Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact information

Cardiovascular disease

Cardiomyopathy

Myosin heavy chain 7 cardiac muscle-b (MYH7)

Mouse studies suggest RNAi that targets a disease-causing mutation in MYH7 could help prevent hypertrophic cardiomyopathy (HCM). The R403Q mutation in MYH7, which encodes the myosin heavy chain, causes progressive and early onset HCM. In ­mice with a heterozygous, homologous mutation, injection of an adenoviral vector encoding RNAi that specifically targets the mutant gene under a cardiac-specific promoter prevented both hypertrophy and fibrosis, whereas injection of a vector encoding control RNAi did not. Next steps include designing a modified adenoviral vector for delivery to the human heart.

SciBX 6(43); doi:10.1038/scibx.2013.1223
Published online Nov. 7, 2013

Patent application filed; unavailable for licensing

Jiang, J. et al. Science; published online Oct. 4, 2013;
doi:10.1126/science.1236921
Contact: Christine E. Seidman, Harvard Medical School, Boston, Mass.
e-mail:

cseidman@genetics.med.harvard.edu