Personalized therapeutic strategies for patients with retinitis pigmentosa

Patient-specific induced pluripotent stem cells (iPSCs) for the study and treatment of retinal degenerative diseases

(2015) Luke A. Wiley et al.   PROGRESS IN RETINAL AND EYE RESEARCH

CRISPR/Cas9-mediated genome engineering: An adeno-associated viral (AAV) vector toolbox

(2014) Elena Senís et al.   Biotechnology Journal

Induced pluripotent stem cells as custom therapeutics for retinal repair: Progress and rationale

(2014) Lynda S. Wright et al.   EXPERIMENTAL EYE RESEARCH

CEP290 gene transfer rescues Leber congenital amaurosis cellular phenotype

(2014) E R Burnight et al.   GENE THERAPY

Stem cells for investigation and treatment of inherited retinal disease

(2014) B. A. Tucker 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

Gene Therapy in Patient-specific Stem Cell Lines and a Preclinical Model of Retinitis Pigmentosa With Membrane Frizzled-related Protein Defects

(2014) Yao Li et al.   MOLECULAR THERAPY

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

The special case of gene therapy pricing

(2014) Troyen A Brennan et al.   NATURE BIOTECHNOLOGY

miRNAs 182 and 183 Are Necessary to Maintain Adult Cone Photoreceptor Outer Segments and Visual Function

(2014) Volker Busskamp et al.   NEURON

RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection

(2014) W. Hu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The use of induced pluripotent stem cells to reveal pathogenic gene mutations and explore treatments for retinitis pigmentosa

(2014) Tetsu Yoshida et al.   Molecular Brain

Human Finger-Prick Induced Pluripotent Stem Cells Facilitate the Development of Stem Cell Banking

(2014) Hong-Kee Tan et al.   Stem Cells Translational Medicine

Transplantation of Embryonic and Induced Pluripotent Stem Cell-Derived 3D Retinal Sheets into Retinal Degenerative Mice

(2014) Juthaporn Assawachananont et al.   Stem Cell Reports

One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering

(2013) Haoyi Wang et al.   CELL

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

(2013) F. Ann Ran et al.   CELL

Enhanced Efficiency of Human Pluripotent Stem Cell Genome Editing through Replacing TALENs with CRISPRs

(2013) Qiurong Ding et al.   Cell Stem Cell

Genes and mutations causing retinitis pigmentosa

(2013) S P Daiger et al.   CLINICAL GENETICS

Mid-stage intervention achieves similar efficacy as conventional early-stage treatment using gene therapy in a pre-clinical model of retinitis pigmentosa

(2013) K. J. Wert et al.   HUMAN MOLECULAR GENETICS

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

AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models

(2013) Vidyullatha Vasireddy et al.   PLoS One

Efficient multiplex biallelic zebrafish genome editing using a CRISPR nuclease system

(2013) Li-En Jao et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

(2013) A. V. Cideciyan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Reversal of end-stage retinal degeneration and restoration of visual function by photoreceptor transplantation

(2013) M. S. Singh et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

(2013) A. Aiuti et al.   SCIENCE

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Seeing the Light

(2013) A. P. Wojno et al.   Science Translational Medicine

Harnessing the CRISPR/Cas9 system to disrupt latent HIV-1 provirus

(2013) Hirotaka Ebina et al.   Scientific Reports

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

(2013) Budd A Tucker et al.   eLife

Lessons Learned from the Clinical Development and Market Authorization of Glybera

(2013) Laura M. Bryant et al.   Human Gene Therapy Clinical Development

Embryonic stem cell trials for macular degeneration: a preliminary report

(2012) Steven D Schwartz et al.   LANCET

In vivo genome editing using a high-efficiency TALEN system

(2012) Victoria M. Bedell et al.   NATURE

Restoration of vision after transplantation of photoreceptors

(2012) R. A. Pearson et al.   NATURE

Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria

(2012) G. Gasiunas et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Repair of the degenerate retina by photoreceptor transplantation

