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Results: 1 to 20 of 133

Similar articles for PubMed (Select 18441370)

1.

Safety and efficacy of gene transfer for Leber's congenital amaurosis.

Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr, Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, Rossi S, Lyubarsky A, Arruda VR, Konkle B, Stone E, Sun J, Jacobs J, Dell'Osso L, Hertle R, Ma JX, Redmond TM, Zhu X, Hauck B, Zelenaia O, Shindler KS, Maguire MG, Wright JF, Volpe NJ, McDonnell JW, Auricchio A, High KA, Bennett J.

N Engl J Med. 2008 May 22;358(21):2240-8. doi: 10.1056/NEJMoa0802315. Epub 2008 Apr 27.

2.

[Developments in gene delivery vectors for ocular gene therapy].

Khabou H, Dalkara D.

Med Sci (Paris). 2015 May;31(5):529-37. doi: 10.1051/medsci/20153105015. Epub 2015 Jun 9. French.

PMID:
26059304
3.

Characterization of Leber's Congenital Amaurosis-associated NMNAT1 Mutants.

Sasaki Y, Margolin Z, Borgo B, Havranek JJ, Milbrandt J.

J Biol Chem. 2015 May 27. pii: jbc.M115.637850. [Epub ahead of print]

4.

Long-term effect of gene therapy on Leber's congenital amaurosis.

Bainbridge JW, Mehat MS, Sundaram V, Robbie SJ, Barker SE, Ripamonti C, Georgiadis A, Mowat FM, Beattie SG, Gardner PJ, Feathers KL, Luong VA, Yzer S, Balaggan K, Viswanathan A, de Ravel TJ, Casteels I, Holder GE, Tyler N, Fitzke FW, Weleber RG, Nardini M, Moore AT, Thompson DA, Petersen-Jones SM, Michaelides M, van den Born LI, Stockman A, Smith AJ, Rubin G, Ali RR.

N Engl J Med. 2015 May 14;372(20):1887-97. doi: 10.1056/NEJMoa1414221. Epub 2015 May 4.

PMID:
25938638
5.

Long-term effects of retinal gene therapy in childhood blindness.

Wright AF.

N Engl J Med. 2015 May 14;372(20):1954-5. doi: 10.1056/NEJMe1503419. Epub 2015 May 4. No abstract available.

PMID:
25938534
6.

Improvement and decline in vision with gene therapy in childhood blindness.

Jacobson SG, Cideciyan AV, Roman AJ, Sumaroka A, Schwartz SB, Heon E, Hauswirth WW.

N Engl J Med. 2015 May 14;372(20):1920-6. doi: 10.1056/NEJMoa1412965. Epub 2015 May 3.

PMID:
25936984
7.

Retinal astrocytic hamartoma in a patient with Leber's congenital amaurosis.

Ambiya V, Kuppermann BD, Narayanan R.

BMJ Case Rep. 2015 Mar 3;2015. pii: bcr2014208374. doi: 10.1136/bcr-2014-208374. No abstract available.

PMID:
25737223
8.

Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium.

Thompson DA, Ali RR, Banin E, Branham KE, Flannery JG, Gamm DM, Hauswirth WW, Heckenlively JR, Iannaccone A, Jayasundera KT, Khan NW, Molday RS, Pennesi ME, Reh TA, Weleber RG, Zacks DN; Monaciano Consortium.

Invest Ophthalmol Vis Sci. 2015 Feb 9;56(2):918-31. doi: 10.1167/iovs.14-16049. Review.

PMID:
25667399
9.

The Status of RPE65 Gene Therapy Trials: Safety and Efficacy.

Pierce EA, Bennett J.

Cold Spring Harb Perspect Med. 2015 Jan 29. pii: a017285. doi: 10.1101/cshperspect.a017285. [Epub ahead of print]

PMID:
25635059
10.

Preserved outer retina in AIPL1 Leber's congenital amaurosis: implications for gene therapy.

Aboshiha J, Dubis AM, van der Spuy J, Nishiguchi KM, Cheeseman EW, Ayuso C, Ehrenberg M, Simonelli F, Bainbridge JW, Michaelides M.

