Literature DB >> 30107998

Breaking and Sealing Barriers in Retinal Gene Therapy.

Joan W Miller1, Luk H Vandenberghe2.   

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Year:  2018        PMID: 30107998      PMCID: PMC6127874          DOI: 10.1016/j.ymthe.2018.08.003

Source DB:  PubMed          Journal:  Mol Ther        ISSN: 1525-0016            Impact factor:   11.454


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  8 in total

1.  Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.

Authors:  Stephen Russell; Jean Bennett; Jennifer A Wellman; Daniel C Chung; Zi-Fan Yu; Amy Tillman; Janet Wittes; Julie Pappas; Okan Elci; Sarah McCague; Dominique Cross; Kathleen A Marshall; Jean Walshire; Taylor L Kehoe; Hannah Reichert; Maria Davis; Leslie Raffini; Lindsey A George; F Parker Hudson; Laura Dingfield; Xiaosong Zhu; Julia A Haller; Elliott H Sohn; Vinit B Mahajan; Wanda Pfeifer; Michelle Weckmann; Chris Johnson; Dina Gewaily; Arlene Drack; Edwin Stone; Katie Wachtel; Francesca Simonelli; Bart P Leroy; J Fraser Wright; Katherine A High; Albert M Maguire
Journal:  Lancet       Date:  2017-07-14       Impact factor: 79.321

2.  Preclinical safety evaluation of AAV2-sFLT01- a gene therapy for age-related macular degeneration.

Authors:  Timothy K Maclachlan; Michael Lukason; Margaret Collins; Robert Munger; Elisabete Isenberger; Cindy Rogers; Shana Malatos; Elizabeth Dufresne; James Morris; Roberto Calcedo; Gabor Veres; Abraham Scaria; Laura Andrews; Samuel Wadsworth
Journal:  Mol Ther       Date:  2010-11-30       Impact factor: 11.454

3.  Synaptic pathology and therapeutic repair in adult retinoschisis mouse by AAV-RS1 transfer.

Authors:  Jingxing Ou; Camasamudram Vijayasarathy; Lucia Ziccardi; Shan Chen; Yong Zeng; Dario Marangoni; Jodie G Pope; Ronald A Bush; Zhijian Wu; Wei Li; Paul A Sieving
Journal:  J Clin Invest       Date:  2015-06-22       Impact factor: 14.808

4.  Age-related macular degeneration revisited--piecing the puzzle: the LXIX Edward Jackson memorial lecture.

Authors:  Joan W Miller
Journal:  Am J Ophthalmol       Date:  2013-01       Impact factor: 5.258

5.  Intravitreal delivery of AAV8 retinoschisin results in cell type-specific gene expression and retinal rescue in the Rs1-KO mouse.

Authors:  T K Park; Z Wu; S Kjellstrom; Y Zeng; R A Bush; P A Sieving; P Colosi
Journal:  Gene Ther       Date:  2009-05-21       Impact factor: 5.250

6.  Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous.

Authors:  Deniz Dalkara; Kathleen D Kolstad; Natalia Caporale; Meike Visel; Ryan R Klimczak; David V Schaffer; John G Flannery
Journal:  Mol Ther       Date:  2009-08-11       Impact factor: 11.454

7.  Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model.

Authors:  A Auricchio; G Kobinger; V Anand; M Hildinger; E O'Connor; A M Maguire; J M Wilson; J Bennett
Journal:  Hum Mol Genet       Date:  2001-12-15       Impact factor: 6.150

8.  Ocular and systemic safety of a recombinant AAV8 vector for X-linked retinoschisis gene therapy: GLP studies in rabbits and Rs1-KO mice.

Authors:  Dario Marangoni; Ronald A Bush; Yong Zeng; Lisa L Wei; Lucia Ziccardi; Camasamudram Vijayasarathy; Joshua T Bartoe; Kiran Palyada; Maria Santos; Suja Hiriyanna; Zhijian Wu; Peter Colosi; Paul A Sieving
Journal:  Mol Ther Methods Clin Dev       Date:  2016-03-16       Impact factor: 6.698

  8 in total
  4 in total

1.  Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa.

Authors:  Harry O Orlans; Michelle E McClements; Alun R Barnard; Cristina Martinez-Fernandez de la Camara; Robert E MacLaren
Journal:  Nat Commun       Date:  2021-08-16       Impact factor: 14.919

Review 2.  Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy.

Authors:  Albert M Maguire; Jean Bennett; Elena M Aleman; Bart P Leroy; Tomas S Aleman
Journal:  Mol Ther       Date:  2020-12-03       Impact factor: 11.454

Review 3.  Progress in treating inherited retinal diseases: Early subretinal gene therapy clinical trials and candidates for future initiatives.

Authors:  Alexandra V Garafalo; Artur V Cideciyan; Elise Héon; Rebecca Sheplock; Alexander Pearson; Caberry WeiYang Yu; Alexander Sumaroka; Gustavo D Aguirre; Samuel G Jacobson
Journal:  Prog Retin Eye Res       Date:  2019-12-30       Impact factor: 21.198

Review 4.  Updates on Gene Therapy for Diabetic Retinopathy.

Authors:  Jiang-Hui Wang; Georgina Eloise Roberts; Guei-Sheung Liu
Journal:  Curr Diab Rep       Date:  2020-05-16       Impact factor: 4.810

  4 in total

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