My Bibliography

select

Wilson JM, Caplan AL. The Responsible Development of Adeno-Associated Virus Gene Therapies. Hum Gene Ther. 2025 Nov;36(21-22):1369-1370. doi: 10.1177/10430342251359668. Epub 2025 Jul 11. PubMed PMID: 40643951.

select

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. doi: 10.1016/j.ymthe.2025.05.017. Epub 2025 May 28. PubMed PMID: 40441149; PubMed Central PMCID: PMC12172266.

select

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. doi: 10.1038/s41467-024-55137-6. PubMed PMID: 39856035; PubMed Central PMCID: PMC11759712.

select

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. doi: 10.1038/s41551-024-01267-7. Epub 2024 Nov 22. PubMed PMID: 39578640.

select

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. doi: 10.1038/s41565-024-01747-6. Epub 2024 Oct 1. PubMed PMID: 39354147; PubMed Central PMCID: PMC12207990.

select

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. doi: 10.1089/hum.2024.050. Epub 2024 Aug 13. PubMed PMID: 39001819.

select

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 Sep 12;32(3):101318. doi: 10.1016/j.omtm.2024.101318. eCollection 2024 Sep 12. PubMed PMID: 39282076; PubMed Central PMCID: PMC11401230.

select

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. doi: 10.1089/hum.2024.047. Epub 2024 Aug 5. PubMed PMID: 39001827.

select

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. doi: 10.1038/s41557-024-01557-2. Epub 2024 Jul 9. PubMed PMID: 38982196; PubMed Central PMCID: PMC11446653.

select

Wilson JM, Philippidis A, Davies K. The State of Gene Therapy: An Interview with Jim Wilson. GEN Biotechnology. 2024 April; 3(2).

select

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. doi: 10.1038/s41417-024-00751-1. Epub 2024 Mar 13. PubMed PMID: 38480976.

select

Han X, Xu J, Xu Y, Alameh MG, Xue L, Gong N, El-Mayta R, Palanki R, Warzecha CC, Zhao G, Vaughan AE, Wilson JM, Weissman D, Mitchell MJ. In situ combinatorial synthesis of degradable branched lipidoids for systemic delivery of mRNA therapeutics and gene editors. Nat Commun. 2024 Feb 26;15(1):1762. doi: 10.1038/s41467-024-45537-z. PubMed PMID: 38409275; PubMed Central PMCID: PMC10897129.

select

Hordeaux J, Lamontagne RJ, Song C, Buchlis G, Dyer C, Buza EL, Ramezani A, Wielechowski E, Greig JA, Chichester JA, Bell P, Wilson JM. High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury. Mol Ther. 2024 Apr 3;32(4):952-968. doi: 10.1016/j.ymthe.2024.02.002. Epub 2024 Feb 6. PubMed PMID: 38327046; PubMed Central PMCID: PMC11163197.

select

Qiu L, Xu E, Chambule S, LaTourette P 2nd, Dyer CD, Wallace CK, Donocoff R, Wilson JM, Lucas TH, Chen HI. Magnetic Resonance Imaging-Guided Frameless Stereotactic Injections of the Bilateral Cerebellar Dentate Nuclei in Nonhuman Primates: Technical Note. Oper Neurosurg. 2024 Feb 1;. doi: 10.1227/ons.0000000000001050. [Epub ahead of print] PubMed PMID: 38310346.

select

Greig JA, Martins KM, Breton C, Lamontagne RJ, Zhu Y, He Z, White J, Zhu JX, Chichester JA, Zheng Q, Zhang Z, Bell P, Wang L, Wilson JM. Integrated vector genomes may contribute to long-term expression in primate liver after AAV administration. Nat Biotechnol. 2024 Aug;42(8):1232-1242. doi: 10.1038/s41587-023-01974-7. Epub 2023 Nov 6. PubMed PMID: 37932420; PubMed Central PMCID: PMC11324525.

select

Martins KM, Breton C, Zheng Q, Zhang Z, Latshaw C, Greig JA, Wilson JM. Prevalent and Disseminated Recombinant and Wild-Type Adeno-Associated Virus Integration in Macaques and Humans. Hum Gene Ther. 2023 Nov;34(21-22):1081-1094. doi: 10.1089/hum.2023.134. Epub 2023 Nov 6. PubMed PMID: 37930949; PubMed Central PMCID: PMC10659022.

