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

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

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adaptor Proteins, Signal Transducing
  • Adolescent
  • Adult
  • Carrier Proteins / genetics*
  • Child
  • Child, Preschool
  • Eye Proteins / genetics*
  • Female
  • Genetic Therapy*
  • Humans
  • Infant
  • Leber Congenital Amaurosis / genetics*
  • Leber Congenital Amaurosis / therapy
  • Male
  • Photoreceptor Cells, Vertebrate / cytology*
  • Photoreceptor Cells, Vertebrate / physiology
  • Retinal Pigment Epithelium / cytology*
  • Retinal Pigment Epithelium / physiology
  • Tomography, Optical Coherence
  • Visual Acuity
  • Young Adult

Substances

  • AIPL1 protein, human
  • Adaptor Proteins, Signal Transducing
  • Carrier Proteins
  • Eye Proteins

Supplementary concepts

  • Leber congenital amaurosis, type 4