Gene therapy into photoreceptors and Müller glial cells restores retinal structure and function in CRB1 retinitis pigmentosa mouse models

CRB2 acts as a modifying factor of CRB1-related retinal dystrophies in mice

(2014) Lucie P. Pellissier et al.   HUMAN MOLECULAR GENETICS

Targeted ablation of Crb2 in photoreceptor cells induces retinitis pigmentosa

(2014) Celso Henrique Alves et al.   HUMAN MOLECULAR GENETICS

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial

(2014) Robert E MacLaren et al.   LANCET

Whole Exome Sequencing Identifies CRB1 Defect in an Unusual Maculopathy Phenotype

(2014) Stephen H. Tsang et al.   OPHTHALMOLOGY

The CRB1 and adherens junction complex proteins in retinal development and maintenance

(2014) Celso Henrique Alves et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Specific tools for targeting and expression in Müller glial cells

(2014) Lucie P Pellissier et al.   Molecular Therapy-Methods & Clinical Development

Microarray and Morphological Analysis of Early Postnatal CRB2 Mutant Retinas on a Pure C57BL/6J Genetic Background

(2013) Celso Henrique Alves et al.   PLoS One

Targeted Ablation of Crb1 and Crb2 in Retinal Progenitor Cells Mimics Leber Congenital Amaurosis

(2013) Lucie P. Pellissier et al.   PLoS Genetics

Immunology of AAV-Mediated Gene Transfer in the Eye

(2013) Keirnan Willett et al.   Frontiers in Immunology

Crb Apical Polarity Proteins Maintain Zebrafish Retinal Cone Mosaics via Intercellular Binding of Their Extracellular Domains

(2012) Jian Zou et al.   DEVELOPMENTAL CELL

Loss of CRB2 in the mouse retina mimics human retinitis pigmentosa due to mutations in the CRB1 gene

(2012) Celso Henrique Alves et al.   HUMAN MOLECULAR GENETICS

Clinical applications of retinal gene therapy

(2012) Daniel M. Lipinski et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9

(2011) D Dalkara et al.   GENE THERAPY

CRB1mutations in inherited retinal dystrophies

(2011) Kinga Bujakowska et al.   HUMAN MUTATION

Retro-orbital injections in mice

(2011) Tal Yardeni et al.   LAB ANIMAL

Multiple domains in the Crumbs Homolog 2a (Crb2a) protein are required for regulating rod photoreceptor size

(2010) Ya-Chu Hsu et al.   BMC CELL BIOLOGY

GFAP-Driven GFP Expression in Activated Mouse Müller Glial Cells Aligning Retinal Blood Vessels Following Intravitreal Injection of AAV2/6 Vectors

(2010) Wendy M. Aartsen et al.   PLoS One

A Novel Adeno-Associated Viral Variant for Efficient and Selective Intravitreal Transduction of Rat Müller Cells

(2009) Ryan R. Klimczak et al.   PLoS One

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