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Year | Number of Results |
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2022 | 2 |
2023 | 2 |
2024 | 0 |
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Page 1
Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies.
J Clin Invest. 2023 May 15;133(10):e164575. doi: 10.1172/JCI164575.
J Clin Invest. 2023.
PMID: 36951961
Free PMC article.
AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease.
Chen X, Dong T, Hu Y, Shaffo FC, Belur NR, Mazzulli JR, Gray SJ.
Chen X, et al. Among authors: dong t.
J Clin Invest. 2022 Mar 1;132(5):e146286. doi: 10.1172/JCI146286.
J Clin Invest. 2022.
PMID: 35025759
Free PMC article.
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SMRT Sequencing Enables High-Throughput Identification of Novel AAVs from Capsid Shuffling and Directed Evolution.
Casy W, Garza IT, Chen X, Dong T, Hu Y, Kanchwala M, Trygg CB, Shyng C, Xing C, Bunnell BA, Braun SE, Gray SJ.
Casy W, et al. Among authors: dong t.
Genes (Basel). 2023 Aug 21;14(8):1660. doi: 10.3390/genes14081660.
Genes (Basel). 2023.
PMID: 37628711
Free PMC article.
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Long-term progression of retinal degeneration in a preclinical model of CLN7 Batten disease as a baseline for testing clinical therapeutics.
Rowe AA, Chen X, Nettesheim ER, Issioui Y, Dong T, Hu Y, Messahel S, Kayani SN, Gray SJ, Wert KJ.
Rowe AA, et al. Among authors: dong t.
EBioMedicine. 2022 Nov;85:104314. doi: 10.1016/j.ebiom.2022.104314. Epub 2022 Oct 29.
EBioMedicine. 2022.
PMID: 36374771
Free PMC article.
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