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.
· Clinical Genetics · 0 citations