Recombinant AAV-Mediated BEST1 Transfer to the Retinal Pigment Epithelium: Analysis of Serotype-Dependent Retinal Effects

Frequency, Genotype, and Clinical Spectrum of Best Vitelliform Macular Dystrophy: Data From a National Center in Denmark

(2012) Hanna Bitner et al.   AMERICAN JOURNAL OF OPHTHALMOLOGY

Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa

(2012) W. A. Beltran et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

Disease-associated missense mutations in bestrophin-1 affect cellular trafficking and anion conductance

(2011) Vladimir M. Milenkovic et al.   JOURNAL OF CELL SCIENCE

Genetic and phenotypic variations of inherited retinal diseases in dogs: the power of within- and across-breed studies

(2011) Keiko Miyadera et al.   MAMMALIAN GENOME

Interaction of Bestrophin-1 and Ca2+ Channel β-Subunits: Identification of New Binding Domains on the Bestrophin-1 C-Terminus

(2011) Vladimir M. Milenkovic et al.   PLoS One

AUTOSOMAL RECESSIVE VITELLIFORM MACULAR DYSTROPHY IN A LARGE COHORT OF VITELLIFORM MACULAR DYSTROPHY PATIENTS

(2011) Tyson R Kinnick et al.   RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES

rAAV2/5 gene-targeting to rods:dose-dependent efficiency and complications associated with different promoters

(2010) W A Beltran et al.   GENE THERAPY

The spectrum of ocular phenotypes caused by mutations in the BEST1 gene

(2009) Camiel J.F. Boon et al.   PROGRESS IN RETINAL AND EYE RESEARCH

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

(2008) William W. Hauswirth et al.   HUMAN GENE THERAPY

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

(2008) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Molecular Physiology of Bestrophins: Multifunctional Membrane Proteins Linked to Best Disease and Other Retinopathies

(2008) H. Criss Hartzell et al.   PHYSIOLOGICAL REVIEWS