Improving Gene Therapy for Angelman Syndrome with Secreted Human UBE3A
Austin W. Nenninger, Matthew Willman, Jonathan Willman, Emma Stewart, Philippe Mesidor, Michelle Novoa, Nicole K. Morrill, Luis Alvarez, Aurélie Joly‐Amado, Melinda Peters, Danielle Gulick, Kevin Nash
Topics & Concepts
UBE3AAngelman syndromeAdeno-associated virusGenetic enhancementBiologyMedicineGeneRecombinant DNAUbiquitin ligaseGeneticsVector (molecular biology)UbiquitinGenetic Syndromes and ImprintingGenetics and Neurodevelopmental DisordersCancer-related gene regulation