scholarly article | Q13442814 |
P50 | author | Luke A. Wiley | Q55127324 |
Robert F. Mullins | Q55272334 | ||
Elliott H Sohn | Q58398731 | ||
Audrey A Tran | Q91024721 | ||
Edwin M. Stone | Q37381855 | ||
Budd A. Tucker | Q38326878 | ||
P2093 | author name string | George Q Daley | |
Jason W Ross | |||
Manav Gupta | |||
Chunhua Jiao | |||
Robinson Triboulet | |||
Thorsten M Schlaeger | |||
Erin R Burnight | |||
Kristin R Anfinson | |||
Malavika K Adur | |||
Jeremy M Hoffmann | |||
Darcey L Klaahsen | |||
Jeaneen L Andorf | |||
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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 | Q24320041 | ||
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Mutations in the CEP290 (NPHP6) gene are a frequent cause of Leber congenital amaurosis | Q24678616 | ||
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Spectrum of NPHP6/CEP290 mutations in Leber congenital amaurosis and delineation of the associated phenotype | Q28292007 | ||
Centrosomal-ciliary gene CEP290/NPHP6 mutations result in blindness with unexpected sparing of photoreceptors and visual brain: implications for therapy of Leber congenital amaurosis | Q28305398 | ||
Precise gene modification mediated by TALEN and single-stranded oligodeoxynucleotides in human cells | Q28541724 | ||
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DNA targeting specificity of RNA-guided Cas9 nucleases | Q29615793 | ||
Retinitis pigmentosa | Q29616538 | ||
CRISPR RNA-guided activation of endogenous human genes | Q29617072 | ||
Embryonic stem cell trials for macular degeneration: a preliminary report | Q29620714 | ||
Generation, purification and transplantation of photoreceptors derived from human induced pluripotent stem cells | Q33526318 | ||
A linkage survey of 20 dominant retinitis pigmentosa families: frequencies of the nine known loci and evidence for further heterogeneity | Q33679975 | ||
Effect of genome size on AAV vector packaging | Q33730405 | ||
Minimum requirements for efficient transduction of dividing and nondividing cells by feline immunodeficiency virus vectors | Q33814215 | ||
Differential macular morphology in patients with RPE65-, CEP290-, GUCY2D-, and AIPL1-related Leber congenital amaurosis | Q33845544 | ||
Allele-specific genome editing and correction of disease-associated phenotypes in rats using the CRISPR-Cas platform | Q33851650 | ||
Transplantation of adult mouse iPS cell-derived photoreceptor precursors restores retinal structure and function in degenerative mice | Q33895271 | ||
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Humanized Mice Reveal Differential Immunogenicity of Cells Derived from Autologous Induced Pluripotent Stem Cells | Q34044685 | ||
The cellular fate of mutant rhodopsin: quality control, degradation and aggresome formation. | Q34135152 | ||
Blindness in children: control priorities and research opportunities | Q34345377 | ||
GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases | Q34454104 | ||
High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects. | Q34507554 | ||
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Adult donor rod photoreceptors integrate into the mature mouse retina | Q35221330 | ||
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The nature of dominant mutations of rhodopsin and implications for gene therapy | Q35566986 | ||
Enhanced differentiation and delivery of mouse retinal progenitor cells using a micropatterned biodegradable thin-film polycaprolactone scaffold | Q39568788 | ||
Use of a synthetic xeno-free culture substrate for induced pluripotent stem cell induction and retinal differentiation | Q40883512 | ||
Characterization of Staphylococcus aureus Cas9: a smaller Cas9 for all-in-one adeno-associated virus delivery and paired nickase applications. | Q41105167 | ||
Genetic blindness: current concepts in the pathogenesis of human outer retinal dystrophies | Q41737723 | ||
Transplantation of human embryonic stem cell-derived photoreceptors restores some visual function in Crx-deficient mice | Q41927703 | ||
CEP290 gene transfer rescues Leber congenital amaurosis cellular phenotype. | Q42051635 | ||
Unbiased detection of off-target cleavage by CRISPR-Cas9 and TALENs using integrase-defective lentiviral vectors. | Q42174678 | ||
A comparison of neural differentiation and retinal transplantation with bone marrow-derived cells and retinal progenitor cells | Q48410413 | ||
c-Kit⁺ cells isolated from human fetal retinas represent a new population of retinal progenitor cells. | Q53527228 | ||
Directed differentiation of human induced pluripotent stem cells: a retina perspective. | Q54449904 | ||
Abstract | Q57784395 | ||
Preservation of the Inner Retina in Retinitis Pigmentosa | Q64117612 | ||
Preservation of inner retinal responses in the aged Royal College of Surgeons rat. Evidence against glutamate excitotoxicity in photoreceptor degeneration | Q72016842 | ||
Generation of an inbred miniature pig model of retinitis pigmentosa. | Q35797314 | ||
Transplantation of human embryonic stem cell-derived retinal tissue in two primate models of retinal degeneration | Q35877231 | ||
Inner retinal preservation in rat models of retinal degeneration implanted with subretinal photovoltaic arrays. | Q36051555 | ||
Broadening the targeting range of Staphylococcus aureus CRISPR-Cas9 by modifying PAM recognition | Q36398694 | ||
North Carolina Macular Dystrophy Is Caused by Dysregulation of the Retinal Transcription Factor PRDM13. | Q36414650 | ||
CRISPR/Cas9 DNA cleavage at SNP-derived PAM enables both in vitro and in vivo KRT12 mutation-specific targeting | Q36442476 | ||
