Advances in Gene Therapy for Rare Diseases: Targeting Functional Haploinsufficiency Through AAV and mRNA Approaches - Bara-Ledesma, Nuria; Viteri-Noel, Adrian; Lopez Rodriguez, Monica; Stamatakis, Konstantinos; Fabregate, Martin; Vazquez-Santos, Almudena; Gomez del Olmo, Vicente (INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2025)