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Items: 1 to 20 of 234

1.

Getting Tough on Capsid Screening: Tough Decoys Enable Barcoding of Vectors Capable of both Entry and Expression.

Flotte TR.

Hum Gene Ther. 2019 Aug;30(8):919-920. doi: 10.1089/hum.2019.29090.trf. No abstract available.

PMID:
31418605
2.

Senior Gene Therapy Scientists Take a Stand Against Human Embryo Gene Editing.

Flotte TR.

Hum Gene Ther. 2019 Jul;30(7):793. doi: 10.1089/hum.2019.29088.trf. No abstract available.

PMID:
31334692
3.

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

Keeler AM, Flotte TR.

Annu Rev Virol. 2019 Jul 5. doi: 10.1146/annurev-virology-092818-015530. [Epub ahead of print]

PMID:
31283441
4.

AAV9 gene replacement therapy for respiratory insufficiency in very-long chain acyl-CoA dehydrogenase deficiency.

Zieger M, Keeler AM, Flotte TR, ElMallah MK.

J Inherit Metab Dis. 2019 Sep;42(5):870-877. doi: 10.1002/jimd.12101. Epub 2019 May 3.

PMID:
30993714
5.

New Horizons for Immune Gene Therapy.

Barzel A, Flotte TR.

Hum Gene Ther. 2019 Apr;30(4):379-380. doi: 10.1089/hum.2019.29085.aba. No abstract available.

PMID:
30950655
6.

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.

Mol Ther Methods Clin Dev. 2019 Feb 2;13:233-242. doi: 10.1016/j.omtm.2019.01.013. eCollection 2019 Jun 14.

7.

The Year in Review: The Top Five Papers of 2018.

Flotte TR.

Hum Gene Ther. 2018 Dec;29(12):1339-1340. doi: 10.1089/hum.2018.29079.trf. No abstract available.

PMID:
30757924
8.

What the Gene Therapy Community Should Do About Sexual Harassment.

Adair JE, Flotte TR.

Hum Gene Ther. 2019 Mar;30(3):249-251. doi: 10.1089/hum.2019.028. No abstract available.

PMID:
30755038
9.

Top Five Gene Therapy Stories of 2019.

Flotte TR.

Hum Gene Ther. 2019 Jan;30(1):1-2. doi: 10.1089/hum.2018.29080.trf. No abstract available.

PMID:
30628861
10.

Assuring Integrity in the Residency Match Process.

Chimienti SN, DeMarco DM, Flotte TR, Collins MF.

Acad Med. 2019 Mar;94(3):321-323. doi: 10.1097/ACM.0000000000002531.

PMID:
30431456
11.

Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer.

Wang D, Zhong L, Li M, Li J, Tran K, Ren L, He R, Xie J, Moser RP, Fraser C, Kuchel T, Sena-Esteves M, Flotte TR, Aronin N, Gao G.

Mol Ther Methods Clin Dev. 2018 Oct 4;11:65-72. doi: 10.1016/j.omtm.2018.09.003. eCollection 2018 Dec 14.

12.

DNA Vaccination in 2018: An Update.

Flotte TR, Lu S.

Hum Gene Ther. 2018 Sep;29(9):963-965. doi: 10.1089/hum.2018.29073.trf. No abstract available.

PMID:
30230936
13.

Alpha-1 Antitrypsin Deficiency as a Candidate for Gene Editing.

Flotte TR.

Hum Gene Ther. 2018 Aug;29(8):843-844. doi: 10.1089/hum.2018.29070.trf. No abstract available.

PMID:
30096037
14.

Airway Basal Cells Are the Key for Cystic Fibrosis Gene Therapy.

Flotte TR.

Hum Gene Ther. 2018 Jun;29(6):641-642. doi: 10.1089/hum.2018.29066.trf. No abstract available.

PMID:
29902084
15.

Results at 5 Years After Gene Therapy for RPE65-Deficient Retinal Dystrophy.

Pennesi ME, Weleber RG, Yang P, Whitebirch C, Thean B, Flotte TR, Humphries M, Chegarnov E, Beasley KN, Stout JT, Chulay JD.

Hum Gene Ther. 2018 Jul 24. doi: 10.1089/hum.2018.014. [Epub ahead of print]

PMID:
29869534
16.

A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates.

Wang D, Li S, Gessler DJ, Xie J, Zhong L, Li J, Tran K, Van Vliet K, Ren L, Su Q, He R, Goetzmann JE, Flotte TR, Agbandje-McKenna M, Gao G.

Mol Ther Methods Clin Dev. 2018 Mar 16;9:234-246. doi: 10.1016/j.omtm.2018.03.004. eCollection 2018 Jun 15.

17.

Recombinant Adeno-Associated Virus-Based Gene Therapy for Disorders Detected by Newborn Screening: Inherent Limitations of This Approach.

Flotte TR.

Hum Gene Ther. 2018 Apr;29(4):401-402. doi: 10.1089/hum.2018.29064.trf. No abstract available.

PMID:
29668376
18.

The rapidly evolving state of gene therapy.

Gruntman AM, Flotte TR.

FASEB J. 2018 Apr;32(4):1733-1740. doi: 10.1096/fj.201700982R. Review.

PMID:
31282760
19.

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.

Hum Gene Ther. 2018 Aug;29(8):853-860. doi: 10.1089/hum.2017.225. Epub 2018 May 14.

20.

Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema.

Borel F, Sun H, Zieger M, Cox A, Cardozo B, Li W, Oliveira G, Davis A, Gruntman A, Flotte TR, Brodsky MH, Hoffman AM, Elmallah MK, Mueller C.

Proc Natl Acad Sci U S A. 2018 Mar 13;115(11):2788-2793. doi: 10.1073/pnas.1713689115. Epub 2018 Feb 16.

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