With its first AAV gene therapy program, DiNA-001, in development for the treatment of MYBPC3 hypertrophic cardiomyopathy (HCM), the company ... In a large number of mouse, non-human primate ...

Then they wanted to know if they could selectively incorporate the enhancer elements into an adult mouse using adeno-associated virus, a familiar gene therapy tool for introducing gene sequences into ...

Figure 1: Diagram ... to nude mice 11. The hair follicle has a high capacity for manufacturing proteins, a characteristic which could be exploited by in vivo and ex vivo gene therapy to produce ...

The MyPEAK-1 trial investigator Milind Desai said: “TN-201 is the first gene therapy to be tested in HCM patients whose disease is caused by mutations to the MYBPC3 gene. For patients with this ...

Mice rendered infertile through ovary cell–targeting mutations gave birth to seemingly normal offspring through natural mating after a virus-based gene therapy was injected into their ovaries. Dan is ...