MyPeak-1: A Phase 1b Study to Evaluate Safety and Efficacy of TN-201, an Adeno-Associated Virus Serotype 9 (AAV9) Investigational Gene Therapy, in Adults with MYBPC3-Associated Hypertrophic Cardiomyopathy (HCM)
Jeff Haroldson, Will Harrison, Laura M. Lombardi, Gretchen Argast, Zoe Duclos, Shelly Nelson, Sunita Sethi, LaTanya Tomlinson, N.A.M. Paterson, Matthew J. Pollman, Whit Tingley, Laura Robertson
Topics & Concepts
MedicineTolerabilityHypertrophic cardiomyopathyAdeno-associated virusClinical trialGenetic enhancementInternal medicineCardiomyopathyAdverse effectHeart failureGeneVector (molecular biology)Recombinant DNAChemistryBiochemistryCardiomyopathy and Myosin StudiesViral Infections and Immunology ResearchCRISPR and Genetic Engineering