Literature DB >> 28054582

Gene therapy approaches for prevention of retinal degeneration in Usher syndrome.

D S Williams1,2, A Chadha1,2, R Hazim1,2,3, D Gibbs4.   

Abstract

Entities:  

Mesh：

Year:  2017        PMID： 28054582      PMCID： PMC6055994          DOI： 10.1038/gt.2016.81

Source DB:  PubMed          Journal:  Gene Ther        ISSN： 0969-7128            Impact factor:   5.250


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  38 in total

1.  Myosin VIIa, the product of the Usher 1B syndrome gene, is concentrated in the connecting cilia of photoreceptor cells.

Authors:  X Liu; G Vansant; I P Udovichenko; U Wolfrum; D S Williams
Journal:  Cell Motil Cytoskeleton       Date:  1997

2.  Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system.

Authors:  Bernd Zetsche; Jonathan S Gootenberg; Omar O Abudayyeh; Ian M Slaymaker; Kira S Makarova; Patrick Essletzbichler; Sara E Volz; Julia Joung; John van der Oost; Aviv Regev; Eugene V Koonin; Feng Zhang
Journal:  Cell       Date:  2015-09-25       Impact factor: 41.582

3.  A novel gene for Usher syndrome type 2: mutations in the long isoform of whirlin are associated with retinitis pigmentosa and sensorineural hearing loss.

Authors:  Inga Ebermann; Hendrik P N Scholl; Peter Charbel Issa; Elvir Becirovic; Jürgen Lamprecht; Bernhard Jurklies; José M Millán; Elena Aller; Diana Mitter; Hanno Bolz
Journal:  Hum Genet       Date:  2006-12-15       Impact factor: 4.132

4.  In vitro and in vivo rescue of aberrant splicing in CEP290-associated LCA by antisense oligonucleotide delivery.

Authors:  Alejandro Garanto; Daniel C Chung; Lonneke Duijkers; Julio C Corral-Serrano; Muriël Messchaert; Ru Xiao; Jean Bennett; Luk H Vandenberghe; Rob W J Collin
Journal:  Hum Mol Genet       Date:  2016-04-22       Impact factor: 6.150

5.  Myosin VIIa participates in opsin transport through the photoreceptor cilium.

Authors:  X Liu; I P Udovichenko; S D Brown; K P Steel; D S Williams
Journal:  J Neurosci       Date:  1999-08-01       Impact factor: 6.167

6.  Effect of genome size on AAV vector packaging.

Authors:  Zhijian Wu; Hongyan Yang; Peter Colosi
Journal:  Mol Ther       Date:  2009-11-10       Impact factor: 11.454

7.  Human myosin VIIA responsible for the Usher 1B syndrome: a predicted membrane-associated motor protein expressed in developing sensory epithelia.

Authors:  D Weil; G Levy; I Sahly; F Levi-Acobas; S Blanchard; A El-Amraoui; F Crozet; H Philippe; M Abitbol; C Petit
Journal:  Proc Natl Acad Sci U S A       Date:  1996-04-16       Impact factor: 11.205

8.  Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.

Authors:  William W Hauswirth; Tomas S Aleman; Shalesh Kaushal; Artur V Cideciyan; Sharon B Schwartz; Lili Wang; Thomas J Conlon; Sanford L Boye; Terence R Flotte; Barry J Byrne; Samuel G Jacobson
Journal:  Hum Gene Ther       Date:  2008-10       Impact factor: 5.695

9.  Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

Authors:  Jean Bennett; Jennifer Wellman; Kathleen A Marshall; Sarah McCague; Manzar Ashtari; Julie DiStefano-Pappas; Okan U Elci; Daniel C Chung; Junwei Sun; J Fraser Wright; Dominique R Cross; Puya Aravand; Laura L Cyckowski; Jeannette L Bennicelli; Federico Mingozzi; Alberto Auricchio; Eric A Pierce; Jason Ruggiero; Bart P Leroy; Francesca Simonelli; Katherine A High; Albert M Maguire
Journal:  Lancet       Date:  2016-06-30       Impact factor: 79.321

10.  Alterations of the CIB2 calcium- and integrin-binding protein cause Usher syndrome type 1J and nonsyndromic deafness DFNB48.

Authors:  Saima Riazuddin; Inna A Belyantseva; Arnaud P J Giese; Kwanghyuk Lee; Artur A Indzhykulian; Sri Pratima Nandamuri; Rizwan Yousaf; Ghanshyam P Sinha; Sue Lee; David Terrell; Rashmi S Hegde; Rana A Ali; Saima Anwar; Paula B Andrade-Elizondo; Asli Sirmaci; Leslie V Parise; Sulman Basit; Abdul Wali; Muhammad Ayub; Muhammad Ansar; Wasim Ahmad; Shaheen N Khan; Javed Akram; Mustafa Tekin; Sheikh Riazuddin; Tiffany Cook; Elke K Buschbeck; Gregory I Frolenkov; Suzanne M Leal; Thomas B Friedman; Zubair M Ahmed
Journal:  Nat Genet       Date:  2012-09-30       Impact factor: 38.330
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  4 in total

Review 1.  Restoring Vision to the Blind with Chemical Photoswitches.

Authors:  Ivan Tochitsky; Michael A Kienzler; Ehud Isacoff; Richard H Kramer
Journal:  Chem Rev       Date:  2018-06-06       Impact factor: 60.622

2.  USH2A Gene Editing Using the CRISPR System.

Authors:  Carla Fuster-García; Gema García-García; Elisa González-Romero; Teresa Jaijo; María D Sequedo; Carmen Ayuso; Rafael P Vázquez-Manrique; José M Millán; Elena Aller
Journal:  Mol Ther Nucleic Acids       Date:  2017-08-12       Impact factor: 8.886

Review 3.  The genetic and phenotypic landscapes of Usher syndrome: from disease mechanisms to a new classification.

Authors:  Sedigheh Delmaghani; Aziz El-Amraoui
Journal:  Hum Genet       Date:  2022-03-30       Impact factor: 5.881

Review 4.  Usher Syndrome: Genetics and Molecular Links of Hearing Loss and Directions for Therapy.

Authors:  Meg Whatley; Abbie Francis; Zi Ying Ng; Xin Ee Khoh; Marcus D Atlas; Rodney J Dilley; Elaine Y M Wong
Journal:  Front Genet       Date:  2020-10-22       Impact factor: 4.599
  4 in total

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