US EditionJCI A haploinsufficiency restoration strategy corrects neurobehavioral deficits in Nf1+/– mice
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JCI A haploinsufficiency restoration strategy corrects neurobehavioral deficits in Nf1+/– mice
Neurofibromatosis type 1 (NF1) is a genetic disorder caused by mutations of the NF1 tumor suppressor gene resulting in the loss of function of neurofibromin, a GTPase-activating protein (GAP) for Ras. While the malignant manifestations of NF1 are associated with loss of heterozygosity of the residual WT allele, the nonmalignant neurodevelopmental sequelae, including autism spectrum disorder (ASD) and/or attention deficit hyperactivity disorder (…
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