Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation

Search Page

Filters

My NCBI Filters

Text availability

Article attribute

Article type

Publication date

Search Results

171 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Results By Year timeline is not available.
Page 1
The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins.
Paul-Smith MC, Pytel KM, Gelinas JF, McIntosh J, Pringle I, Davies L, Chan M, Meng C, Bell R, Cammack L, Moran C, Cameron L, Inoue M, Tsugumine S, Hironaka T, Gill DR, Hyde SC, Nathwani A, Alton EWFW, Griesenbach U. Paul-Smith MC, et al. Among authors: nathwani a. Gene Ther. 2018 Aug;25(5):345-358. doi: 10.1038/s41434-018-0025-8. Epub 2018 Jul 18. Gene Ther. 2018. PMID: 30022127 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: nathwani a. 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.
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease.
Jeyakumar JM, Kia A, Tam LCS, McIntosh J, Spiewak J, Mills K, Heywood W, Chisari E, Castaldo N, Verhoef D, Hosseini P, Kalcheva P, Cocita C, Miranda CJ, Canavese M, Khinder J, Rosales C, Hughes D, Sheridan R, Corbau R, Nathwani A. Jeyakumar JM, et al. Among authors: nathwani a. Gene Ther. 2023 Jun;30(6):487-502. doi: 10.1038/s41434-022-00381-y. Epub 2023 Jan 11. Gene Ther. 2023. PMID: 36631545 Free PMC article.
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: nathwani a. Sci Transl Med. 2023 Aug 9;15(708):eabc8226. doi: 10.1126/scitranslmed.abc8226. Epub 2023 Aug 9. Sci Transl Med. 2023. PMID: 37556557
Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.
Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G. Tremblay JP, et al. Among authors: nathwani a. Mol Ther. 2013 Feb;21(2):266-8. doi: 10.1038/mt.2013.4. Mol Ther. 2013. PMID: 23369965 Free PMC article. No abstract available.
171 results