Literature DB >> 25671556

Dog models for blinding inherited retinal dystrophies.

Simon M Petersen-Jones1, András M Komáromy.   

Abstract

Spontaneous canine models exist for several inherited retinal dystrophies. This review will summarize the models and indicate where they have been used in translational gene therapy trials. The RPE65 gene therapy trials to treat childhood blindness are a good example of how studies in dogs have contributed to therapy development. Outcomes in human clinical trials are compared and contrasted with the result of the preclinical dog trials.

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Year:  2015        PMID： 25671556      PMCID： PMC4442585          DOI： 10.1089/humc.2014.155

Source DB:  PubMed          Journal:  Hum Gene Ther Clin Dev        ISSN： 2324-8637            Impact factor:   5.032


  101 in total

1.  Eye movement recordings as an effectiveness indicator of gene therapy in RPE65-deficient canines: implications for the ocular motor system.

Authors:  Jonathan B Jacobs; Louis F Dell'Osso; Richard W Hertle; Gregory M Acland; Jean Bennett
Journal:  Invest Ophthalmol Vis Sci       Date:  2006-07       Impact factor: 4.799

2.  Molecular anthropology meets genetic medicine to treat blindness in the North African Jewish population: human gene therapy initiated in Israel.

Authors:  Eyal Banin; Dikla Bandah-Rozenfeld; Alexey Obolensky; Artur V Cideciyan; Tomas S Aleman; Devora Marks-Ohana; Malka Sela; Sanford Boye; Alexander Sumaroka; Alejandro J Roman; Sharon B Schwartz; William W Hauswirth; Samuel G Jacobson; Itzhak Hemo; Dror Sharon
Journal:  Hum Gene Ther       Date:  2010-11-03       Impact factor: 5.695

3.  Misexpression of the constitutive Rpgr(ex1-19) variant leads to severe photoreceptor degeneration.

Authors:  Rachel N Wright; Dong-Hyun Hong; Brian Perkins
Journal:  Invest Ophthalmol Vis Sci       Date:  2011-07-15       Impact factor: 4.799

4.  Targeting gene expression to cones with human cone opsin promoters in recombinant AAV.

Authors:  A M Komáromy; J J Alexander; A E Cooper; V A Chiodo; L G Glushakova; G M Acland; W W Hauswirth; G D Aguirre
Journal:  Gene Ther       Date:  2008-03-13       Impact factor: 5.250

5.  Bestrophin gene mutations cause canine multifocal retinopathy: a novel animal model for best disease.

Authors:  Karina E Guziewicz; Barbara Zangerl; Sarah J Lindauer; Robert F Mullins; Lynne S Sandmeyer; Bruce H Grahn; Edwin M Stone; Gregory M Acland; Gustavo D Aguirre
Journal:  Invest Ophthalmol Vis Sci       Date:  2007-05       Impact factor: 4.799

6.  Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.

Authors:  Albert M Maguire; Katherine A High; Alberto Auricchio; J Fraser Wright; Eric A Pierce; Francesco Testa; Federico Mingozzi; Jeannette L Bennicelli; Gui-shuang Ying; Settimio Rossi; Ann Fulton; Kathleen A Marshall; Sandro Banfi; Daniel C Chung; Jessica I W Morgan; Bernd Hauck; Olga Zelenaia; Xiaosong Zhu; Leslie Raffini; Frauke Coppieters; Elfride De Baere; Kenneth S Shindler; Nicholas J Volpe; Enrico M Surace; Carmela Acerra; Arkady Lyubarsky; T Michael Redmond; Edwin Stone; Junwei Sun; Jennifer Wellman McDonnell; Bart P Leroy; Francesca Simonelli; Jean Bennett
Journal:  Lancet       Date:  2009-10-23       Impact factor: 79.321

7.  In utero gene therapy rescues vision in a murine model of congenital blindness.

Authors:  Nadine S Dejneka; Enrico M Surace; Tomas S Aleman; Artur V Cideciyan; Arkady Lyubarsky; Andrey Savchenko; T Michael Redmond; Waixing Tang; Zhangyong Wei; Tonia S Rex; Ernest Glover; Albert M Maguire; Edward N Pugh; Samuel G Jacobson; Jean Bennett
Journal:  Mol Ther       Date:  2004-02       Impact factor: 11.454

8.  Evaluation of a behavioral method for objective vision testing and identification of achromatopsia in dogs.

Authors:  Monique M Garcia; Gui-shuang Ying; Christina A Cocores; Jacqueline C Tanaka; András M Komáromy
Journal:  Am J Vet Res       Date:  2010-01       Impact factor: 1.156

9.  Human cone photoreceptor dependence on RPE65 isomerase.

Authors:  Samuel G Jacobson; Tomas S Aleman; Artur V Cideciyan; Elise Heon; Marcin Golczak; William A Beltran; Alexander Sumaroka; Sharon B Schwartz; Alejandro J Roman; Elizabeth A M Windsor; James M Wilson; Gustavo D Aguirre; Edwin M Stone; Krzysztof Palczewski
Journal:  Proc Natl Acad Sci U S A       Date:  2007-09-11       Impact factor: 11.205

10.  Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration.