select

Rojsajjakul T, Hordeaux JJ, Choudhury GR, Hinderer CJ, Mesaros C, Wilson JM, Blair IA. Quantification of human mature frataxin protein expression in nonhuman primate hearts after gene therapy. Commun Biol. 2023 Oct 27;6(1):1093. doi: 10.1038/s42003-023-05472-z. PubMed PMID: 37891254; PubMed Central PMCID: PMC10611776.

select

Rosenberg JB, Fung EK, Dyke JP, De BP, Lou H, Kelly JM, Reejhsinghani L, Ricart Arbona RJ, Sondhi D, Kaminsky SM, Cartier N, Hinderer C, Hordeaux J, Wilson JM, Ballon DJ, Crystal RG. Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid. Hum Gene Ther. 2023 Nov;34(21-22):1095-1106. doi: 10.1089/hum.2023.060. Epub 2023 Oct 23. PubMed PMID: 37624734; PubMed Central PMCID: PMC10659018.

select

Vaden SL, Kendall AR, Foster JD, New HL, Eagleson JS, May JL, Traas AM, Wilson MJ, McIntyre BH, Hinderer CJ, Olenick LK, Wilson JM. Adeno-associated virus-vectored erythropoietin gene therapy for anemia in cats with chronic kidney disease. J Vet Intern Med. 2023 Nov-Dec;37(6):2200-2210. doi: 10.1111/jvim.16900. Epub 2023 Oct 17. PubMed PMID: 37847024; PubMed Central PMCID: PMC10658539.

select

Kistner A, Chichester JA, Wang L, Calcedo R, Greig JA, Cardwell LN, Wright MC, Couthouis J, Sethi S, McIntosh BE, McKeever K, Wadsworth S, Wilson JM, Kakkis E, Sullivan BA. Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques. Gene Ther. 2024 Mar;31(3-4):128-143. doi: 10.1038/s41434-023-00423-z. Epub 2023 Oct 13. PubMed PMID: 37833563; PubMed Central PMCID: PMC10940161.

select

Giles A, Lock M, Chen SJ, Turner K, Wesolowski G, Prongay A, Petkov BN, Olagbegi K, Yan H, Wilson JM. Significant Differences in Capsid Properties and Potency Between Adeno-Associated Virus Vectors Produced in Sf9 and HEK293 Cells. Hum Gene Ther. 2023 Oct;34(19-20):1003-1021. doi: 10.1089/hum.2022.116. Epub 2023 Oct 4. PubMed PMID: 37597192.

select

Martino RA, Wang Q, Xu H, Hu G, Bell P, Arroyo EJ, Sims JJ, Wilson JM. Vector Affinity and Receptor Distribution Define Tissue-Specific Targeting in an Engineered AAV Capsid. J Virol. 2023 Jun 29;97(6):e0017423. doi: 10.1128/jvi.00174-23. Epub 2023 May 18. PubMed PMID: 37199615; PubMed Central PMCID: PMC10308920.

select

Hordeaux J, Ramezani A, Tuske S, Mehta N, Song C, Lynch A, Lupino K, Chichester JA, Buza EL, Dyer C, Yu H, Bell P, Weimer JM, Do H, Wilson JM. Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase. Front Immunol. 2023;14:1094279. doi: 10.3389/fimmu.2023.1094279. eCollection 2023. PubMed PMID: 37033976; PubMed Central PMCID: PMC10073725.

select

Horiuchi M, Hinderer CJ, Shankle HN, Hayashi PM, Chichester JA, Kissel C, Bell P, Dyer C, Wilson JM. Neonatal Fc Receptor Inhibition Enables Adeno-Associated Virus Gene Therapy Despite Pre-Existing Humoral Immunity. Hum Gene Ther. 2023 Oct;34(19-20):1022-1032. doi: 10.1089/hum.2022.216. Epub 2023 Mar 17. PubMed PMID: 36719773.

select

Greig JA, Chorazeczewski JK, Chowdhary V, Smith MK, Jennis M, Tarrant JC, Buza EL, Coughlan K, Martini PGV, Wilson JM. Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model. Mol Ther Methods Clin Dev. 2023 Jun 8;29:32-39. doi: 10.1016/j.omtm.2023.02.007. eCollection 2023 Jun 8. PubMed PMID: 36936447; PubMed Central PMCID: PMC10017950.

