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Gene therapy with AAV9-SGPL1 in an animal model of lung fibrosis.
Bhattacharyya A, Khan R, Lee JY, Tassew G, Oskouian B, Allende ML, Proia RL, Yin X, Ortega JG, Bhattacharya M, Saba JD. Bhattacharyya A, et al. J Pathol. 2024 May;263(1):22-31. doi: 10.1002/path.6256. Epub 2024 Feb 9. J Pathol. 2024. PMID: 38332723
Responsiveness of sphingosine phosphate lyase insufficiency syndrome to vitamin B6 cofactor supplementation.
Zhao P, Liu ID, Hodgin JB, Benke PI, Selva J, Torta F, Wenk MR, Endrizzi JA, West O, Ou W, Tang E, Goh DL, Tay SK, Yap HK, Loh A, Weaver N, Sullivan B, Larson A, Cooper MA, Alhasan K, Alangari AA, Salim S, Gumus E, Chen K, Zenker M, Hildebrandt F, Saba JD. Zhao P, et al. J Inherit Metab Dis. 2020 Sep;43(5):1131-1142. doi: 10.1002/jimd.12238. Epub 2020 May 4. J Inherit Metab Dis. 2020. PMID: 32233035 Free PMC article.
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