Article

Efficient delivery of <i>FMR1</i> across the blood brain barrier using AAVphp construct in adult <i>FMR1</i> KO mice demonstrates the feasibility of gene therapy for fragile X syndrome

2022-12-23

Abstract excerpt

<h4>Background</h4> Fragile X syndrome (FXS) is the most common inherited cause of intellectual disability and autism. Gene therapy may offer an efficient method to ameliorate symptoms of the disorder. <h4>Methods</h4> An AAVphp.eb-hSyn-mFMR1IOS7 vector and an empty control were injected in the tail vein of Fmr1 knock out (KO) mouse and wildtype (WT) controls. KO mice were injected with 2 × 10 13 vg/kg of the...

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Literature Corpus work: a7bfb660-3777-5204-8871-00ec145509e7
DOI: 10.1101/2022.12.22.520936

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