ssODN-mediated knock-in with CRISPR-Cas for large genomic regions in zygotes | Q36529479 | ||
Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL Nucleases | Q36675286 | ||
A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells | Q36782735 | ||
Nucleotide-resolution DNA double-strand break mapping by next-generation sequencing | Q36834533 | ||
Transplantation of Photoreceptor Precursors Isolated via a Cell Surface Biomarker Panel From Embryonic Stem Cell-Derived Self-Forming Retina. | Q36886550 | ||
Identification and Correction of Mechanisms Underlying Inherited Blindness in Human iPSC-Derived Optic Cups. | Q36985099 | ||
In vivo gene editing in dystrophic mouse muscle and muscle stem cells | Q37045850 | ||
Subretinal implantation of retinal pigment epithelial cells derived from human embryonic stem cells: improved survival when implanted as a monolayer | Q37053158 | ||
Patient-specific iPSC-derived photoreceptor precursor cells as a means to investigate retinitis pigmentosa | Q37126951 | ||
cGMP production of patient-specific iPSCs and photoreceptor precursor cells to treat retinal degenerative blindness | Q37137455 | ||
Search for a correlation between telomere length and severity of retinitis pigmentosa due to the dominant rhodopsin Pro23His mutation | Q37140625 | ||
Subretinal gene therapy of mice with Bardet-Biedl syndrome type 1. | Q37167396 | ||
Photoreceptor precursors derived from three-dimensional embryonic stem cell cultures integrate and mature within adult degenerate retina. | Q37304490 | ||
Non-exomic and synonymous variants in ABCA4 are an important cause of Stargardt disease. | Q37346967 | ||
Ocular gene therapy: current progress and future prospects | Q37355359 | ||
Human retinal progenitor cell transplantation preserves vision | Q37622521 | ||
Autosomal recessive retinitis pigmentosa due to ABCA4 mutations: clinical, pathologic, and molecular characterization. | Q37716568 | ||
Recent advances in targeted genome engineering in mammalian systems | Q38025056 | ||
Stem cells for investigation and treatment of inherited retinal disease | Q38197508 | ||
Approaches to cell delivery: substrates and scaffolds for cell therapy | Q38204642 | ||
Genome-wide translocation sequencing reveals mechanisms of chromosome breaks and rearrangements in B cells | Q38626707 | ||
Two-photon polymerization for production of human iPSC-derived retinal cell grafts. | Q38738347 | ||
P4510 | describes a project that uses | CRISPR-Cas method | Q17310682 |
P433 | issue | 9 | |
P921 | main subject | Cas9 | Q16965677 |
CRISPR-Cas method | Q17310682 | ||
CRISPR | Q412563 | ||
P304 | page(s) | 1999-2013 | |
P577 | publication date | 2017-06-12 | |
P1433 | published in | Molecular Therapy | Q15762400 |
P1476 | title | Using CRISPR-Cas9 to Generate Gene-Corrected Autologous iPSCs for the Treatment of Inherited Retinal Degeneration | |
P478 | volume | 25 |
Q90298681 | Allele-Specific CRISPR-Cas9 Genome Editing of the Single-Base P23H Mutation for Rhodopsin-Associated Dominant Retinitis Pigmentosa |
Q90481753 | Allele-specific genome targeting in the development of precision medicine |
Q88627116 | CRISPR GENOME SURGERY IN THE RETINA IN LIGHT OF OFF-TARGETING |
Q52430638 | CRISPR-Cas9-Based Genome Editing of Human Induced Pluripotent Stem Cells. |
Q90298722 | CRISPR-Cas9-Mediated Correction of the 1.02 kb Common Deletion in CLN3 in Induced Pluripotent Stem Cells from Patients with Batten Disease |
Q55091758 | Cellular regeneration strategies for macular degeneration: past, present and future. |
Q64937510 | Correction of NR2E3 Associated Enhanced S-cone Syndrome Patient-specific iPSCs using CRISPR-Cas9. |
Q52568713 | Feeder-free differentiation of cells exhibiting characteristics of corneal endothelium from human induced pluripotent stem cells. |
Q100512500 | Gene therapy and gene correction: targets, progress, and challenges for treating human diseases |
Q92276489 | Genome Editing as a Treatment for the Most Prevalent Causative Genes of Autosomal Dominant Retinitis Pigmentosa |
Q92464626 | Genome Editing in Patient iPSCs Corrects the Most Prevalent USH2A Mutations and Reveals Intriguing Mutant mRNA Expression Profiles |
Q55000684 | Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy. |
Q58554380 | Human eye conditions: insights from the fly eye |
Q90152961 | Leber Congenital Amaurosis (LCA): Potential for Improvement of Vision |
Q64275334 | Mechanisms Underlying the Visual Benefit of Cell Transplantation for the Treatment of Retinal Degenerations |
Q92988365 | Molecular Therapies for Inherited Retinal Diseases-Current Standing, Opportunities and Challenges |
Q59354224 | Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector |
Q89115884 | NAD+ and sirtuins in retinal degenerative diseases: A look at future therapies |
Q90027776 | Optimizing Donor Cellular Dissociation and Subretinal Injection Parameters for Stem Cell-Based Treatments |
Q99417940 | Precise allele-specific genome editing by spatiotemporal control of CRISPR-Cas9 via pronuclear transplantation |
Q92301681 | Progress in Gene Therapy for Rhodopsin Autosomal Dominant Retinitis Pigmentosa |
Q88957422 | Regenerating Eye Tissues to Preserve and Restore Vision |
Q55387849 | Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA. |
Q45873271 | The molecular and cellular basis of rhodopsin retinitis pigmentosa reveals potential strategies for therapy |
Q45874106 | The pigmented epithelium, a bright partner against photoreceptor degeneration |
Q89791404 | Transplantation of iPSC-TM stimulates division of trabecular meshwork cells in human eyes |
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