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Items: 1 to 20 of 44

1.

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.

Biffi A, Montini E, Lorioli L, Cesani M, Fumagalli F, Plati T, Baldoli C, Martino S, Calabria A, Canale S, Benedicenti F, Vallanti G, Biasco L, Leo S, Kabbara N, Zanetti G, Rizzo WB, Mehta NA, Cicalese MP, Casiraghi M, Boelens JJ, Del Carro U, Dow DJ, Schmidt M, Assanelli A, Neduva V, Di Serio C, Stupka E, Gardner J, von Kalle C, Bordignon C, Ciceri F, Rovelli A, Roncarolo MG, Aiuti A, Sessa M, Naldini L.

Science. 2013 Aug 23;341(6148):1233158. doi: 10.1126/science.1233158. Epub 2013 Jul 11.

2.

Development of gene therapy for blood disorders: an update.

Nienhuis AW.

Blood. 2013 Aug 29;122(9):1556-64. doi: 10.1182/blood-2013-04-453209. Epub 2013 Jul 10. Review.

3.

Lessons learned from the clinical development and market authorization of Glybera.

Bryant LM, Christopher DM, Giles AR, Hinderer C, Rodriguez JL, Smith JB, Traxler EA, Tycko J, Wojno AP, Wilson JM.

Hum Gene Ther Clin Dev. 2013 Jun;24(2):55-64. doi: 10.1089/humc.2013.087. Epub 2013 Jun 29. Review. No abstract available.

4.

History of gene therapy.

Wirth T, Parker N, Ylä-Herttuala S.

Gene. 2013 Aug 10;525(2):162-9. doi: 10.1016/j.gene.2013.03.137. Epub 2013 Apr 23. Review.

PMID:
23618815
5.

Persistence, localization, and external control of transgene expression after single injection of adeno-associated virus into injured joints.

Lee HH, O'Malley MJ, Friel NA, Payne KA, Qiao C, Xiao X, Chu CR.

Hum Gene Ther. 2013 Apr;24(4):457-66. doi: 10.1089/hum.2012.118.

6.

Viral vectors: a look back and ahead on gene transfer technology.

Vannucci L, Lai M, Chiuppesi F, Ceccherini-Nelli L, Pistello M.

New Microbiol. 2013 Jan;36(1):1-22. Epub 2013 Jan 1. Review.

7.

Gene therapy clinical trials worldwide to 2012 - an update.

Ginn SL, Alexander IE, Edelstein ML, Abedi MR, Wixon J.

J Gene Med. 2013 Feb;15(2):65-77. doi: 10.1002/jgm.2698. Review.

PMID:
23355455
8.

Gene therapy enters the pharma market: the short story of a long journey.

Büning H.

EMBO Mol Med. 2013 Jan;5(1):1-3. doi: 10.1002/emmm.201202291. No abstract available.

9.

Convergence of gene and cell therapy.

Bersenev A, Levine BL.

Regen Med. 2012 Nov;7(6 Suppl):50-6. doi: 10.2217/rme.12.71. Review.

PMID:
23210811
10.

Gene therapy to be authorised for first time in EU.

Watts G.

BMJ. 2012 Jul 23;345:e5009. doi: 10.1136/bmj.e5009. No abstract available.

PMID:
22826599
11.

Gene therapy matures in the clinic.

Seymour LW, Thrasher AJ.

Nat Biotechnol. 2012 Jul 10;30(7):588-93. doi: 10.1038/nbt.2290. No abstract available.

PMID:
22781675
12.

Gene therapy for primary immunodeficiency.

Booth C, Gaspar HB, Thrasher AJ.

Curr Opin Pediatr. 2011 Dec;23(6):659-66. doi: 10.1097/MOP.0b013e32834cd67a. Review.

PMID:
21970832
13.

Considerations for the clinical application of chimeric antigen receptor T cells: observations from a recombinant DNA Advisory Committee Symposium held June 15, 2010.

Ertl HC, Zaia J, Rosenberg SA, June CH, Dotti G, Kahn J, Cooper LJ, Corrigan-Curay J, Strome SE.

Cancer Res. 2011 May 1;71(9):3175-81. doi: 10.1158/0008-5472.CAN-10-4035. Erratum in: Cancer Res. 2011 Jun 15;71(12):4325.

14.

Progress and prospects: hurdles to cardiovascular gene therapy clinical trials.

Hedman M, Hartikainen J, Ylä-Herttuala S.

Gene Ther. 2011 Aug;18(8):743-9. doi: 10.1038/gt.2011.43. Epub 2011 Apr 14. Review.

PMID:
21490683
15.

State-of-the-art gene-based therapies: the road ahead.

Kay MA.

Nat Rev Genet. 2011 May;12(5):316-28. doi: 10.1038/nrg2971. Epub 2011 Apr 6. Review.

PMID:
21468099
16.

Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

Nathwani AC, Rosales C, McIntosh J, Rastegarlari G, Nathwani D, Raj D, Nawathe S, Waddington SN, Bronson R, Jackson S, Donahue RE, High KA, Mingozzi F, Ng CY, Zhou J, Spence Y, McCarville MB, Valentine M, Allay J, Coleman J, Sleep S, Gray JT, Nienhuis AW, Davidoff AM.

Mol Ther. 2011 May;19(5):876-85. doi: 10.1038/mt.2010.274. Epub 2011 Jan 18.

17.

Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial.

Marks WJ Jr, Bartus RT, Siffert J, Davis CS, Lozano A, Boulis N, Vitek J, Stacy M, Turner D, Verhagen L, Bakay R, Watts R, Guthrie B, Jankovic J, Simpson R, Tagliati M, Alterman R, Stern M, Baltuch G, Starr PA, Larson PS, Ostrem JL, Nutt J, Kieburtz K, Kordower JH, Olanow CW.

Lancet Neurol. 2010 Dec;9(12):1164-1172. doi: 10.1016/S1474-4422(10)70254-4. Epub 2010 Oct 20.

PMID:
20970382
18.

The ethics of gene therapy: balancing the risks.

Deakin CT, Alexander IE, Kerridge I.

Curr Opin Mol Ther. 2010 Oct;12(5):578-85. Review.

PMID:
20886390
19.

Case report of a serious adverse event following the administration of T cells transduced with a chimeric antigen receptor recognizing ERBB2.

Morgan RA, Yang JC, Kitano M, Dudley ME, Laurencot CM, Rosenberg SA.

Mol Ther. 2010 Apr;18(4):843-51. doi: 10.1038/mt.2010.24. Epub 2010 Feb 23.

20.

Personal reflections on the origins and emergence of recombinant DNA technology.

Berg P, Mertz JE.

Genetics. 2010 Jan;184(1):9-17. doi: 10.1534/genetics.109.112144.

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