Recent Publications
Wilson JM, Caplan AL. The Responsible Development of Adeno-Associated Virus Gene Therapies. Hum Gene Ther. 2025 Jul 11. PMID: 40643951.
Hordeaux J, Lamontagne RJ, Bandyopadhyay S, Bell P, Wilson JM, Flotte TR. Lung endothelial transduction in a patient that succumbed to acute respiratory distress syndrome following high-dose rAAV9 gene therapy. Mol Ther. 2025 Jun 4;33(6):2339-2342. PMID: 40441149.
Padilla MS, Mrksich K, Wang Y, Haley RM, Li JJ, Han EL, El-Mayta R, Kim EH, Dias S, Gong N, Teerdhala SV, Han X, Chowdhary V, Xue L, Siddiqui Z, Yamagata HM, Kim D, Yoon IC, Wilson JM, Radhakrishnan R, Mitchell MJ. Branched endosomal disruptor (BEND) lipids mediate delivery of mRNA and CRISPR-Cas9 ribonucleoprotein complex for hepatic gene editing and T cell engineering. Nat Commun. 2025 Jan 24;16(1):996. PMID: 39856035.
Han X, Alameh MG, Xu Y, Palanki R, El-Mayta R, Dwivedi G, Swingle KL, Xu J, Gong N, Xue L, Shi Q, Yoon IC, Warzecha CC, Wilson JM, Weissman D, Mitchell MJ. Optimization of the activity and biodegradability of ionizable lipids for mRNA delivery via directed chemical evolution. Nat Biomed Eng. 2024 Nov;8(11):1412-1424. PMID: 39578640.
Xue L, Zhao G, Gong N, Han X, Shepherd SJ, Xiong X, Xiao Z, Palanki R, Xu J, Swingle KL, Warzecha CC, El-Mayta R, Chowdhary V, Yoon IC, Xu J, Cui J, Shi Y, Alameh MG, Wang K, Wang L, Pochan DJ, Weissman D, Vaughan AE, Wilson JM, Mitchell MJ. Combinatorial design of siloxane-incorporated lipid nanoparticles augments intracellular processing for tissue-specific mRNA therapeutic delivery. Nat Nanotechnol. 2025 Jan;20(1):132-143. PMID: 39354147.
Quadrini KJ, Vrentas C, Duke C, Wilson C, Hinderer CJ, Weinstein DA, Al-Zaidy SA, Browne SE, Wilson JM, Ni YG. Validation of high-sensitivity assays to quantitate cerebrospinal fluid and serum β-galactosidase activity in patients with GM1-gangliosidosis. Mol Ther Methods Clin Dev. 2024 Aug 10;32(3):101318. PMID: 39282076.
Hoffman JA, Denton N, Sims JJ, Meggersee R, Zhang Z, Olagbegi K, Wilson JM. Modulation of AAV9 Galactose Binding Yields Novel Gene Therapy Vectors and Predicts Cross-Species Differences in Glycan Avidity. Hum Gene Ther. 2024 Sep;35(17-18):734-753. PMID: 39001819.
Greig JA, Jennis M, Dandekar A, Chorazeczewski JK, Param N, So M, Nayal M, Bell P, Coughlan K, Choi M, Giangrande PH, Martini PGV, Wilson JM. Lipid Nanoparticle mRNA Therapy Improves Survival and Reduces Serum Branched-Chain Amino Acids in Mouse Models of Maple Syrup Urine Disease. Hum Gene Ther. 2024 Sep;35(17-18):726-733. PMID: 39001827.
Han X, Alameh MG, Gong N, Xue L, Ghattas M, Bojja G, Xu J, Zhao G, Warzecha CC, Padilla MS, El-Mayta R, Dwivedi G, Xu Y, Vaughan AE, Wilson JM, Weissman D, Mitchell MJ. Fast and facile synthesis of amidine-incorporated degradable lipids for versatile mRNA delivery in vivo. Nat Chem. 2024 Oct;16(10):1687-1697. PMID: 38982196.
Werner MS, Aras S, Morgan AR, Roamer J, Param NJ, Olagbegi K, Lamontagne RJ, Greig JA, Wilson JM. Adeno-associated virus-mediated trastuzumab delivery to the central nervous system for human epidermal growth factor receptor 2+ brain metastasis. Cancer Gene Ther. 2024 May;31(5):766-777. PMID: 38480976.
A complete list of James M. Wilson’s published work is available in MyBibliography.
Recent Presentations
American Society for Gene & Cell Therapy (ASGCT) 2025
946. Prevalence of Neutralizing Antibodies Against Adeno-Associated Virus (AAV) 8 and 9 in a Specific Pathogen-Free (SPF) Colony of Cynomolgus Macaques. Marcela Salazar Werner, Jenny Greig, Surina Boyd, Qiuyue Qin, Chelsea Wallace, Jessica Chichester, James M. Wilson.
410. Position-Specific Impact of RGD Motif in AAV Capsid Variants on Muscle Tissue Targeting and Integrin Binding Affinity. Rosemary L. Meggersee, Payal Grover, Swapnil Shewale, R. Jason Lamontagne, Juliette Hordeaux, Jenny A. Greig, James M. Wilson.
1025. Gene Replacement Therapy for Barth Syndrome Using a Novel Cardiotropic Capsid. Juliette Hordeaux, Nicholas Roberts, Yingxian Chen, Swapnil Shewale, Ali Ramezani, Clarissa Shoffler, Dina Abbasian, Christopher Petucci, James M. Wilson.
2011. Single Cell Analysis of AAV9 Transduction in a Patient that Succumbed to Acute Respiratory Distress Syndrome Following Systemic High Dose Gene Therapy. Juliette Hordeaux, R. Jason Lamontagne, Peter Bell, Sushobhana Bandyopadhyay, Jing-Xu Zhu, Hongwei Yu, Yanqing Zhu, Darcy Reil, Juan Putra, Richard Horgan, James M. Wilson, Terence R. Flotte.