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American Journal of Human Genetics|July 29, 2022
Rescue of a familial dysautonomia mouse model by AAV9-Exon-specific U1 snRNAGiulia Romano, Federico Riccardi, Erica Bussani, et al.
Molecular Therapy. Methods & Clinical Development|December 25, 2018
AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe MicePasqualina Colella, Pauline Sellier, Helena Costa Verdera, et al.
Frontiers in Cardiovascular Medicine|January 26, 2024
Harnessing the power of RNA therapeutics in treating ischemic heart failure: the TRAIN-HEART storyAlba M Albert, Deepak Balamurali, Evangelia Beslika, et al.
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