title: Preserved outer retina in AIPL1 Leber's congenital amaurosis: implications for gene therapy.
authors.affiliation: UCL Institute of Ophthalmology, University College London, London, UK; Moorfields Eye Hospital, London, UK.
authors.forename: Jonathan
authors.initials: J
authors.lastname: Aboshiha
authors.affiliation: UCL Institute of Ophthalmology, University College London, London, UK; Moorfields Eye Hospital, London, UK.
authors.forename: Adam M
authors.initials: AM
authors.lastname: Dubis
authors.affiliation: UCL Institute of Ophthalmology, University College London, London, UK.
authors.forename: Jacqueline
authors.initials: J
authors.lastname: van der Spuy
authors.affiliation: UCL Institute of Ophthalmology, University College London, London, UK; Moorfields Eye Hospital, London, UK.
authors.forename: Koji M
authors.initials: KM
authors.lastname: Nishiguchi
authors.affiliation: Medical University of South Carolina Storm Eye Institute, Charleston, South Carolina.
authors.forename: Edward W
authors.initials: EW
authors.lastname: Cheeseman
authors.affiliation: Instituto de Investigación Sanitaria-Fundación Jiménez Díaz University Hospital, Centro de Investigación Biomédica en Red de Enfermedades Raras, Madrid, Spain.
authors.forename: Carmen
authors.initials: C
authors.lastname: Ayuso
authors.affiliation: Schneider Children's Medical Center of Israel, Petah Tikva, Israel.
authors.forename: Miriam
authors.initials: M
authors.lastname: Ehrenberg
authors.affiliation: Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, Second University of Naples, Naples, Italy.
authors.forename: Francesca
authors.initials: F
authors.lastname: Simonelli
authors.affiliation: UCL Institute of Ophthalmology, University College London, London, UK; Moorfields Eye Hospital, London, UK.
authors.forename: James W
authors.initials: JW
authors.lastname: Bainbridge
authors.affiliation: UCL Institute of Ophthalmology, University College London, London, UK; Moorfields Eye Hospital, London, UK. Electronic address: michel.michaelides@ucl.ac.uk.
authors.forename: Michel
authors.initials: M
authors.lastname: Michaelides
mesh_terms: D048868:Adaptor Proteins, Signal Transducing; D000293:Adolescent; D000328:Adult; D002352:Carrier Proteins / Q000235:genetics*; D002648:Child; D002675:Child, Preschool; D005136:Eye Proteins / Q000235:genetics*; D005260:Female; D015316:Genetic Therapy; D006801:Humans; D007223:Infant; D057130:Leber Congenital Amaurosis / Q000235:genetics* / Q000628:therapy; D008297:Male; D020419:Photoreceptor Cells, Vertebrate / Q000166:cytology* / Q000502:physiology; D055213:Retinal Pigment Epithelium / Q000166:cytology* / Q000502:physiology; D041623:Tomography, Optical Coherence; D014792:Visual Acuity; D055815:Young Adult
chemical_list: C403625:AIPL1 protein, human; D048868:Adaptor Proteins, Signal Transducing; D002352:Carrier Proteins; D005136:Eye Proteins