Cancer Gene Therapy Research Center (CGRC)- Gene Therapy Application
Gene Therapy Application

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Gene Therapy Applications
Gene targeting through ZFNs or TALEN-based approaches can also be used to modify defective genes at their endogenous chromosomal locations. Examples include the treatment of X-linked severe combined immunodeficiency (X-SCID) by ex vivo gene correction with DNA carrying the interleukin-2 receptor common gamma chain (IL-2Rγ) and the correction of Xeroderma pigmentosum mutations in vitro using TALEN. Insertional mutagenesis by the retroviral vector genome induced leukemia in some patients, a problem that is predicted to be avoided by these technologies. However, ZFNs may also cause off-target mutations, in a different way from viral transductions. Currently, many measures are taken to improve off-target detection and ensure safety before treatment.
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