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

22 results

Filters applied: . Clear all
Page 1
Large-scale molecular epidemiological analysis of AAV in a cancer patient population.
Qin W, Xu G, Tai PWL, Wang C, Luo L, Li C, Hu X, Xue J, Lu Y, Zhou Q, Wei Q, Wen T, Hu J, Xiao Y, Yang L, Li W, Flotte TR, Wei Y, Gao G. Qin W, et al. Among authors: flotte tr. Oncogene. 2021 Apr;40(17):3060-3071. doi: 10.1038/s41388-021-01725-5. Epub 2021 Mar 29. Oncogene. 2021. PMID: 33782545 Free PMC article.
However, previous reports have suggested that wild-type AAV is linked to hepatocellular carcinoma, raising concern with the safety of rAAVs. In addition, a recent long-term follow-up study in canines, which received rAAVs for factor VIII gene therapy, demonstrated vector i …
However, previous reports have suggested that wild-type AAV is linked to hepatocellular carcinoma, raising concern with the safety of rAAVs. …
Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study.
Gruntman AM, Gernoux G, Tang Q, Ye GJ, Knop DR, Wang G, Benson J, Coleman KE, Keeler AM, Mueller C, Chicoine LG, Chulay JD, Flotte TR. Gruntman AM, et al. Among authors: flotte tr. Mol Ther Methods Clin Dev. 2019 Feb 2;13:233-242. doi: 10.1016/j.omtm.2019.01.013. eCollection 2019 Jun 14. Mol Ther Methods Clin Dev. 2019. PMID: 30828586 Free PMC article.
Phase 1 and phase 2 gene therapy trials using intramuscular (IM) administration of a recombinant adeno-associated virus serotype 1 (rAAV1) for replacement of serum alpha-1 antitrypsin (AAT) deficiency have shown long-term (5-year) stable transgene expression at approximate …
Phase 1 and phase 2 gene therapy trials using intramuscular (IM) administration of a recombinant adeno-associated virus serotype 1 (rAAV1) f …
In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency.
Song CQ, Wang D, Jiang T, O'Connor K, Tang Q, Cai L, Li X, Weng Z, Yin H, Gao G, Mueller C, Flotte TR, Xue W. Song CQ, et al. Among authors: flotte tr. Hum Gene Ther. 2018 Aug;29(8):853-860. doi: 10.1089/hum.2017.225. Epub 2018 May 14. Hum Gene Ther. 2018. PMID: 29597895 Free PMC article.
CRISPR (clustered regularly interspaced short palindromic repeats) genome editing holds promise in the treatment of genetic diseases that currently lack effective long-term therapies. Patients with alpha-1 antitrypsin (AAT) deficiency develop progressive lung disease due t …
CRISPR (clustered regularly interspaced short palindromic repeats) genome editing holds promise in the treatment of genetic diseases that cu …
Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression.
Mueller C, Chulay JD, Trapnell BC, Humphries M, Carey B, Sandhaus RA, McElvaney NG, Messina L, Tang Q, Rouhani FN, Campbell-Thompson M, Fu AD, Yachnis A, Knop DR, Ye GJ, Brantly M, Calcedo R, Somanathan S, Richman LP, Vonderheide RH, Hulme MA, Brusko TM, Wilson JM, Flotte TR. Mueller C, et al. Among authors: flotte tr. J Clin Invest. 2013 Dec;123(12):5310-8. doi: 10.1172/JCI70314. Epub 2013 Nov 15. J Clin Invest. 2013. PMID: 24231351 Free PMC article. Clinical Trial.
To determine whether rAAV vector expression can persist long term, we administered rAAV vectors expressing normal, M-type alpha-1 antitrypsin (M-AAT) to AAT-deficient subjects at various doses by multiple i.m. injections. ...
To determine whether rAAV vector expression can persist long term, we administered rAAV vectors expressing normal, M-type alpha-1 ant …
Long-term, efficient inhibition of microRNA function in mice using rAAV vectors.
Xie J, Ameres SL, Friedline R, Hung JH, Zhang Y, Xie Q, Zhong L, Su Q, He R, Li M, Li H, Mu X, Zhang H, Broderick JA, Kim JK, Weng Z, Flotte TR, Zamore PD, Gao G. Xie J, et al. Among authors: flotte tr. Nat Methods. 2012 Mar 4;9(4):403-9. doi: 10.1038/nmeth.1903. Nat Methods. 2012. PMID: 22388288 Free PMC article.
No immune responses by the expression of the yeast Ndi1 protein in rats.
Marella M, Seo BB, Flotte TR, Matsuno-Yagi A, Yagi T. Marella M, et al. Among authors: flotte tr. PLoS One. 2011;6(10):e25910. doi: 10.1371/journal.pone.0025910. Epub 2011 Oct 3. PLoS One. 2011. PMID: 21991386 Free PMC article.
In the so-called transkingdom gene therapy, one major concern is the fact that the yeast protein is foreign in mammals. Long term expression of Ndi1 observed in rodents with no apparent damage to the target tissue was indicative of no action by the host's immune system. .. …
In the so-called transkingdom gene therapy, one major concern is the fact that the yeast protein is foreign in mammals. Long term exp …
Recombinant adeno-associated virus-mediated gene transfer for the potential therapy of adenosine deaminase-deficient severe combined immune deficiency.
Silver JN, Elder M, Conlon T, Cruz P, Wright AJ, Srivastava A, Flotte TR. Silver JN, et al. Among authors: flotte tr. Hum Gene Ther. 2011 Aug;22(8):935-49. doi: 10.1089/hum.2010.121. Epub 2011 Mar 13. Hum Gene Ther. 2011. PMID: 21142972 Free PMC article.
Alternatively, recombinant adeno-associated virus (rAAV) vectors have gained attention as valuable tools for gene transfer, having demonstrated no pathogenicity in humans, minimal immunogenicity, long-term efficacy, ease of administration, and broad tissue tropism (Muzyczk …
Alternatively, recombinant adeno-associated virus (rAAV) vectors have gained attention as valuable tools for gene transfer, having demonstra …
Endocrine parameters of cystic fibrosis: back to basics.
Stalvey MS, Flotte TR. Stalvey MS, et al. Among authors: flotte tr. J Cell Biochem. 2009 Oct 1;108(2):353-61. doi: 10.1002/jcb.22284. J Cell Biochem. 2009. PMID: 19670266 Review.
Dramatic changes in the life expectancy of cystic fibrosis (CF) patients are occurring, creating a cohort of aging individuals experiencing long-term complications of this chronic disease. The two most common of these complications include CF-related diabetes and CF bone d …
Dramatic changes in the life expectancy of cystic fibrosis (CF) patients are occurring, creating a cohort of aging individuals experiencing …
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.
Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, Wang L, Conlon TJ, Boye SL, Flotte TR, Byrne BJ, Jacobson SG. Hauswirth WW, et al. Among authors: flotte tr. Hum Gene Ther. 2008 Oct;19(10):979-90. doi: 10.1089/hum.2008.107. Hum Gene Ther. 2008. PMID: 18774912 Free PMC article. Clinical Trial.
22 results