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Items: 1 to 50 of 232

1.

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
2.

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 Apr 17. doi: 10.1002/jimd.12101. [Epub ahead of print]

PMID:
30993714
3.

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
4.

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.

5.

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
6.

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
7.

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
8.

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
9.

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.

10.

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
11.

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
12.

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
13.

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
14.

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.

15.

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
16.

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
17.

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.

PMID:
29597895
18.

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.

19.

Gene and Cell Therapy in China: Highlighting Excellence in the 21st Century : 21.

Flotte TR, Gao 高光坪 G.

Hum Gene Ther. 2018 Feb;29(2):97. doi: 10.1089/hum.2018.29062.trf. No abstract available.

PMID:
29446999
20.

Severe Toxicity in Nonhuman Primates and Piglets with Systemic High-Dose Administration of Adeno-Associated Virus Serotype 9-Like Vectors: Putting Patients First.

Flotte TR, Büning H.

Hum Gene Ther. 2018 Mar;29(3):283-284. doi: 10.1089/hum.2018.021. Epub 2018 Feb 7. No abstract available.

PMID:
29378415
21.

Gene and Cell Therapy in 2018: A Look Ahead.

Flotte TR.

Hum Gene Ther. 2018 Jan;29(1):1. doi: 10.1089/hum.2017.29060.trf. No abstract available.

PMID:
29328843
22.

Therapeutic Advances in Germany and Beyond.

Flotte TR.

Hum Gene Ther. 2017 Dec;28(12):1117. doi: 10.1089/hum.2017.29059.trf. No abstract available.

PMID:
29265969
23.

The Gene Therapy Resource Program: A Decade of Dedication to Translational Research by the National Heart, Lung, and Blood Institute.

Flotte TR, Daniels E, Benson J, Bevett-Rose JM, Cornetta K, Diggins M, Johnston J, Sepelak S, van der Loo JCM, Wilson JM, McDonald CL.

Hum Gene Ther Clin Dev. 2017 Dec;28(4):178-186. doi: 10.1089/humc.2017.170. Epub 2017 Nov 27.

24.

It Is Time for Zero Tolerance for Sexual Harassment in Academic Medicine.

Bates CK, Jagsi R, Gordon LK, Travis E, Chatterjee A, Gillis M, Means O, Chaudron L, Ganetzky R, Gulati M, Fivush B, Sharma P, Grover A, Lautenberger D, Flotte TR.

Acad Med. 2018 Feb;93(2):163-165. doi: 10.1097/ACM.0000000000002050.

25.

The Renaissance of Gene and Cell Therapy: Florence 2016.

Naldini L, Parker GC, Flotte TR.

Hum Gene Ther. 2016 Oct;27(10):727-728. doi: 10.1089/hum.2016.29034.lna. No abstract available.

PMID:
29112477
26.

European Society of Gene and Cell Therapy (ESGCT) at 25: A Gene Therapy Community at Its Prime and on the Move.

Flotte TR.

Hum Gene Ther. 2017 Nov;28(11):940. doi: 10.1089/hum.2017.29049.trf. No abstract available.

PMID:
29035113
27.

Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency.

Borel F, Tang Q, Gernoux G, Greer C, Wang Z, Barzel A, Kay MA, Shultz LD, Greiner DL, Flotte TR, Brehm MA, Mueller C.

Mol Ther. 2017 Nov 1;25(11):2477-2489. doi: 10.1016/j.ymthe.2017.09.020. Epub 2017 Sep 25.

28.

Gene Therapy for Alcoholism and Other Substance Use Disorders.

Flotte TR.

Hum Gene Ther. 2017 Sep;28(9):701-702. doi: 10.1089/hum.2017.29048.trf. No abstract available.

PMID:
28854046
29.

Adeno-Associated Virus-Human Bocavirus 1 Chimeric Vectors: Ferreting Out Their Role in Airway Gene Therapy.

Flotte TR.

Hum Gene Ther. 2017 Aug;28(8):611. doi: 10.1089/hum.2017.29047.trf. No abstract available.

PMID:
28796554
30.

Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency.

Gruntman AM, Flotte TR.

Methods Mol Biol. 2017;1639:267-275. doi: 10.1007/978-1-4939-7163-3_27.

PMID:
28752467
31.

Quantification of Total Human Alpha-1 Antitrypsin by Sandwich ELISA.

Tang Q, Gruntman AM, Flotte TR.

Methods Mol Biol. 2017;1639:211-216. doi: 10.1007/978-1-4939-7163-3_20.

PMID:
28752460
32.

Sleeping Beauty Awakens New Interest.

Flotte TR.

Hum Gene Ther. 2017 Jul;28(7):533. doi: 10.1089/hum.2017.29046.trf. No abstract available.

PMID:
28719250
33.

One More Controversy: Adeno-Associated Virus in Stem Cells.

Flotte TR.

Hum Gene Ther. 2017 Jun;28(6):449. doi: 10.1089/hum.2017.29043.trf. No abstract available.

PMID:
28604173
34.

