治疗人类视网膜色素上皮特异性65 kDa蛋白（RPE65）缺乏症的联合项目。 - Suppr

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超能文献

The consortium project to treat RPE65 deficiency in humans.

作者信息

Hauswirth William W

机构信息

Ophthalmology and Molecular Genetics at the University of Florida School of Medicine, Gainesville, FL, USA.

出版信息

Retina. 2005 Dec;25(8 Suppl):S60. doi: 10.1097/00006982-200512001-00027.

DOI:10.1097/00006982-200512001-00027

PMID:16374340

Abstract

摘要

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引用本文的文献

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The consortium project to treat RPE65 deficiency in humans.

作者信息

Hauswirth William W

机构信息

Ophthalmology and Molecular Genetics at the University of Florida School of Medicine, Gainesville, FL, USA.

出版信息

Retina. 2005 Dec;25(8 Suppl):S60. doi: 10.1097/00006982-200512001-00027.

DOI:10.1097/00006982-200512001-00027

PMID:16374340

Abstract

摘要

相似文献

The consortium project to treat RPE65 deficiency in humans.

Retina. 2005 Dec;25(8 Suppl):S60. doi: 10.1097/00006982-200512001-00027.

Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness.

Mol Ther. 2005 Dec;12(6):1072-82. doi: 10.1016/j.ymthe.2005.08.008. Epub 2005 Oct 14.

Canine and human visual cortex intact and responsive despite early retinal blindness from RPE65 mutation.

PLoS Med. 2007 Jun;4(6):e230. doi: 10.1371/journal.pmed.0040230.

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Gene Ther. 2008 May;15(9):633-4. doi: 10.1038/gt.2008.28. Epub 2008 Mar 13.

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Retina. 2005 Dec;25(8 Suppl):S69-S71. doi: 10.1097/00006982-200512001-00033.

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Retina. 2005 Dec;25(8 Suppl):S10-S12. doi: 10.1097/00006982-200512001-00004.

Proceedings of the First International Symposium on Translational Clinical Research for Inherited and Orphan Retinal Diseases. November 5-7, 2004. Washington, DC, USA.

Retina. 2005 Dec;25(8 Suppl):S1-113.

Versatility of AAV vectors for retinal gene transfer.

Vision Res. 2008 Feb;48(3):353-9. doi: 10.1016/j.visres.2007.07.027. Epub 2007 Oct 17.

Gene therapy restores vision in a canine model of childhood blindness.

Nat Genet. 2001 May;28(1):92-5. doi: 10.1038/ng0501-92.

引用本文的文献

Subretinal delivery and electroporation in pigmented and nonpigmented adult mouse eyes.

Methods Mol Biol. 2012;884:53-69. doi: 10.1007/978-1-61779-848-1_4.

Therapeutic challenges to retinitis pigmentosa: from neuroprotection to gene therapy.

Curr Genomics. 2011 Jun;12(4):276-84. doi: 10.2174/138920211795860062.

Technical brief: subretinal injection and electroporation into adult mouse eyes.

Mol Vis. 2008;14:2211-26. Epub 2008 Dec 5.

Perspective on genes and mutations causing retinitis pigmentosa.

Arch Ophthalmol. 2007 Feb;125(2):151-8. doi: 10.1001/archopht.125.2.151.

Diseases caused by defects in the visual cycle: retinoids as potential therapeutic agents.

Annu Rev Pharmacol Toxicol. 2007;47:469-512. doi: 10.1146/annurev.pharmtox.47.120505.105225.