gene editing tools. |
| Databáze: | MEDLINE |
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