Gene Therapy Extends Life, Nerve Defects in CMT4J Mice

Gene Therapy Extends Life, Nerve Defects in CMT4J Mice

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Gene Therapy Extends Life, Nerve Defects in CMT4J Mice

In a mouse model of Charcot-Marie-Tooth disease type 4J (CMT4J), gene therapy administered in the first few days of life significantly prolonged survival and eased motor and nerve defects.  The study with that finding, “AAV9-mediated FIG4 delivery prolongs life span in Charcot Marie Tooth disease type 4J mouse model, was published in The Journal of Clinical Investigation.  CMT4J develops when an individual inherits a mutant copy of the FIG4 gene from both parents. The disorder…

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