My child has Leber congenital amaurosis: why is he/she not eligible for gene therapy trials?
- PMID: 19896875
- DOI: 10.1016/j.jaapos.2009.09.006
My child has Leber congenital amaurosis: why is he/she not eligible for gene therapy trials?
Comment on
-
Which Leber congenital amaurosis patients are eligible for gene therapy trials?J AAPOS. 2009 Oct;13(5):463-5. doi: 10.1016/j.jaapos.2009.08.006. J AAPOS. 2009. PMID: 19840725 Free PMC article.
Similar articles
-
Leber congenital amaurosis: clinical correlations with genotypes, gene therapy trials update, and future directions.J AAPOS. 2009 Dec;13(6):587-92. doi: 10.1016/j.jaapos.2009.10.004. J AAPOS. 2009. PMID: 20006823 Review.
-
Seeing is believing: gene therapy shows promise for ocular disorders.JAMA. 2010 Oct 13;304(14):1543-5. doi: 10.1001/jama.2010.1412. JAMA. 2010. PMID: 20940376 No abstract available.
-
Gene augmentation trials using the Rpe65-deficient dog: contributions towards development and refinement of human clinical trials.Adv Exp Med Biol. 2012;723:177-82. doi: 10.1007/978-1-4614-0631-0_24. Adv Exp Med Biol. 2012. PMID: 22183331 Review. No abstract available.
-
Which Leber congenital amaurosis patients are eligible for gene therapy trials?J AAPOS. 2009 Oct;13(5):463-5. doi: 10.1016/j.jaapos.2009.08.006. J AAPOS. 2009. PMID: 19840725 Free PMC article.
-
Manufacturing and regulatory strategies for clinical AAV2-hRPE65.Curr Gene Ther. 2010 Oct;10(5):341-9. doi: 10.2174/156652310793180715. Curr Gene Ther. 2010. PMID: 20712582 Review.
Cited by
-
"Is a cure in my sight?" Multi-stakeholder perspectives on phase I choroideremia gene transfer clinical trials.Genet Med. 2014 May;16(5):379-85. doi: 10.1038/gim.2013.148. Epub 2013 Sep 26. Genet Med. 2014. PMID: 24071795 Free PMC article. Clinical Trial.
-
Ocular gene transfer in the spotlight: implications of newspaper content for clinical communications.BMC Med Ethics. 2014 Jul 16;15:58. doi: 10.1186/1472-6939-15-58. BMC Med Ethics. 2014. PMID: 25027482 Free PMC article.
Publication types
MeSH terms
Substances
LinkOut - more resources
Full Text Sources
Medical