Ophthalmology. 2015 Apr;122(4):862-4. doi: 10.1016/j.ophtha.2014.11.019. Epub 2015 Jan 14. No abstract available.

PMID:
25596619
11.

Gene therapy for the eye focus on mutation-independent approaches.

Dalkara D, Duebel J, Sahel JA.

Curr Opin Neurol. 2015 Feb;28(1):51-60. doi: 10.1097/WCO.0000000000000168.

PMID:
25545056
12.

Pseudo-fovea formation after gene therapy for RPE65-LCA.

Cideciyan AV, Aguirre GK, Jacobson SG, Butt OH, Schwartz SB, Swider M, Roman AJ, Sadigh S, Hauswirth WW.

Invest Ophthalmol Vis Sci. 2014 Dec 23;56(1):526-37. doi: 10.1167/iovs.14-15895.

13.

Gene therapy for eye as regenerative medicine? Lessons from RPE65 gene therapy for Leber's Congenital Amaurosis.

Rakoczy EP, Narfström K.

Int J Biochem Cell Biol. 2014 Nov;56:153-7. doi: 10.1016/j.biocel.2014.09.022. Epub 2014 Oct 5.

PMID:
25286304
14.

Nature of the visual loss in observers with Leber's congenital amaurosis caused by specific mutations in RPE65.

Ripamonti C, Henning GB, Ali RR, Bainbridge JW, Robbie SJ, Sundaram V, Luong VA, van den Born LI, Casteels I, de Ravel TJ, Moore AT, Stockman A.

Invest Ophthalmol Vis Sci. 2014 Sep 25;55(10):6817-28. doi: 10.1167/iovs.14-14923.

PMID:
25257057
15.

Nanoparticle-assisted targeted delivery of eye-specific genes to eyes significantly improves the vision of blind mice in vivo.

Rajala A, Wang Y, Zhu Y, Ranjo-Bishop M, Ma JX, Mao C, Rajala RV.

Nano Lett. 2014 Sep 10;14(9):5257-63. doi: 10.1021/nl502275s. Epub 2014 Aug 12.

PMID:
25115433
16.

Oral 9-cis retinoid for childhood blindness due to Leber congenital amaurosis caused by RPE65 or LRAT mutations: an open-label phase 1b trial.

Koenekoop RK, Sui R, Sallum J, van den Born LI, Ajlan R, Khan A, den Hollander AI, Cremers FP, Mendola JD, Bittner AK, Dagnelie G, Schuchard RA, Saperstein DA.

Lancet. 2014 Oct 25;384(9953):1513-20. doi: 10.1016/S0140-6736(14)60153-7. Epub 2014 Jul 13.

PMID:
25030840
17.

Gene therapy for inherited retinal degenerations.

Dalkara D, Sahel JA.

C R Biol. 2014 Mar;337(3):185-92. doi: 10.1016/j.crvi.2014.01.002. Epub 2014 Mar 11. Review.

PMID:
24702845
18.

Persistence of non-viral vector mediated RPE65 expression: case for viability as a gene transfer therapy for RPE-based diseases.

Koirala A, Conley SM, Makkia R, Liu Z, Cooper MJ, Sparrow JR, Naash MI.

J Control Release. 2013 Dec 28;172(3):745-52. doi: 10.1016/j.jconrel.2013.08.299. Epub 2013 Sep 11.

19.

Successful gene therapy in older Rpe65-deficient dogs following subretinal injection of an adeno-associated vector expressing RPE65.

Annear MJ, Mowat FM, Bartoe JT, Querubin J, Azam SA, Basche M, Curran PG, Smith AJ, Bainbridge JW, Ali RR, Petersen-Jones SM.

Hum Gene Ther. 2013 Oct;24(10):883-93. doi: 10.1089/hum.2013.146.

PMID:
24028205
20.

Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2.

Testa F, Maguire AM, Rossi S, Pierce EA, Melillo P, Marshall K, Banfi S, Surace EM, Sun J, Acerra C, Wright JF, Wellman J, High KA, Auricchio A, Bennett J, Simonelli F.

Ophthalmology. 2013 Jun;120(6):1283-91. doi: 10.1016/j.ophtha.2012.11.048. Epub 2013 Mar 6.

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