Stem cells for investigation and treatment of inherited retinal disease

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Double Nicking by RNA-Guided CRISPR Cas9 for Enhanced Genome Editing Specificity

(2013) F. Ann Ran et al.   CELL

Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus

(2013) V S Lopes et al.   GENE THERAPY

Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo

(2013) Tanya Tolmachova et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

Photoreceptor precursors derived from three-dimensional embryonic stem cell cultures integrate and mature within adult degenerate retina

(2013) Anai Gonzalez-Cordero et al.   NATURE BIOTECHNOLOGY

CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering

(2013) Prashant Mali et al.   NATURE BIOTECHNOLOGY

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

CRISPR RNA–guided activation of endogenous human genes

(2013) Morgan L Maeder et al.   NATURE METHODS

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

RNA-programmed genome editing in human cells

(2013) Martin Jinek et al.   eLife

Patient-specific iPSC-derived photoreceptor precursor cells as a means to investigate retinitis pigmentosa

(2013) Budd A Tucker et al.   eLife

Self-Formation of Optic Cups and Storable Stratified Neural Retina from Human ESCs

(2012) Tokushige Nakano et al.   Cell Stem Cell

RPE65 gene therapy slows cone loss in Rpe65-deficient dogs

(2012) F M Mowat et al.   GENE THERAPY

AAV-Mediated Gene Therapy in the Guanylate Cyclase (RetGC1/RetGC2) Double Knockout Mouse Model of Leber Congenital Amaurosis

(2012) Sanford L. Boye et al.   HUMAN GENE THERAPY

Gene therapy restores vision and delays degeneration in the CNGB1-/- mouse model of retinitis pigmentosa

(2012) S. Koch et al.   HUMAN MOLECULAR GENETICS

CHM/REP1 cDNA delivery by lentiviral vectors provides functional expression of the transgene in the retinal pigment epithelium of choroideremia mice

(2012) Tanya Tolmachova et al.   JOURNAL OF GENE MEDICINE

Expression of Wild-Type Rp1 Protein in Rp1 Knock-in Mice Rescues the Retinal Degeneration Phenotype

(2012) Qin Liu et al.   PLoS One

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 delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model

(2012) H. Yu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Use of a Synthetic Xeno-Free Culture Substrate for Induced Pluripotent Stem Cell Induction and Retinal Differentiation

(2012) Budd A. Tucker et al.   Stem Cells Translational Medicine

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

The Use of Induced Pluripotent Stem Cells in Drug Development

(2011) H Inoue et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Long-Term Preservation of Cones and Improvement in Visual Function Following Gene Therapy in a Mouse Model of Leber Congenital Amaurosis Caused by Guanylate Cyclase-1 Deficiency

(2011) Marija Mihelec et al.   HUMAN GENE THERAPY

Accumulation of murine subretinal macrophages: effects of age, pigmentation and CX3CR1

(2011) Holly R. Chinnery et al.   NEUROBIOLOGY OF AGING

Transplantation of Adult Mouse iPS Cell-Derived Photoreceptor Precursors Restores Retinal Structure and Function in Degenerative Mice

(2011) Budd A. Tucker et al.   PLoS One

Anti-Aβ Drug Screening Platform Using Human iPS Cell-Derived Neurons for the Treatment of Alzheimer's Disease

(2011) Naoki Yahata et al.   PLoS One

Gene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model

(2011) D. L. Simons et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Optic Vesicle-like Structures Derived from Human Pluripotent Stem Cells Facilitate a Customized Approach to Retinal Disease Treatment

(2011) Jason S. Meyer et al.   STEM CELLS

Brief Exposure to Damaging Light Causes Focal Recruitment of Macrophages, and Long-Term Destabilization of Photoreceptors in the Albino Rat Retina

(2010) Matt Rutar et al.   CURRENT EYE RESEARCH

Generation, Purification and Transplantation of Photoreceptors Derived from Human Induced Pluripotent Stem Cells

(2010) Deepak A. Lamba et al.   PLoS One

Growth kinetics and transplantation of human retinal progenitor cells

(2009) Unber Aftab et al.   EXPERIMENTAL EYE RESEARCH

Localization of complement 1 inhibitor (C1INH/SERPING1) in human eyes with age-related macular degeneration

(2009) Robert F. Mullins et al.   EXPERIMENTAL EYE RESEARCH

In vitro differentiation of retinal cells from human pluripotent stem cells by small-molecule induction

(2009) F. Osakada et al.   JOURNAL OF CELL SCIENCE

Stepwise differentiation of pluripotent stem cells into retinal cells

(2009) Fumitaka Osakada et al.   Nature Protocols

Modeling early retinal development with human embryonic and induced pluripotent stem cells

(2009) J. S. Meyer et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Retinal cells integrate into the outer nuclear layer and differentiate into mature photoreceptors after subretinal transplantation into adult mice

(2008) Udo Bartsch et al.   EXPERIMENTAL EYE RESEARCH

Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer

(2008) Jeannette Bennicelli et al.   MOLECULAR THERAPY

Toward the generation of rod and cone photoreceptors from mouse, monkey and human embryonic stem cells

(2008) Fumitaka Osakada et al.   NATURE BIOTECHNOLOGY