A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells
Giang N. Nguyen, J.K. Everett, Samita Kafle, Aoife M. Roche, Hayley E. Raymond, Jacob S. Leiby, Christian M. Wood, Charles‐Antoine Assenmacher, Elizabeth P. Merricks, Christian Long, Haig H. Kazazian, Timothy C. Nichols, Frederic D. Bushman, Denise E. Sabatino
Topics & Concepts
Genetic enhancementGeneBiologyFactor IXVirologyVector (molecular biology)Adeno-associated virusImmunologyMedicineMolecular biologyGeneticsRecombinant DNAVirus-based gene therapy researchViral Infectious Diseases and Gene Expression in InsectsCAR-T cell therapy research