AAV Is Now a Medicine: We Had Better Get This Right.

Flotte TR, Gao G.

Hum Gene Ther. 2017 Apr;28(4):307. doi: 10.1089/hum.2017.29041.trf. No abstract available.

PMID:
28414552
35.

5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.

Mueller C, Gernoux G, Gruntman AM, Borel F, Reeves EP, Calcedo R, Rouhani FN, Yachnis A, Humphries M, Campbell-Thompson M, Messina L, Chulay JD, Trapnell B, Wilson JM, McElvaney NG, Flotte TR.

Mol Ther. 2017 Jun 7;25(6):1387-1394. doi: 10.1016/j.ymthe.2017.03.029. Epub 2017 Apr 10.

36.

Gene Therapy 2017: Progress and Future Directions.

Keeler AM, ElMallah MK, Flotte TR.

Clin Transl Sci. 2017 Jul;10(4):242-248. doi: 10.1111/cts.12466. Epub 2017 May 23. Review. No abstract available.

37.

CAR T-Cell Therapy: Progress and Prospects.

Wilkins O, Keeler AM, Flotte TR.

Hum Gene Ther Methods. 2017 Apr;28(2):61-66. doi: 10.1089/hgtb.2016.153. Review.

38.
39.

The Negative Effects of Immigration Restrictions on the Gene Therapy Community.

Flotte TR.

Hum Gene Ther. 2017 Mar;28(3):227. doi: 10.1089/hum.2017.29040.trf. No abstract available.

PMID:
28319443
40.

Restrictive Lung Disease in the Cu/Zn Superoxide-Dismutase 1 G93A Amyotrophic Lateral Sclerosis Mouse Model.

Stoica L, Keeler AM, Xiong L, Kalfopoulos M, Desrochers K, Brown RH Jr, Sena-Esteves M, Flotte TR, ElMallah MK.

Am J Respir Cell Mol Biol. 2017 Mar;56(3):405-408. doi: 10.1165/rcmb.2016-0258LE. No abstract available.

41.

Empty Adeno-Associated Virus Capsids: Contaminant or Natural Decoy?

Flotte TR.

Hum Gene Ther. 2017 Feb;28(2):147-148. doi: 10.1089/hum.2017.29039.trf. No abstract available.

PMID:
28186848
42.

Class I-restricted T-cell responses to a polymorphic peptide in a gene therapy clinical trial for α-1-antitrypsin deficiency.

Calcedo R, Somanathan S, Qin Q, Betts MR, Rech AJ, Vonderheide RH, Mueller C, Flotte TR, Wilson JM.

Proc Natl Acad Sci U S A. 2017 Feb 14;114(7):1655-1659. doi: 10.1073/pnas.1617726114. Epub 2017 Jan 30.

43.

The Science Policy Implications of a Trump Presidency.

Flotte TR.

Hum Gene Ther. 2017 Jan;28(1):1-2. doi: 10.1089/hum.2016.29037.trf. Epub 2016 Dec 6. No abstract available.

PMID:
27922749
44.

Developing Core Competencies for the Prevention and Management of Prescription Drug Misuse: A Medical Education Collaboration in Massachusetts.

Antman KH, Berman HA, Flotte TR, Flier J, Dimitri DM, Bharel M.

Acad Med. 2016 Oct;91(10):1348-1351.

PMID:
27532868
45.

Gene Drives: Biological Shield or Ecological Menace?

Flotte TR.

Hum Gene Ther. 2016 Aug;27(8):561-2. doi: 10.1089/hum.2016.29032.trf. No abstract available.

PMID:
27513596
46.

The Target's the Thing.

Flotte TR.

Hum Gene Ther. 2016 Jul;27(7):477. doi: 10.1089/hum.2016.29030.trf. No abstract available.

PMID:
27428657
47.

Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy.

Weleber RG, Pennesi ME, Wilson DJ, Kaushal S, Erker LR, Jensen L, McBride MT, Flotte TR, Humphries M, Calcedo R, Hauswirth WW, Chulay JD, Stout JT.

Ophthalmology. 2016 Jul;123(7):1606-20. doi: 10.1016/j.ophtha.2016.03.003. Epub 2016 Apr 19.

48.

Why Human Gene Therapy Scientists Should Care About Model Organisms.

Flotte TR.

Hum Gene Ther. 2016 May;27(5):339-40. doi: 10.1089/hum.2016.29026.trf. No abstract available.

PMID:
27082033
49.

A Gene Therapy Scientist's Life Well-Lived.

Flotte TR.

Hum Gene Ther. 2016 Apr;27(4):279. doi: 10.1089/hum.2016.29023.trf. No abstract available.

PMID:
27074150
50.

Development of rAAV2-CFTR: History of the First rAAV Vector Product to be Used in Humans.

Loring HS, ElMallah MK, Flotte TR.

Hum Gene Ther Methods. 2016 Apr;27(2):49-58. doi: 10.1089/hgtb.2015.150. Epub 2016 Feb 19. Review.

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