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Literature DB >> 25605753

Gene Therapy for the Retinal Degeneration of Usher Syndrome Caused by Mutations in MYO7A.

Abstract

Usher syndrome is a deaf-blindness disorder. One of the subtypes, Usher 1B, is caused by loss of function of the gene encoding the unconventional myosin, MYO7A. A variety of different viral-based delivery approaches have been tested for retinal gene therapy to prevent the blindness of Usher 1B, and a clinical trial based on one of these approaches has begun. This review evaluates the different approaches.

Entities: Disease Gene

Mesh：

Substances：

Year: 2015 PMID： 25605753 PMCID： PMC4448706 DOI： 10.1101/cshperspect.a017319

Source DB: PubMed Journal: Cold Spring Harb Perspect Med ISSN： 2157-1422 Impact factor: 6.915

48 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. Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector.

Authors: H Miyoshi; M Takahashi; F H Gage; I M Verma
Journal: Proc Natl Acad Sci U S A Date: 1997-09-16 Impact factor: 11.205

3. Lentiviral vector-mediated gene transfer in adult mouse photoreceptors is impaired by the presence of a physical barrier.

Authors: O Grüter; C Kostic; S V Crippa; M-T R Perez; L Zografos; D F Schorderet; F L Munier; Y Arsenijevic
Journal: Gene Ther Date: 2005-06 Impact factor: 5.250

4. Analysis of the linkage of MYRIP and MYO7A to melanosomes by RAB27A in retinal pigment epithelial cells.

Authors: Adriana E Klomp; Karen Teofilo; Erin Legacki; David S Williams
Journal: Cell Motil Cytoskeleton Date: 2007-06

5. Characterization of genome integrity for oversized recombinant AAV vector.

Authors: Biao Dong; Hiroyuki Nakai; Weidong Xiao
Journal: Mol Ther Date: 2009-11-10 Impact factor: 11.454

6. Abnormal phagocytosis by retinal pigmented epithelium that lacks myosin VIIa, the Usher syndrome 1B protein.

Authors: Daniel Gibbs; Junko Kitamoto; David S Williams
Journal: Proc Natl Acad Sci U S A Date: 2003-05-12 Impact factor: 11.205

7. Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy.

Authors: J Kong; S-R Kim; K Binley; I Pata; K Doi; J Mannik; J Zernant-Rajang; O Kan; S Iqball; S Naylor; J R Sparrow; P Gouras; R Allikmets
Journal: Gene Ther Date: 2008-05-08 Impact factor: 5.250

8. Lentiviral gene replacement therapy of retinas in a mouse model for Usher syndrome type 1B.

Authors: T Hashimoto; D Gibbs; C Lillo; S M Azarian; E Legacki; X-M Zhang; X-J Yang; D S Williams
Journal: Gene Ther Date: 2007-02-01 Impact factor: 4.184

9. Myosin7a deficiency results in reduced retinal activity which is improved by gene therapy.

Authors: Pasqualina Colella; Andrea Sommella; Elena Marrocco; Umberto Di Vicino; Elena Polishchuk; Marina Garcia Garrido; Mathias W Seeliger; Roman Polishchuk; Alberto Auricchio
Journal: PLoS One Date: 2013-08-26 Impact factor: 3.240

10. Localization of Usher 1 proteins to the photoreceptor calyceal processes, which are absent from mice.

Authors: Iman Sahly; Eric Dufour; Cataldo Schietroma; Vincent Michel; Amel Bahloul; Isabelle Perfettini; Elise Pepermans; Amrit Estivalet; Diane Carette; Asadollah Aghaie; Inga Ebermann; Andrea Lelli; Maria Iribarne; Jean-Pierre Hardelin; Dominique Weil; José-Alain Sahel; Aziz El-Amraoui; Christine Petit
Journal: J Cell Biol Date: 2012-10-08 Impact factor: 10.539

4 in total