Construct a conclusion of the overall of how gene therapy if beneficial to X-linked hyper IgM.

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Answer-Targated gene editing restores regulated CD40L function in X-linked hyper --IgM .Loss of cd40 ligand expression of function results in X-linked hyper immunoglobulin syndrome,characterized by recurrent infections due to impaired immunoglobulin class switching and somatic hypermutation.This disease is amenable to gene therapy using retroviral vectors ,but dysregulated gene expression results in abnormal lymphoproliferation in mouse models.we demonstrate the ability of both the transcription activator like effector nuclease(TALEN) and clustered regularly interspaced short palindromic repeats associated protein 9 platforms to efficiently drive integration of a normal copy of cd40l ,cDNA delivered by Adeno associated virus.site specific insertion of the donor sequence downstream of the endogenous cd40l promoter maintained physiological expression of cd40l while overriding all reported downstream mutations.high levels of gene modification were achieved in primary human hematopoietic stem cells.as well as in cell lines and xhim patient derived t-cell .

gladiator answered 2 years ago

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