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Adeno-associated virus gene therapy prevents progression of kidney disease in genetic models of nephrotic syndrome.
Ding WY, Kuzmuk V, Hunter S, Lay A, Hayes B, Beesley M, Rollason R, Hurcombe JA, Barrington F, Masson C, Cathery W, May C, Tuffin J, Roberts T, Mollet G, Chu CJ, McIntosh J, Coward RJ, Antignac C, Nathwani A, Welsh GI, Saleem MA. Ding WY, et al. Among authors: mcintosh j. Sci Transl Med. 2023 Aug 9;15(708):eabc8226. doi: 10.1126/scitranslmed.abc8226. Epub 2023 Aug 9. Sci Transl Med. 2023. PMID: 37556557
An update on gene therapy for hemophilia.
Nathwani AC, McIntosh J, Davidoff AM. Nathwani AC, et al. Among authors: mcintosh j. Curr Hematol Rep. 2005 Jul;4(4):287-93. Curr Hematol Rep. 2005. PMID: 16009043
Liver Gene Therapy.
Nathwani AC, McIntosh J, Sheridan R. Nathwani AC, et al. Among authors: mcintosh j. Hum Gene Ther. 2022 Sep;33(17-18):879-888. doi: 10.1089/hum.2022.169. Hum Gene Ther. 2022. PMID: 36082993 Review.
Enhancing transduction of the liver by adeno-associated viral vectors.
Nathwani AC, Cochrane M, McIntosh J, Ng CY, Zhou J, Gray JT, Davidoff AM. Nathwani AC, et al. Among authors: mcintosh j. Gene Ther. 2009 Jan;16(1):60-9. doi: 10.1038/gt.2008.137. Epub 2008 Aug 14. Gene Ther. 2009. PMID: 18701909 Free PMC article.
Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors.
Tan MH, Smith AJ, Pawlyk B, Xu X, Liu X, Bainbridge JB, Basche M, McIntosh J, Tran HV, Nathwani A, Li T, Ali RR. Tan MH, et al. Among authors: mcintosh j. Hum Mol Genet. 2009 Jun 15;18(12):2099-114. doi: 10.1093/hmg/ddp133. Epub 2009 Mar 19. Hum Mol Genet. 2009. PMID: 19299492 Free PMC article.
Preliminary evaluation of a self-complementary AAV2/8 vector for hepatic gene transfer of human apoE3 to inhibit atherosclerotic lesion development in apoE-deficient mice.
Osman E, Evans V, Graham IR, Athanasopoulos T, McIntosh J, Nathwani AC, Simons JP, Dickson G, Owen JS. Osman E, et al. Among authors: mcintosh j. Atherosclerosis. 2009 May;204(1):121-6. doi: 10.1016/j.atherosclerosis.2008.08.043. Epub 2008 Sep 11. Atherosclerosis. 2009. PMID: 18930458
1,275 results