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This review will summarize the advent and clinical translation of ex vivo gene therapy, with the focus on the milestones during the exploitation of genetically engineered hematopoietic stem cells (HSCs) tackling a variety of pathological conditions which led to marketing approval.
- Precision oncology using ex vivo technology: a step towards ...
For example, in T-cell prolymphocytic leukaemia, in a small...
- Precision oncology using ex vivo technology: a step towards ...
Ex vivo gene therapy occurs when cells are removed from a patient and modified outside the body, in a lab, using specialized approaches like adding a new gene to the cell or fixing a gene in a cell that is causing a disease.
Read about the different techniques used in gene therapy, and how genetic material is delivered to cells via ex vivo and in vivo methods.
31 Ιουλ 2019 · An example of ex vivo delivery of gene therapy is the treatment of β-thalassemia, involving gene transfer to hematopoietic stem and progenitor cells (HSPCs).
16 Νοε 2020 · For example, the success of in vivo AAV gene transfer to the human retina and central nervous system by Luxturna and Zolgensma for Leber’s congenital amaurosis and spinal muscular atrophy,...
26 Οκτ 2021 · One early-phase clinical trial employed an ex vivo gene-editing method to treat people with sickle cell disease or with a related blood disorder called beta thalassemia.
3 Οκτ 2022 · For example, in T-cell prolymphocytic leukaemia, in a small number of patients, ex vivo screening identified novel therapies, and outperformed molecular characterisation alone in correlating with clinical response to treatment (Ref. 35). The success of ex vivo-based techniques used to direct patient treatment in haematological cancers enabling ...
