Selected Publications
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 : 100253,2024.
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 : 2003-2014,2023.
Musunuru K.: Playing the genetic lottery: an interview with Kiran Musunuru Dis Model Mech 16 : dmm050508,2023.
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 : 2075-2077,2023.
Allyse MA, Meagher KM, Michie M, Isasi R, Ormond KE, Bonhomme N, Bombard Y, Howard H, Musunuru K, Riggan KA, Rubeck S.: Translational justice in human gene editing: bringing end user engagement and policy together Am J Bioeth 23 : 55-58,2023.
Testa LC, Musunuru K.: Base editing and prime editing: potential therapeutic options for rare and common diseases BioDrugs 37 : 453-462,2023.
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 : 3451,2023.
Gawronski KAB, Bone WP, Park Y, Pashos EE, Wenz BM, Dudek MF, Wang X, Yang W, Rader DJ, Musunuru K, Voight BF, Brown CD.: Evaluating the contribution of cell type-specific alternative splicing to variation in lipid levels Circ Genom Precis Med 16 : 248-257,2023.
Kasiewicz LN, Biswas S, Beach A, Ren H, Dutta C, Mazzola AM, Rohde E, Chadwick A, Cheng C, Garcia SP, Iyer S, Matsumoto Y, Khera AV, Musunuru K, Kathiresan S, Malyala P, Rajeev KG, Bellinger AM.: GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy Nat Commun 14 : 2776,2023.
Davis JR, Banskota S, Levy JM, Newby GA, Wang X, Anzalone AV, Nelson AT, Chen PJ, Hennes AD, An M, Roh H, Randolph PB, Musunuru K, Liu DR.: Efficient prime editing in mouse brain, liver and heart with dual AAVs Nat Biotechnol : 2023.
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