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

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

(2012) M. Jinek et al.   SCIENCE

Defining the Integration Capacity of Embryonic Stem Cell-Derived Photoreceptor Precursors

(2012) Emma L. West et al.   STEM CELLS

The Detection of Motion by Blind Subjects With the Epiretinal 60-Electrode (Argus II) Retinal Prosthesis

(2012) Jessy D. Dorn et al.   JAMA Ophthalmology

PTC124-Mediated Translational Readthrough of a Nonsense Mutation Causing Usher Syndrome Type 1C

(2011) T. Goldmann et al.   HUMAN GENE THERAPY

Modeling familial Alzheimer's disease with induced pluripotent stem cells

(2011) Takuya Yagi et al.   HUMAN MOLECULAR GENETICS

In vivo genome editing restores haemostasis in a mouse model of haemophilia

(2011) Hojun Li et al.   NATURE

Modelling the long QT syndrome with induced pluripotent stem cells

(2011) Ilanit Itzhaki et al.   NATURE

Self-organizing optic-cup morphogenesis in three-dimensional culture

(2011) Mototsugu Eiraku et al.   NATURE

Modeling Retinal Degeneration Using Patient-Specific Induced Pluripotent Stem Cells

(2011) Zi-Bing Jin et al.   PLoS One

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

Isogenic Pairs of Wild Type and Mutant Induced Pluripotent Stem Cell (iPSC) Lines from Rett Syndrome Patients as In Vitro Disease Model

(2011) Gene Ananiev et al.   PLoS One

Exome sequencing and analysis of induced pluripotent stem cells identify the cilia-related gene male germ cell-associated kinase (MAK) as a cause of retinitis pigmentosa

(2011) B. A. Tucker et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Differentiation of Induced Pluripotent Stem Cells of Swine into Rod Photoreceptors and Their Integration into the Retina

(2011) Liang Zhou et al.   STEM CELLS

Clinical Trial of Lutein in Patients With Retinitis Pigmentosa Receiving Vitamin A

(2010)   ARCHIVES OF OPHTHALMOLOGY

Blind subjects implanted with the Argus II retinal prosthesis are able to improve performance in a spatial-motor task

(2010) A. K. Ahuja et al.   BRITISH JOURNAL OF OPHTHALMOLOGY

Gene therapy rescues cone function in congenital achromatopsia

(2010) András M. Komáromy et al.   HUMAN MOLECULAR GENETICS

Patient-specific induced pluripotent stem-cell-derived models of LEOPARD syndrome

(2010) Xonia Carvajal-Vergara et al.   NATURE

A TALE nuclease architecture for efficient genome editing

(2010) Jeffrey C Miller et al.   NATURE BIOTECHNOLOGY

Transcriptional regulation of photoreceptor development and homeostasis in the mammalian retina

(2010) Anand Swaroop et al.   NATURE REVIEWS NEUROSCIENCE

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

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

iPS Cells: Mapping the Policy Issues

(2009) Amy Zarzeczny et al.   CELL

Transplantation of Human Embryonic Stem Cell-Derived Photoreceptors Restores Some Visual Function in Crx-Deficient Mice

(2009) Deepak A. Lamba et al.   Cell Stem Cell

Generation of Human Induced Pluripotent Stem Cells by Direct Delivery of Reprogramming Proteins

(2009) Dohoon Kim et al.   Cell Stem Cell

Generation of red fluorescent protein transgenic dogs

(2009) So Gun Hong et al.   GENESIS

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

(2009) Albert M Maguire et al.   LANCET

A Combined Chemical and Genetic Approach for the Generation of Induced Pluripotent Stem Cells

(2008) Yan Shi et al.   Cell Stem Cell

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

Stimulation of the insulin/mTOR pathway delays cone death in a mouse model of retinitis pigmentosa

(2008) Claudio Punzo et al.   NATURE NEUROSCIENCE

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