A new era in medical therapy for retinal degenerative disease?

The Lancet

Volumn 384, Issue 9953, 2014, Pages 1482-1484

  Iannaccone, Alessandro ^a   Zarbin, Marco A ^b  

^a UNIVERSITY OF TENNESSEE HEALTH SCIENCE CENTER   (United States)

^b INSTITUTE OF OPHTHALMOLOGY AND VISUAL SCIENCE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

9 CIS RETINOL ACETATE; PLACEBO; RETINOL; RETINOL ACETATE;

BRAIN PSEUDOTUMOR; CONTROLLED CLINICAL TRIAL (TOPIC); DRUG EFFICACY; DRUG INDUCED HEADACHE; ELECTRORETINOGRAM; FUNCTIONAL MAGNETIC RESONANCE IMAGING; GENE; GENE MUTATION; GOLDMANN VISUAL FIELD; HUMAN; LEBER CONGENITAL AMAUROSIS; LRAT GENE; NOTE; PHOTORECEPTOR; PLACEBO EFFECT; RETINA DEGENERATION; RPE65 GENE; TREATMENT DURATION; VISUAL ACUITY; VISUAL FIELD; ANALOGS AND DERIVATIVES; BLINDNESS;

BLINDNESS; HUMANS; LEBER CONGENITAL AMAUROSIS; VITAMIN A;

EID: 84908346985     PISSN: 01406736     EISSN: 1474547X     Source Type: Journal    
DOI: 10.1016/S0140-6736(14)60956-9     Document Type: Note

References (16)

1. RK Koenekoop, R Sui, J Sallum et al. Oral 9-cis retinoid for childhood blindness due to Leber congenital amaurosis caused by RPE65 or LRAT mutations: an open-label phase 1b trial Lancet 2014 published online July 14. http://dx.doi.org/10.1016/S0140-6736(14)60153-7
2. A Iannaccone, E Rispoli, EM Vingolo et al. Correlation between Goldmann perimetry and maximal electroretinogram response in retinitis pigmentosa Doc Ophthalmol 90 1995 129 142
3. G Dagnelie Conversion of planimetric visual field data into solid angles and retinal areas Clin Vision Sci 5 1990 95 103
4. AK Bittner, MH Iftikhar, G Dagnelie Test-retest, within-visit variability of Goldmann visual fields in retinitis pigmentosa Invest Ophthalmol Vis Sci 52 2011 8042 8046
5. K Mykytyn, VC Sheffield Establishing a connection between cilia and Bardet-Biedl Syndrome Trends Mol Med 10 2004 106 109
6. KF Cox, NC Kerr, M Kedrov et al. Phenotypic expression of Bardet-Biedl syndrome in patients homozygous for the common M390R mutation in the BBS1 gene Vision Res 75 2012 77 87
7. A Iannaccone, EM Vingolo, E Rispoli, G De Propris, P Tanzilli, MR Pannarale Electroretinographic alterations in the Laurence-Moon-Bardet-Biedl phenotype Acta Ophthalmol Scand 74 1996 8 13
8. SG Jacobson, TS Aleman, AV Cideciyan et al. Defining the residual vision in Leber congenital amaurosis caused by RPE65 mutations Invest Ophthalmol Vis Sci 50 2009 2368 2375
9. RG Weleber, M Michaelides, KM Trzupek, NB Stover, EM Stone The phenotype of Severe Early Childhood Onset Retinal Dystrophy (SECORD) from mutation of RPE65 and differentiation from Leber congenital amaurosis Invest Ophthalmol Vis Sci 52 2011 292 302
10. A Dev Borman, LA Ocaka, DS Mackay et al. Early onset retinal dystrophy due to mutations in LRAT: molecular analysis and detailed phenotypic study Invest Ophthalmol Vis Sci 53 2012 3927 3938
11. JP Van Hooser, TS Aleman, YG He et al. Rapid restoration of visual pigment and function with oral retinoid in a mouse model of childhood blindness Proc Natl Acad Sci USA 97 2000 8623 8628
12. T Maeda, AV Cideciyan, A Maeda et al. Loss of cone photoreceptors caused by chromophore depletion is partially prevented by the artificial chromophore pro-drug, 9-cis-retinyl acetate Hum Mol Genet 18 2009 2277 2287
13. SG Jacobson, AV Cideciyan, R Ratnakaram et al. Gene therapy for Leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years Arch Ophthalmol 130 2012 9 24
14. AJ Roman, AV Cideciyan, SB Schwartz, MB Olivares, E Heon, SG Jacobson Intervisit variability of visual parameters in Leber congenital amaurosis caused by RPE65 mutations Invest Ophthalmol Vis Sci 54 2013 1378 1383
15. K Nowomiejska, A Brzozowska, T Zarnowski, R Rejdak, RG Weleber, U Schiefer Variability in isopter position and fatigue during semi-automated kinetic perimetry Ophthalmologica 227 2012 166 172
16. S Randhawa, GP Van Stavern Idiopathic intracranial hypertension (pseudotumor cerebri) Curr Opin Ophthalmol 19 2008 445 453