Musunuru's researches the genetics and genomics of cardiovascular and metabolic diseases, and is a leading expert in genome-editing techniques, particularly
CRISPR-Cas9. His lab was the first to develop an efficient technique to genetically modify human pluripotent stem cells, and differentiate them to model disease. He discovered two novel genes involved in
coronary artery disease: SORT1 and ANGPTL3. Musunuru has pioneered the use of genome-editing tools as therapies for heart disease. For example, some people with a variant of the PCSK9 gene have lower levels of low-density lipoprotein (LDL) cholesterol levels, and therefore have a reduced risk of heart attack. 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. 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. They used the test to determine that a 65-year-old woman's specific variant of TNNT2, a gene which has been associated with cardiomyopathy, was not pathogenic. He is now a Professor of Medicine at the University of Pennsylvania Perelman School of Medicine. Musunuru is the author of The CRISPR Generation: The Story of the World's First Gene-Edited Babies, in which he delves into the scientific breakthroughs that enabled
He Jiankui to create the world's first
gene-edited babies, a scandal Musunuru describes as a "historic ethical fiasco, a deeply flawed experiment". The disorder is caused by a mutation in the CPS1 gene. == Awards and honours ==