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Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders

17 Oct 2024

Abstract excerpt

Pediatric monogenetic diseases encompass a spectrum of debilitating neurological disorders that affect infants and children, often resulting in profound cognitive and motor impairments. Gene replacement therapy holds immense promise in addressing the underlying genetic defects responsible for these conditions. Adeno-associated virus (AAV) vectors have emerged as a leading platform for delivering therapeutic genes...

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CRISPR and Genetic Engineering
Neurogenetic and Muscular Disorders Research
Virus-based gene therapy research

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 39559557
DOI: 10.1016/j.omtm.2024.101357

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