Selected Publications

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 May 2025.

Madelynn N. Whittaker, Lauren C. Testa, Aidan Quigley, Dominique L. Brooks, Sarah A. Grandinette, Hooda Said, Garima Dwivedi, Ishaan Jindal, Daphne Volpp, Julia L. Hacker, Ping Qu, Josh Zhiyong Wang, Michael A. Levine, Rebecca C. Ahrens-Nicklas, Qiaoli Li, Kiran Musunuru, Mohamad-Gabriel Alameh, William H. Peranteau1 & Xiao Wang: Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs. Nature Biomedical Engineering 2025 Notes: accepted for publication; in press now.

Choe D, Burke M, Brandimarto JA, Marti-Pamies I, Yob J, Yang Y, Morley MP, Drivas TG, Day S, Levin M, Damrauer S, Wang X*, Cappola TP*.: Sex-Specific Effects of MTSS1 Downregulation on Dilated Cardiomyopathy. Circulation 150: 1648-1651, Nov 2024 Notes: (*These authors jointly directed this work).

Brooks DL, Whittaker MN, Said H, Dwivedi G, Qu P, Musunuru K, Ahrens-Nicklas RC, Alameh MG, Wang X.: A base editing strategy using mRNA-LNPs for in vivo correction of the most frequent phenylketonuria variant. HGG Adv 5: 100253, Jan 2024.

Brooks DL, Whittaker MN, Qu P, Musunuru K, Ahrens-Nicklas RC, Wang X.: Efficient in vivo prime editing corrects the most frequent phenylketonuria variant, associated with high unmet medical need. Am J Hum Genet 110: 2003-2014, Dec 2023.

Whittaker MN, Testa LC, Quigley A, Jindal I, Cortez-Alvarado SV, Qu P, Yang Y, Alameh MG, Musunuru K, Wang X.: Epigenome Editing Durability Varies Widely Across Cardiovascular Disease Target Genes. Arterioscler Thromb Vasc Biol Oct 2023.

Brooks DL, Carrasco MJ, Qu P, Peranteau WH, Ahrens-Nicklas RC, Musunuru K, Alameh MG, Wang X.: Rapid and definitive treatment of phenylketonuria in variant-humanized mice with corrective editing. Nat Commun 14: 3451, Jun 2023.

Davis JR, Wang X, Witte IP, Huang TP, Levy JM, Raguram A, Banskota S, Seidah NG, Musunuru K, Liu DR.: Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors. Nat Biomed Eng 6(11): 1272-1283, Nov 2022.

Banskota S, Raguram A, Suh S, Du SW, Davis JR, Choi EH, Wang X, Nielsen SC, Newby GA, Randolph PB, Osborn MJ, Musunuru K, Palczewski K, Liu DR: Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins. Cell 185(2): 250-265, Jan 2022.

Morley MP*, Wang X*, Hu R, Brandimarto J, Tucker NR, Felix JF, Smith NL, Harst P, Ellinor PT, Margulies KB, Musunuru K, Cappola TP: Cardioprotective Effects of MTSS1 Enhancer Variants. Circulation 139(17): 2073–2076, Apr 2019 Notes: (*These authors contributed equally to this work).