select

Horiuchi M, Hinderer CJ, Greig JA, Dyer C, Buza EL, Bell P, Chichester JA, Hayashi PM, Yan H, Goode T, Wilson JM. Erratum: Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors. Mol Ther Methods Clin Dev. 2022 Dec 8;27:450-451. doi: 10.1016/j.omtm.2022.11.001. eCollection 2022 Dec 8. PubMed PMID: 36419470; PubMed Central PMCID: PMC9668676.

select

Greig JA, Breton C, Ashley SN, Martins KM, Gorsuch C, Chorazeczewski JK, Furmanak T, Smith MK, Zhu Y, Bell P, Shoop W, Li H, Smith J, Tomberlin G, Clark P, Mitchell TW, Buza EL, Yan H, Jantz D, Wilson JM. Treating Transthyretin Amyloidosis via Adeno-Associated Virus Vector Delivery of Meganucleases. Hum Gene Ther. 2022 Nov;33(21-22):1174-1186. doi: 10.1089/hum.2022.061. PubMed PMID: 36375122; PubMed Central PMCID: PMC9700363.

select

Horiuchi M, Hinderer CJ, Greig JA, Dyer C, Buza EL, Bell P, Chichester JA, Hayashi PM, Yan H, Goode T, Wilson JM. Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors. Mol Ther Methods Clin Dev. 2022 Dec 8;27:272-280. doi: 10.1016/j.omtm.2022.09.017. eCollection 2022 Dec 8. PubMed PMID: 36320416; PubMed Central PMCID: PMC9593247.

select

Nambiar K, Wang Q, Yan H, Wilson JM. Characterizing Complex Populations of Endogenous Adeno-Associated Viruses by Single-Genome Amplification. Hum Gene Ther. 2022 Nov;33(21-22):1164-1173. doi: 10.1089/hum.2022.099. Epub 2022 Sep 22. PubMed PMID: 35906801.

select

Sims JJ, Lian S, Meggersee RL, Kasimsetty A, Wilson JM. High activity of an affinity-matured ACE2 decoy against Omicron SARS-CoV-2 and pre-emergent coronaviruses. PLoS One. 2022;17(8):e0271359. doi: 10.1371/journal.pone.0271359. eCollection 2022. PubMed PMID: 36006993; PubMed Central PMCID: PMC9409550.

select

Zabaleta N, Bhatt U, Hérate C, Maisonnasse P, Sanmiguel J, Diop C, Castore S, Estelien R, Li D, Dereuddre-Bosquet N, Cavarelli M, Gallouët AS, Pascal Q, Naninck T, Kahlaoui N, Lemaitre J, Relouzat F, Ronzitti G, Thibaut HJ, Montomoli E, Wilson JM, Le Grand R, Vandenberghe LH. Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques. Mol Ther. 2022 Sep 7;30(9):2952-2967. doi: 10.1016/j.ymthe.2022.05.007. Epub 2022 May 10. PubMed PMID: 35546782; PubMed Central PMCID: PMC9088091.

select

Hordeaux J, Jeffrey BA, Jian J, Choudhury GR, Michalson K, Mitchell TW, Buza EL, Chichester J, Dyer C, Bagel J, Vite CH, Bradbury AM, Wilson JM. Efficacy and Safety of a Krabbe Disease Gene Therapy. Hum Gene Ther. 2022 May;33(9-10):499-517. doi: 10.1089/hum.2021.245. Epub 2022 Mar 22. PubMed PMID: 35333110; PubMed Central PMCID: PMC9142772.

select

Wang L, Warzecha CC, Kistner A, Chichester JA, Bell P, Buza EL, He Z, Pampena MB, Couthouis J, Sethi S, McKeever K, Betts MR, Kakkis E, Wilson JM, Wadsworth S, Sullivan BA. Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression. Mol Ther Methods Clin Dev. 2022 Mar 10;24:292-305. doi: 10.1016/j.omtm.2022.01.007. eCollection 2022 Mar 10. PubMed PMID: 35211641; PubMed Central PMCID: PMC8841522.

select

Greig JA, Smith MK, Nordin JML, Goode T, Chroscinski EA, Buza EL, Schmidt N, Kattenhorn LM, Wadsworth S, Wilson JM. Determining the Minimally Effective Dose of a Clinical Candidate Adeno-Associated Virus Vector in a Mouse Model of Hemophilia A. Hum Gene Ther. 2022 Apr;33(7-8):421-431. doi: 10.1089/hum.2021.108. Epub 2021 Dec 8. PubMed PMID: 34652966; PubMed Central PMCID: PMC9063151.

