Long-term effects of retinal gene therapy in childhood blindness

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

Publication types

  • Editorial
  • Comment

MeSH terms

  • Animals
  • DNA, Complementary / administration & dosage*
  • Genetic Therapy*
  • Humans
  • Leber Congenital Amaurosis / therapy*
  • Photoreceptor Cells, Vertebrate / pathology*
  • cis-trans-Isomerases / genetics*

Substances

  • DNA, Complementary
  • retinoid isomerohydrolase
  • cis-trans-Isomerases