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AAV gene therapy for Tay-Sachs disease.
Flotte TR, Cataltepe O, Puri A, Batista AR, Moser R, McKenna-Yasek D, Douthwright C, Gernoux G, Blackwood M, Mueller C, Tai PWL, Jiang X, Bateman S, Spanakis SG, Parzych J, Keeler AM, Abayazeed A, Rohatgi S, Gibson L, Finberg R, Barton BA, Vardar Z, Shazeeb MS, Gounis M, Tifft CJ, Eichler FS, Brown RH Jr, Martin DR, Gray-Edwards HL, Sena-Esteves M. Flotte TR, et al. Among authors: jiang x. Nat Med. 2022 Feb;28(2):251-259. doi: 10.1038/s41591-021-01664-4. Epub 2022 Feb 10. Nat Med. 2022. PMID: 35145305 Free PMC article.
Lipidomic Evaluation of Feline Neurologic Disease after AAV Gene Therapy.
Gray-Edwards HL, Jiang X, Randle AN, Taylor AR, Voss TL, Johnson AK, McCurdy VJ, Sena-Esteves M, Ory DS, Martin DR. Gray-Edwards HL, et al. Among authors: jiang x. Mol Ther Methods Clin Dev. 2017 Jul 26;6:135-142. doi: 10.1016/j.omtm.2017.07.005. eCollection 2017 Sep 15. Mol Ther Methods Clin Dev. 2017. PMID: 28808666 Free PMC article.
Neural stem cells for disease modeling and evaluation of therapeutics for Tay-Sachs disease.
Vu M, Li R, Baskfield A, Lu B, Farkhondeh A, Gorshkov K, Motabar O, Beers J, Chen G, Zou J, Espejo-Mojica AJ, Rodríguez-López A, Alméciga-Díaz CJ, Barrera LA, Jiang X, Ory DS, Marugan JJ, Zheng W. Vu M, et al. Among authors: jiang x. Orphanet J Rare Dis. 2018 Sep 17;13(1):152. doi: 10.1186/s13023-018-0886-3. Orphanet J Rare Dis. 2018. PMID: 30220252 Free PMC article.
A novel gene editing system to treat both Tay-Sachs and Sandhoff diseases.
Ou L, Przybilla MJ, Tăbăran AF, Overn P, O'Sullivan MG, Jiang X, Sidhu R, Kell PJ, Ory DS, Whitley CB. Ou L, et al. Among authors: jiang x. Gene Ther. 2020 May;27(5):226-236. doi: 10.1038/s41434-019-0120-5. Epub 2020 Jan 2. Gene Ther. 2020. PMID: 31896760 Free PMC article.
Krabbe disease successfully treated via monotherapy of intrathecal gene therapy.
Bradbury AM, Bagel JH, Nguyen D, Lykken EA, Pesayco Salvador J, Jiang X, Swain GP, Assenmacher CA, Hendricks IJ, Miyadera K, Hess RS, Ostrager A, ODonnell P, Sands MS, Ory DS, Shelton GD, Bongarzone ER, Gray SJ, Vite CH. Bradbury AM, et al. Among authors: jiang x. J Clin Invest. 2020 Sep 1;130(9):4906-4920. doi: 10.1172/JCI133953. J Clin Invest. 2020. PMID: 32773406 Free PMC article.
Natural history of Tay-Sachs disease in sheep.
Story B, Taghian T, Gallagher J, Koehler J, Taylor A, Randle A, Nielsen K, Gross A, Maguire A, Carl S, Johnson S, Fernau D, Diffie E, Cuddon P, Corado C, Chandra S, Sena-Esteves M, Kolodny E, Jiang X, Martin D, Gray-Edwards H. Story B, et al. Among authors: jiang x. Mol Genet Metab. 2021 Sep-Oct;134(1-2):164-174. doi: 10.1016/j.ymgme.2021.08.009. Epub 2021 Aug 21. Mol Genet Metab. 2021. PMID: 34456134 Free PMC article.
Glb1 knockout mouse model shares natural history with type II GM1 gangliosidosis patients.
Nicoli ER, Huebecker M, Han ST, Garcia K, Munasinghe J, Lizak M, Latour Y, Yoon R, Glase B, Tyrlik M, Peiravi M, Springer D, Baker EH, Priestman D, Sidhu R, Kell P, Jiang X, Kolstad J, Kuhn AL, Shazeeb MS, Acosta MT, Proia RL, Platt FM, Tifft CJ. Nicoli ER, et al. Among authors: jiang x. Mol Genet Metab. 2023 Feb;138(2):107508. doi: 10.1016/j.ymgme.2023.107508. Epub 2023 Jan 13. Mol Genet Metab. 2023. PMID: 36709532 Free PMC article.
A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis.
Kell P, Sidhu R, Qian M, Mishra S, Nicoli ER, D'Souza P, Tifft CJ, Gross AL, Gray-Edwards HL, Martin DR, Sena-Esteves M, Dietzen DJ, Singh M, Luo J, Schaffer JE, Ory DS, Jiang X. Kell P, et al. Among authors: jiang x. EBioMedicine. 2023 Jun;92:104627. doi: 10.1016/j.ebiom.2023.104627. Epub 2023 May 31. EBioMedicine. 2023. PMID: 37267847 Free PMC article.
Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 mice.
Davidson CD, Gibson AL, Gu T, Baxter LL, Deverman BE, Beadle K, Incao AA, Rodriguez-Gil JL, Fujiwara H, Jiang X, Chandler RJ, Ory DS, Gradinaru V, Venditti CP, Pavan WJ. Davidson CD, et al. Among authors: jiang x. Life Sci Alliance. 2021 Aug 18;4(10):e202101040. doi: 10.26508/lsa.202101040. Print 2021 Oct. Life Sci Alliance. 2021. PMID: 34407999 Free PMC article.
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