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Items: 1 to 50 of 217

1.

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
2.

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 Mar 29. doi: 10.1089/hum.2017.225. [Epub ahead of print]

PMID:
29597895
3.

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.

4.

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
5.

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
6.

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
7.

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
8.

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.

PMID:
29130351
9.

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.

PMID:
29116986
10.

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
11.

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
12.

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.

13.

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
14.

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
15.

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
16.

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
17.

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
18.

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
19.

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
20.

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.

21.

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.

22.

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.

23.
24.

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
25.

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.

26.

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
27.

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.

28.

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
29.

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
30.

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
31.

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
32.

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.

33.

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
34.

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
35.

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.

36.

Special Note from the Editor-in-Chief.

Flotte TR.

Hum Gene Ther. 2016 Feb;27(2):95. doi: 10.1089/hum.2016.29019.trf. No abstract available.

PMID:
26886829
37.

The Role of Patient Advocacy Organizations in Advancing Human Gene Therapy.

Flotte TR.

Hum Gene Ther. 2015 Dec;26(12):782. doi: 10.1089/hum.2015.29011.trf. No abstract available.

38.

Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?

Berns KI, Byrne BJ, Flotte TR, Gao G, Hauswirth WW, Herzog RW, Muzyczka N, VandenDriessche T, Xiao X, Zolotukhin S, Srivastava A.

Hum Gene Ther. 2015 Dec;26(12):779-81. doi: 10.1089/hum.2015.29014.kib. No abstract available.

39.

Recombinant Adeno-Associated Virus Vector Genomes Take the Form of Long-Lived, Transcriptionally Competent Episomes in Human Muscle.

Schnepp BC, Chulay JD, Ye GJ, Flotte TR, Trapnell BC, Johnson PR.

Hum Gene Ther. 2016 Jan;27(1):32-42. doi: 10.1089/hum.2015.136.

40.

Transcription Activator-Like Nucleases Enable Allogeneic Chimeric Antigen Receptor-T Cell Therapy in Humans.

Flotte TR.

Hum Gene Ther. 2015 Nov;26(11):iv. doi: 10.1089/hum.2015.29013.trf. No abstract available.

PMID:
26565678
41.

Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors.

Li S, Ling C, Zhong L, Li M, Su Q, He R, Tang Q, Greiner DL, Shultz LD, Brehm MA, Flotte TR, Mueller C, Srivastava A, Gao G.

Mol Ther. 2015 Dec;23(12):1867-76. doi: 10.1038/mt.2015.174. Epub 2015 Sep 25.

42.

Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods.

Gruntman AM, Flotte TR.

Hum Gene Ther Clin Dev. 2015 Sep;26(3):159-64. doi: 10.1089/humc.2015.116. Epub 2015 Sep 10. Review.

43.

Ethical Implications of the Cost of Molecularly Targeted Therapies.

Flotte TR.

Hum Gene Ther. 2015 Sep;26(9):573-4. doi: 10.1089/hum.2015.29010.trf. No abstract available.

PMID:
26355414
44.

The End of the Beginning of Gene Therapy.

Flotte TR.

Hum Gene Ther. 2015 Jul;26(7):407-8. doi: 10.1089/hum.2015.29003.trf. No abstract available.

PMID:
26176429
45.

Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency.

Gruntman AM, Flotte TR.

Hum Gene Ther Methods. 2015 Jun;26(3):77-81. doi: 10.1089/hgtb.2015.086. Review.

46.

Therapeutic Germ Line Alteration: Has CRISPR/Cas9 Technology Forced the Question?

Flotte TR.

Hum Gene Ther. 2015 May;26(5):245-6. doi: 10.1089/hum.2015.28999.tfl. Epub 2015 Apr 30. No abstract available.

47.

Stability and compatibility of recombinant adeno-associated virus under conditions commonly encountered in human gene therapy trials.

Gruntman AM, Su L, Su Q, Gao G, Mueller C, Flotte TR.

Hum Gene Ther Methods. 2015 Apr;26(2):71-6. doi: 10.1089/hgtb.2015.040.

48.

Current status of gene therapy for α-1 antitrypsin deficiency.

Loring HS, Flotte TR.

Expert Opin Biol Ther. 2015 Mar;15(3):329-36. doi: 10.1517/14712598.2015.978854. Epub 2014 Nov 3. Review.

PMID:
25363251
49.

Charting a clear path: the ASGCT Standardized Pathways Conference.

Kiem HP, Baum C, Bushman FD, Byrne BJ, Carter BJ, Cavagnaro J, Malech HL, Mendell JR, Naldini LM, Sorrentino BP, Williams DA, Flotte TR.

Mol Ther. 2014 Jul;22(7):1235-1238. doi: 10.1038/mt.2014.95. No abstract available.

50.

What is suppression of anti-adeno-associated virus capsid T-cells achieving?

Flotte TR, Mueller C.

Hum Gene Ther. 2014 Mar;25(3):178-9. doi: 10.1089/hum.2014.2506. No abstract available.

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