His lab was the first to develop an efficient technique to genetically modify human pluripotent stem cells, and differentiate them to model disease.
[3] Musunuru ultimately hopes to create a one-shot "vaccination" against heart attacks, which would introduce a complementary, loss-of-function mutation in a gene such as ANGPTL3 to lower the risk of developing cardiovascular disease in at-risk populations.
[4][7] Musunuru has also led research into prenatal gene editing of PCSK9 or HPD using both CRISPR-Cas9 and base editor 3 (BE3), offering a proof-of-concept for a potential new therapeutic approach for congenital genetic disorders.
[8][9] Musunuru cofounded Verve Therapeutics to develop gene editing techniques for treat cholesterol altering congenital genetic disorders.
In 2019, Musunuru was among a team of researchers at the University of Pennsylvania to develop a stem cell-based test for the effect of genetic variants on heart muscle cells.