The Lrat−/− Rat: CRISPR/Cas9 Construction and Phenotyping of a New Animal Model for Retinitis Pigmentosa

Importin β1 mediates nuclear import of the factors associated with nonsense-mediated RNA decay

(2021) Jianran Hu et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

A Systematic Review on Transplantation Studies of the Retinal Pigment Epithelium in Animal Models

(2020) Céline Koster et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Voretigene Neparvovec: A Review in RPE65 Mutation-Associated Inherited Retinal Dystrophy

(2020) Connie Kang et al.   Molecular Diagnosis & Therapy

A Premature Termination Codon Mutation in MYBPC3 Causes Hypertrophic Cardiomyopathy via Chronic Activation of Nonsense-Mediated Decay

(2019) Timon Seeger et al.   CIRCULATION

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials

(2019) Albert M. Maguire et al.   OPHTHALMOLOGY

Long-Term Follow-Up of Retinal Degenerations Associated With LRAT Mutations and Their Comparability to Phenotypes Associated With RPE65 Mutations

(2019) Mays Talib et al.   Translational Vision Science & Technology

AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression

(2019) Kun Ding et al.   JOURNAL OF CLINICAL INVESTIGATION

Optical Coherence Tomography of Animal Models of Retinitis Pigmentosa: From Animal Studies to Clinical Applications

(2019) Mitsuru Nakazawa et al.   Biomed Research International

Successful production of genome-edited rats by the rGONAD method

(2018) Tomoe Kobayashi et al.   BMC BIOTECHNOLOGY

Gene therapy for RPE65-related retinal disease

(2018) Virginia Miraldi Utz et al.   OPHTHALMIC GENETICS

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

Human ESC–derived retinal epithelial cell sheets potentiate rescue of photoreceptor cell loss in rats with retinal degeneration

(2017) Karim Ben M’Barek et al.   Science Translational Medicine

Generation of gene-edited rats by delivery of CRISPR/Cas9 protein and donor DNA into intact zygotes using electroporation

(2017) Séverine Remy et al.   Scientific Reports

Evaluation of Retinal Function and Morphology of the Pink-Eyed Royal College of Surgeons (RCS) Rat: A Comparative Study of in Vivo and in Vitro Methods

(2016) Sarah Rösch et al.   CURRENT EYE RESEARCH

Rodent models in neuroscience research: is it a rat race?

(2016) Bart Ellenbroek et al.   Disease Models & Mechanisms

A RATional choice for translational research?

(2016) Tim Aitman et al.   Disease Models & Mechanisms

Retinoid Homeostatic Gene Expression in Liver, Lung and Kidney: Ontogeny and Response to Vitamin A-Retinoic Acid (VARA) Supplementation from Birth to Adult Age

(2016) Sarah A. Owusu et al.   PLoS One

Ultrathin Polyimide Membrane as Cell Carrier for Subretinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigment Epithelium

(2015) Tanja Ilmarinen et al.   PLoS One

Safety and Proof-of-Concept Study of Oral QLT091001 in Retinitis Pigmentosa Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT)

(2015) Hendrik P. N. Scholl et al.   PLoS One

Position of premature termination codons determines susceptibility of hERG mutations to nonsense-mediated mRNA decay in long QT syndrome

(2014) Qiuming Gong et al.   GENE

Oral 9-cis retinoid for childhood blindness due to Leber congenital amaurosis caused by RPE65 or LRAT mutations: an open-label phase 1b trial

(2014) Robert K Koenekoop et al.   LANCET

Tumorigenicity Studies of Induced Pluripotent Stem Cell (iPSC)-Derived Retinal Pigment Epithelium (RPE) for the Treatment of Age-Related Macular Degeneration

(2014) Hoshimi Kanemura et al.   PLoS One

Characterization of Human Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium Cell Sheets Aiming for Clinical Application

(2014) Hiroyuki Kamao et al.   Stem Cell Reports

A murine Zic3 transcript with a premature termination codon evades nonsense-mediated decay during axis formation

(2013) J. N. Ahmed et al.   Disease Models & Mechanisms

Retinal Pigmented Epithelial Cells Obtained from Human Induced Pluripotent Stem Cells Possess Functional Visual Cycle Enzymesin Vitroandin Vivo

(2013) Tadao Maeda et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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(2013) Yao Li et al.   MOLECULAR MEDICINE

A Homozygous Frameshift Mutation in LRAT Causes Retinitis Punctata Albescens

(2012) Karin W. Littink et al.   OPHTHALMOLOGY

The changes of potassium currents in RCS rat Müller cell during retinal degeneration

(2011) TongTao Zhao et al.   BRAIN RESEARCH

Nonsense-mediated mRNA decay caused by a frameshift mutation in a large kindred of type 2 long QT syndrome

(2011) Ignatius Gerardo Zarraga et al.   HEART RHYTHM

Gene Therapy Regenerates Protein Expression in Cone Photoreceptors in Rpe65R91W/R91W Mice

(2011) Corinne Kostic et al.   PLoS One

Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency

(2010) J Pang et al.   GENE THERAPY

Transplantation of tissue-engineered retinal pigment epithelial cell sheets in a rabbit model

(2008) Naoko Yaji et al.   BIOMATERIALS

Leber congenital amaurosis: Genes, proteins and disease mechanisms

(2008) Anneke I. den Hollander et al.   PROGRESS IN RETINAL AND EYE RESEARCH