select

Wang Q, Nambiar K, Wilson JM. Isolating Natural Adeno-Associated Viruses from Primate Tissues with a High-Fidelity Polymerase. Hum Gene Ther. 2021 Dec;32(23-24):1439-1449. doi: 10.1089/hum.2021.055. Epub 2021 Aug 26. PubMed PMID: 34448594.

select

Tycko J, Adam VS, Crosariol M, Ohlstein J, Sanmiguel J, Tretiakova AP, Roy S, Worgall S, Wilson JM, Limberis MP. Adeno-Associated Virus Vector-Mediated Expression of Antirespiratory Syncytial Virus Antibody Prevents Infection in Mouse Airways. Hum Gene Ther. 2021 Dec;32(23-24):1450-1456. doi: 10.1089/hum.2021.079. Epub 2021 Aug 20. PubMed PMID: 34415793.

select

Greig JA, Jennis M, Dandekar A, Chorazeczewski JK, Smith MK, Ashley SN, Yan H, Wilson JM. Muscle-directed AAV gene therapy rescues the maple syrup urine disease phenotype in a mouse model. Mol Genet Metab. 2021 Sep-Oct;134(1-2):139-146. doi: 10.1016/j.ymgme.2021.08.003. Epub 2021 Aug 17. PubMed PMID: 34454844.

select

Zabaleta N, Dai W, Bhatt U, Hérate C, Maisonnasse P, Chichester JA, Sanmiguel J, Estelien R, Michalson KT, Diop C, Maciorowski D, Dereuddre-Bosquet N, Cavarelli M, Gallouët AS, Naninck T, Kahlaoui N, Lemaitre J, Qi W, Hudspeth E, Cucalon A, Dyer CD, Pampena MB, Knox JJ, LaRocque RC, Charles RC, Li D, Kim M, Sheridan A, Storm N, Johnson RI, Feldman J, Hauser BM, Contreras V, Marlin R, Tsong Fang RH, Chapon C, van der Werf S, Zinn E, Ryan A, Kobayashi DT, Chauhan R, McGlynn M, Ryan ET, Schmidt AG, Price B, Honko A, Griffiths A, Yaghmour S, Hodge R, Betts MR, Freeman MW, Wilson JM, Le Grand R, Vandenberghe LH. An AAV-based, room-temperature-stable, single-dose COVID-19 vaccine provides durable immunogenicity and protection in non-human primates. Cell Host Microbe. 2021 Sep 8;29(9):1437-1453.e8. doi: 10.1016/j.chom.2021.08.002. Epub 2021 Aug 7. PubMed PMID: 34428428; PubMed Central PMCID: PMC8346325.

select

Martino RA, Fluck EC 3rd, Murphy J, Wang Q, Hoff H, Pumroy RA, Lee CY, Sims JJ, Roy S, Moiseenkova-Bell VY, Wilson JM. Context-Specific Function of the Engineered Peptide Domain of PHP.B. J Virol. 2021 Sep 27;95(20):e0116421. doi: 10.1128/JVI.01164-21. Epub 2021 Aug 4. PubMed PMID: 34346767; PubMed Central PMCID: PMC8475521.

select

Sims JJ, Greig JA, Michalson KT, Lian S, Martino RA, Meggersee R, Turner KB, Nambiar K, Dyer C, Hinderer C, Horiuchi M, Yan H, Huang X, Chen SJ, Wilson JM. Intranasal gene therapy to prevent infection by SARS-CoV-2 variants. PLoS Pathog. 2021 Jul;17(7):e1009544. doi: 10.1371/journal.ppat.1009544. eCollection 2021 Jul. PubMed PMID: 34265018; PubMed Central PMCID: PMC8282039.

select

Shepherd SJ, Warzecha CC, Yadavali S, El-Mayta R, Alameh MG, Wang L, Weissman D, Wilson JM, Issadore D, Mitchell MJ. Scalable mRNA and siRNA Lipid Nanoparticle Production Using a Parallelized Microfluidic Device. Nano Lett. 2021 Jul 14;21(13):5671-5680. doi: 10.1021/acs.nanolett.1c01353. Epub 2021 Jun 30. PubMed PMID: 34189917; PubMed Central PMCID: PMC10726372.

