Selected Publications

Testa LC, Obiri-Yeboah D, Said H, Qu P, Levine MA, Alameh MG, Musunuru K, Li Q, Wang X.: Liver-directed base editing of ABCC6 prevents ectopic calcification in a variant-humanized mouse model of pseudoxanthoma elasticum. Mol Ther Nucleic Acids 36(4): 102769, Nov 2025.

Quigley A, Jindal I, Campion T, Rutherford D, Han Y, Quigley W, Qu P, Musunuru K, Ahrens-Nicklas R, Hong X, Wang X.: Base editing strategies for in vivo correction of two highly recurrent phenylketonuria variants. Mol Ther Nucleic Acids 36(4): 102770, Nov 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 248(2): 817-834, Oct 2025.

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(22): 2235–2247, May 2025.

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(20): 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(1): 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(12): 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 43(10): 2075–2077, 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 10(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.