Gene Therapy Strategy Curbed Disease Features in CMT1A Rat Model

Gene Therapy Strategy Curbed Disease Features in CMT1A Rat Model

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Gene Therapy Strategy Curbed Disease Features in CMT1A Rat Model

A form of gene therapy that reduces levels of the PMP22 protein prevented the development of Charcot-Marie-Tooth disease 1A (CMT1A) features in a rat model, a study has found.  The study, “AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A,” was published in the journal Nature Communications.  CMT1A is caused by an extra copy of the PMP22 gene, which encodes the PMP22 protein, a critical…

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