Authors:  Francesca Simonelli; Albert M Maguire; Francesco Testa; Eric A Pierce; Federico Mingozzi; Jeannette L Bennicelli; Settimio Rossi; Kathleen Marshall; Sandro Banfi; Enrico M Surace; Junwei Sun; T Michael Redmond; Xiaosong Zhu; Kenneth S Shindler; Gui-Shuang Ying; Carmela Ziviello; Carmela Acerra; J Fraser Wright; Jennifer Wellman McDonnell; Katherine A High; Jean Bennett; Alberto Auricchio
Journal:  Mol Ther       Date:  2009-12-01       Impact factor: 11.454
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  24 in total

1.  Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs.

Authors:  R F Boyd; D G Sledge; S L Boye; S E Boye; W W Hauswirth; A M Komáromy; S M Petersen-Jones; J T Bartoe
Journal:  Gene Ther       Date:  2015-10-15       Impact factor: 5.250

2.  Long-Term Structural Outcomes of Late-Stage RPE65 Gene Therapy.

Authors:  Kristin L Gardiner; Artur V Cideciyan; Malgorzata Swider; Valérie L Dufour; Alexander Sumaroka; András M Komáromy; William W Hauswirth; Simone Iwabe; Samuel G Jacobson; William A Beltran; Gustavo D Aguirre
Journal:  Mol Ther       Date:  2019-09-03       Impact factor: 11.454

3.  Phenotypic characterization of complete CSNB in the inbred research beagle: how common is CSNB in research and companion dogs?

Authors:  Annie Oh; Ellis R Loew; Melanie L Foster; Michael G Davidson; Robert V English; Kristen J Gervais; Ian P Herring; Freya M Mowat
Journal:  Doc Ophthalmol       Date:  2018-07-26       Impact factor: 2.379

4.  Restoring vision at the fovea.

Authors:  Juliette E McGregor
Journal:  Curr Opin Behav Sci       Date:  2019-11-08

5.  Noise-Induced Hearing Loss and its Prevention: Current Issues in Mammalian Hearing.

Authors:  Colleen G Le Prell; Troy A Hackett; Ramnarayan Ramachandran
Journal:  Curr Opin Physiol       Date:  2020-07-12

6.  A modified silent substitution electroretinography protocol to separate photoreceptor subclass function in lightly sedated dogs.

Authors:  E N Wise; M L Foster; J Kremers; F M Mowat
Journal:  Vet Ophthalmol       Date:  2020-11-24       Impact factor: 1.644

7.  ERG assessment of altered retinal function in canine models of retinitis pigmentosa and monitoring of response to translatable gene augmentation therapy.

Authors:  Nate Pasmanter; Laurence M Occelli; Simon M Petersen-Jones
Journal:  Doc Ophthalmol       Date:  2021-04-05       Impact factor: 1.854

8.  A large deletion in RPGR causes XLPRA in Weimaraner dogs.

Authors:  Regina Kropatsch; Denis A Akkad; Matthias Frank; Carsten Rosenhagen; Janine Altmüller; Peter Nürnberg; Jörg T Epplen; Gabriele Dekomien
Journal:  Canine Genet Epidemiol       Date:  2016-07-08

9.  Development of a protocol for maintaining viability while shipping organoid-derived retinal tissue.

Authors:  Ratnesh K Singh; Paige Winkler; Francois Binette; Randolph D Glickman; Magdalene Seiler; Simon M Petersen-Jones; Igor O Nasonkin
Journal:  J Tissue Eng Regen Med       Date:  2020-01-06       Impact factor: 3.963

Review 10.  The use of nonhuman primates in studies of noise injury and treatment.

Authors:  Jane A Burton; Michelle D Valero; Troy A Hackett; Ramnarayan Ramachandran
Journal:  J Acoust Soc Am       Date:  2019-11       Impact factor: 2.482
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