Literature DB >> 30667378

Blinded by the light: a nonhuman primate model of achromatopsia.

Katherine E Uyhazi, Jean Bennett.   

Abstract

Achromatopsia is an inherited retinal degeneration characterized by the loss of cone photoreceptor function. In this issue of the JCI, Moshiri et al. characterize a naturally occurring model of the disease in the rhesus macaque caused by homozygous mutations in the phototransduction enzyme PDE6C. Using retinal imaging, and electrophysiologic and biochemical methods, the authors report a clinical phenotype nearly identical to the human condition. These findings represent the first genetic nonhuman primate model of an inherited retinal disease, and provide an ideal testing ground for the development of novel gene replacement, gene editing, and cell replacement therapies for cone dystrophies.

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Mesh:

Year:  2019        PMID: 30667378      PMCID: PMC6355213          DOI: 10.1172/JCI126205

Source DB:  PubMed          Journal:  J Clin Invest        ISSN: 0021-9738            Impact factor:   14.808


  17 in total

Review 1.  Causes and consequences of inherited cone disorders.

Authors:  Susanne Roosing; Alberta A H J Thiadens; Carel B Hoyng; Caroline C W Klaver; Anneke I den Hollander; Frans P M Cremers
Journal:  Prog Retin Eye Res       Date:  2014-05-22       Impact factor: 21.198

2.  Restoration of cone vision in the CNGA3-/- mouse model of congenital complete lack of cone photoreceptor function.

Authors:  Stylianos Michalakis; Regine Mühlfriedel; Naoyuki Tanimoto; Vidhyasankar Krishnamoorthy; Susanne Koch; M Dominik Fischer; Elvir Becirovic; Lin Bai; Gesine Huber; Susanne C Beck; Edda Fahl; Hildegard Büning; François Paquet-Durand; Xiangang Zong; Tim Gollisch; Martin Biel; Mathias W Seeliger
Journal:  Mol Ther       Date:  2010-07-13       Impact factor: 11.454

3.  Homozygosity mapping of the Achromatopsia locus in the Pingelapese.

Authors:  J D Winick; M L Blundell; B L Galke; A A Salam; S M Leal; M Karayiorgou
Journal:  Am J Hum Genet       Date:  1999-06       Impact factor: 11.025

Review 4.  Achromatopsia: a review.

Authors:  Meredith H Remmer; Neelesh Rastogi; Milan P Ranka; Emily J Ceisler
Journal:  Curr Opin Ophthalmol       Date:  2015-07       Impact factor: 3.761

5.  CNGB3-achromatopsia clinical trial with CNTF: diminished rod pathway responses with no evidence of improvement in cone function.

Authors:  Wadih M Zein; Brett G Jeffrey; Henry E Wiley; Amy E Turriff; Santa J Tumminia; Weng Tao; Ronald A Bush; Dario Marangoni; Rong Wen; Lisa L Wei; Paul A Sieving
Journal:  Invest Ophthalmol Vis Sci       Date:  2014-09-09       Impact factor: 4.799

Review 6.  Achromatopsia: clinical features, molecular genetics, animal models and therapeutic options.

Authors:  Nashila Hirji; Jonathan Aboshiha; Michalis Georgiou; James Bainbridge; Michel Michaelides
Journal:  Ophthalmic Genet       Date:  2018-01-05       Impact factor: 1.803

7.  A prospective longitudinal study of retinal structure and function in achromatopsia.

Authors:  Jonathan Aboshiha; Adam M Dubis; Jill Cowing; Rachel T A Fahy; Venki Sundaram; James W Bainbridge; Robin R Ali; Alfredo Dubra; Marko Nardini; Andrew R Webster; Anthony T Moore; Gary Rubin; Joseph Carroll; Michel Michaelides
Journal:  Invest Ophthalmol Vis Sci       Date:  2014-08-07       Impact factor: 4.799

8.  Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy.

Authors:  Livia S Carvalho; Jianhua Xu; Rachael A Pearson; Alexander J Smith; James W Bainbridge; Lynsie M Morris; Steven J Fliesler; Xi-Qin Ding; Robin R Ali
Journal:  Hum Mol Genet       Date:  2011-05-15       Impact factor: 6.150

9.  Hearing colors: an example of brain plasticity.

Authors:  Arantxa Alfaro; Ángela Bernabeu; Carlos Agulló; Jaime Parra; Eduardo Fernández
Journal:  Front Syst Neurosci       Date:  2015-04-14

Review 10.  Gene Therapy for Color Blindness.

Authors:  Mark M Hassall; Alun R Barnard; Robert E MacLaren
Journal:  Yale J Biol Med       Date:  2017-12-19
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  1 in total

Review 1.  Sensing through Non-Sensing Ocular Ion Channels.

Authors:  Meha Kabra; Bikash Ranjan Pattnaik
Journal:  Int J Mol Sci       Date:  2020-09-21       Impact factor: 6.208

  1 in total

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