Safety and efficacy of gene transfer for Leber's congenital amaurosis.
N Engl J Med. 2008 May 22;358(21):2240-8. doi: 10.1056/NEJMoa0802315. Epub 2008 Apr 27.
N Engl J Med. 2008.
PMID: 18441370
Free PMC article.
Clinical Trial.