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 Custom Filters

Publication date

Text availability

Article attribute

Article type

Additional filters

Article Language

Species

Sex

Age

Other

Search Results

233 results

Filters applied: . Clear all
Results are displayed in a computed author sort order. The Publication Date timeline is not available.
Page 1
Progress and prospects: gene therapy clinical trials (part 2).
Aiuti A, Bachoud-Lévi AC, Blesch A, Brenner MK, Cattaneo F, Chiocca EA, Gao G, High KA, Leen AM, Lemoine NR, McNeish IA, Meneguzzi G, Peschanski M, Roncarolo MG, Strayer DS, Tuszynski MH, Waxman DJ, Wilson JM. Aiuti A, et al. Among authors: high ka. Gene Ther. 2007 Nov;14(22):1555-63. doi: 10.1038/sj.gt.3303033. Gene Ther. 2007. PMID: 17984995 Review.
Gene Therapy.
High KA, Roncarolo MG. High KA, et al. N Engl J Med. 2019 Aug 1;381(5):455-464. doi: 10.1056/NEJMra1706910. N Engl J Med. 2019. PMID: 31365802 Review. No abstract available.
Cardiac gene transfer of short hairpin RNA directed against phospholamban effectively knocks down gene expression but causes cellular toxicity in canines.
Bish LT, Sleeper MM, Reynolds C, Gazzara J, Withnall E, Singletary GE, Buchlis G, Hui D, High KA, Gao G, Wilson JM, Sweeney HL. Bish LT, et al. Among authors: high ka. Hum Gene Ther. 2011 Aug;22(8):969-77. doi: 10.1089/hum.2011.035. Epub 2011 Jun 8. Hum Gene Ther. 2011. PMID: 21542669 Free PMC article.
Perspectives on best practices for gene therapy programs.
Cheever TR, Berkley D, Braun S, Brown RH, Byrne BJ, Chamberlain JS, Cwik V, Duan D, Federoff HJ, High KA, Kaspar BK, Klinger KW, Larkindale J, Lincecum J, Mavilio F, McDonald CL, McLaughlin J, Weiss McLeod B, Mendell JR, Nuckolls G, Stedman HH, Tagle DA, Vandenberghe LH, Wang H, Wernett PJ, Wilson JM, Porter JD, Gubitz AK. Cheever TR, et al. Among authors: high ka. Hum Gene Ther. 2015 Mar;26(3):127-33. doi: 10.1089/hum.2014.147. Epub 2015 Mar 3. Hum Gene Ther. 2015. PMID: 25654329 Free PMC article.
Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012.
O'Reilly M, Kohn DB, Bartlett J, Benson J, Brooks PJ, Byrne BJ, Camozzi C, Cornetta K, Crystal RG, Fong Y, Gargiulo L, Gopal-Srivastava R, High KA, Jacobson SG, Jambou RC, Montgomery M, Rosenthal E, Samulski RJ, Skarlatos SI, Sorrentino B, Wilson JM, Xie Y, Corrigan-Curay J. O'Reilly M, et al. Among authors: high ka. Hum Gene Ther. 2013 Apr;24(4):355-62. doi: 10.1089/hum.2013.064. Hum Gene Ther. 2013. PMID: 23517518 Free PMC article.
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: high ka. 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.
American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells.
Kohn DB, Sadelain M, Dunbar C, Bodine D, Kiem HP, Candotti F, Tisdale J, Riviére I, Blau CA, Richard RE, Sorrentino B, Nolta J, Malech H, Brenner M, Cornetta K, Cavagnaro J, High K, Glorioso J; American Society of Gene Therapy (ASGT). Kohn DB, et al. Mol Ther. 2003 Aug;8(2):180-7. doi: 10.1016/s1525-0016(03)00212-0. Mol Ther. 2003. PMID: 12907140 Free article. Review. No abstract available.
Immune responses to AAV in clinical trials.
Mingozzi F, High KA. Mingozzi F, et al. Among authors: high ka. Curr Gene Ther. 2007 Oct;7(5):316-24. doi: 10.2174/156652307782151425. Curr Gene Ther. 2007. PMID: 17979678 Review.
Immune responses to AAV in clinical trials.
Mingozzi F, High KA. Mingozzi F, et al. Among authors: high ka. Curr Gene Ther. 2011 Aug;11(4):321-30. doi: 10.2174/156652311796150354. Curr Gene Ther. 2011. PMID: 21557723 Review.
233 results