select

Wang L, Muthuramu I, Somanathan S, Zhang H, Bell P, He Z, Yu H, Zhu Y, Tretiakova AP, Wilson JM. Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia. Mol Ther Methods Clin Dev. 2021 Sep 10;22:1-10. doi: 10.1016/j.omtm.2021.04.017. eCollection 2021 Sep 10. PubMed PMID: 34258325; PubMed Central PMCID: PMC8237527.

select

Schmid RS, Deng X, Panikker P, Msackyi M, Breton C, Wilson JM. CRISPR/Cas9 directed to the Ube3a antisense transcript improves Angelman syndrome phenotype in mice. J Clin Invest. 2021 Mar 1;131(5). doi: 10.1172/JCI142574. PubMed PMID: 33411694; PubMed Central PMCID: PMC7919720.

select

Wang L, Breton C, Warzecha CC, Bell P, Yan H, He Z, White J, Zhu Y, Li M, Buza EL, Jantz D, Wilson JM. Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9. Mol Ther. 2021 Jun 2;29(6):2019-2029. doi: 10.1016/j.ymthe.2021.02.020. Epub 2021 Feb 18. PubMed PMID: 33609733; PubMed Central PMCID: PMC8178442.

select

Zabaleta N, Dai W, Bhatt U, Chichester JA, Sanmiguel J, Estelien R, Michalson KT, Diop C, Maciorowski D, Qi W, Hudspeth E, Cucalon A, Dyer CD, Pampena MB, Knox JJ, LaRocque RC, Charles RC, Li D, Kim M, Sheridan A, Storm N, Johnson RI, Feldman J, Hauser BM, Zinn E, Ryan A, Kobayashi DT, Chauhan R, McGlynn M, Ryan ET, Schmidt AG, Price B, Honko A, Griffiths A, Yaghmour S, Hodge R, Betts MR, Freeman MW, Wilson JM, Vandenberghe LH. Immunogenicity of an AAV-based, room-temperature stable, single dose COVID-19 vaccine in mice and non-human primates. bioRxiv. 2021 Jan 19;. doi: 10.1101/2021.01.05.422952. PubMed PMID: 33442684; PubMed Central PMCID: PMC7805441.

select

Zhang R, El-Mayta R, Murdoch TJ, Warzecha CC, Billingsley MM, Shepherd SJ, Gong N, Wang L, Wilson JM, Lee D, Mitchell MJ. Helper lipid structure influences protein adsorption and delivery of lipid nanoparticles to spleen and liver. Biomater Sci. 2021 Feb 21;9(4):1449-1463. doi: 10.1039/d0bm01609h. Epub 2021 Jan 6. PubMed PMID: 33404020; PubMed Central PMCID: PMC8753632.

select

Breton C, Furmanak T, Avitto AN, Smith MK, Latshaw C, Yan H, Greig JA, Wilson JM. Increasing the Specificity of AAV-Based Gene Editing through Self-Targeting and Short-Promoter Strategies. Mol Ther. 2021 Mar 3;29(3):1047-1056. doi: 10.1016/j.ymthe.2020.12.028. Epub 2020 Dec 25. PubMed PMID: 33359790; PubMed Central PMCID: PMC7934631.

select

Hordeaux J, Buza EL, Jeffrey B, Song C, Jahan T, Yuan Y, Zhu Y, Bell P, Li M, Chichester JA, Calcedo R, Wilson JM. MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates. Sci Transl Med. 2020 Nov 11;12(569). doi: 10.1126/scitranslmed.aba9188. PubMed PMID: 33177182.

select

Hinderer C, Nosratbakhsh B, Katz N, Wilson JM. A Single Injection of an Optimized Adeno-Associated Viral Vector into Cerebrospinal Fluid Corrects Neurological Disease in a Murine Model of GM1 Gangliosidosis. Hum Gene Ther. 2020 Nov;31(21-22):1169-1177. doi: 10.1089/hum.2018.206. Epub 2020 Nov 2. PubMed PMID: 33045869; PubMed Central PMCID: PMC7698982.

select

Hinderer C, Miller R, Dyer C, Johansson J, Bell P, Buza E, Wilson JM. Adeno-associated virus serotype 1-based gene therapy for FTD caused by GRN mutations. Ann Clin Transl Neurol. 2020 Oct;7(10):1843-1853. doi: 10.1002/acn3.51165. Epub 2020 Sep 16. PubMed PMID: 32937039; PubMed Central PMCID: PMC7545603.