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Susan F. Rogers

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#gene editing Review Open access Oct 2026

Challenges in Gene-Directed Therapies for Rare Neurodevelopmental Disorders Using HNRNPU as an Exemplar.

Next-generation sequencing has radically transformed the diagnostic capability of rare neurodevelopmental disorders (RNDDs), yet translating these molecular insights into viable therapeutics remains a hurdle. While gene-directed modalities, specifically viral-mediated supplementation, antisense oligonucleotides (ASOs),...

D. Aiyedun, Susan F. Rogers, D. M. Taha et al. · 0 citations

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