Pervasive horizontal transfer of adeno-associated virus capsid genes. [PDF]
Gifford RJ.
europepmc +1 more source
The calcium awakens: new insights in cardiac gene therapy. [PDF]
Santulli G.
europepmc +1 more source
Treatment With Valoctocogene Roxaparvovec in a Patient With Severe Hemophilia A Led to Sustained Normal FVIII Levels. [PDF]
Herbst K +8 more
europepmc +1 more source
First-in-human study of TN-201, an AAV9 gene replacement therapy in MYBPC3-associated hypertrophic cardiomyopathy: initial safety, pharmacodynamic, and imaging results from MyPEAK-1. [PDF]
Desai MY +17 more
europepmc +1 more source
Mosaic AAV mediates efficient transduction across the central and peripheral nervous systems. [PDF]
Jiang M +5 more
europepmc +1 more source
Development of an Agent-Based Model to Investigate Durability of Factor IX Activity in Hemophilia B Patients Treated With Etranacogene Dezaparvovec. [PDF]
Li Y +7 more
europepmc +1 more source
A New <i>Wnt1</i> Mutant Rat Model of Osteogenesis Imperfecta and Its Application in AAV9-Mediated Gene Therapy. [PDF]
Li S +9 more
europepmc +1 more source
Cloning and sequencing of the simian parvovirus genome.
K. Brown +3 more
semanticscholar +1 more source
A Rationally Designed AAV9-DM Capsid with Minimal Liver Tropism. [PDF]
Nabakowski ZC +3 more
europepmc +1 more source

