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Items: 1 to 20 of 21

1.

Promises and challenges of genetic therapy for blindness.

Cremers FP, Collin RW.

Lancet. 2009 Nov 7;374(9701):1569-70. doi: 10.1016/S0140-6736(09)61869-9. Epub 2009 Oct 23. No abstract available.

PMID:
19854500
2.

Gene therapy for Leber congenital amaurosis.

Dejneka NS, Surace EM, Bennett J.

Adv Exp Med Biol. 2003;533:415-22. Review. No abstract available.

PMID:
15180293
3.

Recent advances in early-onset severe retinal degeneration: more than just basic research.

Preising MN, Heegaard S.

Trends Mol Med. 2004 Feb;10(2):51-4. Review.

PMID:
15106616
4.

Gene therapy for Leber congenital amaurosis.

Bennett J.

Novartis Found Symp. 2004;255:195-202; discussion 202-7. Review.

PMID:
14750605
5.

Gene augmentation trials using the Rpe65-deficient dog: contributions towards development and refinement of human clinical trials.

Petersen-Jones SM, Annear MJ, Bartoe JT, Mowat FM, Barker SE, Smith AJ, Bainbridge JW, Ali RR.

Adv Exp Med Biol. 2012;723:177-82. doi: 10.1007/978-1-4614-0631-0_24. Review. No abstract available.

PMID:
22183331
6.

Clinical gene therapy for the treatment of RPE65-associated Leber congenital amaurosis.

Stein L, Roy K, Lei L, Kaushal S.

Expert Opin Biol Ther. 2011 Mar;11(3):429-39. doi: 10.1517/14712598.2011.557358. Review.

PMID:
21299439
7.

Gene therapy for mitochondrial diseases: Leber Hereditary Optic Neuropathy as the first candidate for a clinical trial.

Cwerman-Thibault H, Augustin S, Ellouze S, Sahel JA, Corral-Debrinski M.

C R Biol. 2014 Mar;337(3):193-206. doi: 10.1016/j.crvi.2013.11.011. Epub 2014 Feb 24. Review.

PMID:
24702846
8.

Gene-Based Therapies for Leber Hereditary Optic Neuropathy. Hype or Hope?

Mackey DA, Kearns LS, Hewitt AW.

Asia Pac J Ophthalmol (Phila). 2016 Jul-Aug;5(4):253-5. doi: 10.1097/APO.0000000000000220. Review.

9.

An analysis of retinal gene therapy clinical trials.

MacLaren RE.

Curr Opin Mol Ther. 2009 Oct;11(5):540-6. Review.

10.

Manufacturing and regulatory strategies for clinical AAV2-hRPE65.

Wright JF, Wellman J, High KA.

Curr Gene Ther. 2010 Oct;10(5):341-9. Review.

PMID:
20712582
11.

The consortium project to treat RPE65 deficiency in humans.

Hauswirth WW.

Retina. 2005 Dec;25(8 Suppl):S60. Review. No abstract available.

PMID:
16374340
12.

Leber congenital amaurosis: ciliary proteins on the move.

Koenekoop RK, Cremers FP, den Hollander AI.

Ophthalmic Genet. 2007 Sep;28(3):111-2. Review. No abstract available.

PMID:
17896308
13.

A comprehensive review of retinal gene therapy.

Boye SE, Boye SL, Lewin AS, Hauswirth WW.

Mol Ther. 2013 Mar;21(3):509-19. doi: 10.1038/mt.2012.280. Epub 2013 Jan 29. Review. Erratum in: Mol Ther. 2013 Nov;21(11):2130.

14.

Biochemical function of the LCA linked protein, aryl hydrocarbon receptor interacting protein like-1 (AIPL1). Role of AIPL1 in retina.

Schwartz ML, Hurley JB, Ramamurthy V.

Adv Exp Med Biol. 2006;572:89-94. Review. No abstract available.

PMID:
17249560
15.

The chaperone function of the LCA protein AIPL1. AIPL1 chaperone function.

Van der Spuy J, Cheetham ME.

Adv Exp Med Biol. 2006;572:471-6. Review. No abstract available.

PMID:
17249611
16.

Leber congenital amaurosis--genotyping required for possible inclusion in a clinical trial.

Perrault I, Gerber S, Hanein S, Picaud S, Rozet JM, Dufier JL, Munnich A, Sahel J, Kaplan J.

Adv Exp Med Biol. 2003;533:69-77. Review. No abstract available.

PMID:
15180249
17.

Gene therapy: a bold direction for HIV-1 treatment.

Sarver N, Rossi J.

AIDS Res Hum Retroviruses. 1993 May;9(5):483-7. Review. No abstract available.

PMID:
8391287
18.

Biology of retinoschisin.

Vijayasarathy C, Ziccardi L, Sieving PA.

Adv Exp Med Biol. 2012;723:513-8. doi: 10.1007/978-1-4614-0631-0_64. Review. No abstract available.

19.

[Gene therapy and rare diseases].

Cohen-Haguenauer O.

Rev Med Interne. 2011 Dec;32 Suppl 2:S210-2. doi: 10.1016/j.revmed.2011.09.015. Epub 2011 Oct 28. Review. French. No abstract available.

PMID:
22037347
20.

Do Genes Matter in Treating Eye Disease?

Novack GD.

Ocul Surf. 2015 Oct;13(4):346-9. doi: 10.1016/j.jtos.2015.08.003. Epub 2015 Aug 10. Review. No abstract available.

PMID:
26272274

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