Gurevitz C, Bajaj A, Khera AV, Do R, Schunkert H, Musunuru K, Rosenson RS. Gene therapy and genome editing for lipoprotein disorders , Eur Heart J: 2025
Shuvaev VV, Tam YK, Lee BW, Myerson JW, Herbst A, Kiseleva RY, Glassman PM, Parhiz H, Alameh MG, Pardi N, Muramatsu H, Shuvaeva TI, Arguiri E, Marcos-Contreras OA, Hood ED, Brysgel TV, Nong J, Papp TE, Eaton DM, Riley R, Palanki R, Musunuru K, Brenner JS, Mitchell MJ, Ferrari VA, Mui BL, Semple SC, Weppler SA, Atluri P, Margulies KB, Weissman D, Muzykantov VR. Systemic delivery of biotherapeutic RNA to the myocardium transiently modulates cardiac contractility in vivo , Proc Natl Acad Sci U S A, 122: 2025,e2409266122
Musunuru K, Grandinette SA, Wang X, Hudson TR, Briseno K, Berry AM, Hacker JL, Hsu A, Silverstein RA, Hille LT, Ogul AN, Robinson-Garvin NA, Small JC, McCague S, Burke SM, Wright CM, Bick S, Indurthi V, Sharma S, Jepperson M, Vakulskas CA, Collingwood M, Keogh K, Jacobi A, Sturgeon M, Brommel C, Schmaljohn E, Kurgan G, Osborne T, Zhang H, Kinney K, Rettig G, Barbosa CJ, Semple SC, Tam YK, Lutz C, George LA, Kleinstiver BP, Liu DR, Ng K, Kassim SH, Giannikopoulos P, Alameh MG, Urnov FD, Ahrens-Nicklas RC. Patient-specific in vivo gene editing to treat a rare genetic disease , N Engl J Med, 392: 2025,2235-2243
Urnov F, Kassim S, Musunuru K, Liu D, Lee A, Barrera L, Stetkiewicz P, Bruno J, Hewitt M, Lister T, Malech H, Gasch L, Diver M, Gertler N, Grignon F, Le A, Lehmicke M, Almendro-Navarro V, Lembong J. Advancing gene-editing platforms to improve the viability of rare-disease therapeutics: key insights from a 2024 Scientific Exchange hosted by ARM, ISCT, and Danaher , Cytotherapy: 2025
Musunuru K, Urnov F. Moving Therapeutic Genome Editing into Global Clinical Trials and Medicine , CRISPR J: 2025
Musunuru K. A milestone for gene-editing therapies , N Engl J Med, 392: 2025,508-509
Allyse MA, Agam P, Bombard Y, Feys R, Horstmann M, Kokayi A, Isasi R, Meagher KM, Michie M, Musunuru K, Ormond KE, Riggan KA, Yap JQ. Building better medicine: translational justice and the quest for equity in US healthcare , Am J Bioeth: 2025
Kassim SH, Urnov F, Musunuru K, Lee A, Barrera L, Stetkiewicz P, Bruno J, Hewitt M, Lister T, Malech H, Gasch L, Diver M, Gertler N, Grignon F, Le A, Lehmicke M, Horwitz EM, Liu DR, Lembong J, Almendro-Navarro V. Platform solutions for commercial challenges to expanding patient access and making gene editing sustainable , Nat Biotechnol: 2025
IGVF Consortium Deciphering the impact of genomic variation on function , Nature, 633: 2024,47-57
Riggan KA, Feys R, Kokayi A, Meagher KM, Michie M, Musunuru K, Ormond KE, Schelhaas AJ, Yap JQ, Isasi R, Allyse MA. Grounded in reality: integrating community values and priorities of end users in human gene editing , Am J Bioeth, 24: 2024,43-45
