Using CRISPR-Cas9 to Generate Gene-Corrected Autologous iPSCs for the Treatment of Inherited Retinal Degeneration

scientific article published on 12 June 2017

Using CRISPR-Cas9 to Generate Gene-Corrected Autologous iPSCs for the Treatment of Inherited Retinal Degeneration is …
instance of (P31):
scholarly articleQ13442814

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P356DOI10.1016/J.YMTHE.2017.05.015
P932PMC publication ID5589061
P698PubMed publication ID28619647

P50authorLuke A. WileyQ55127324
Robert F. MullinsQ55272334
Elliott H SohnQ58398731
Audrey A TranQ91024721
Edwin M. StoneQ37381855
Budd A. TuckerQ38326878
P2093author name stringGeorge 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
P2860cites workMechanisms of cell death in rhodopsin retinitis pigmentosa: implications for therapyQ21710681
Genome engineering using the CRISPR-Cas9 systemQ22122027
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 pigmentosaQ24320041
RNA-guided human genome engineering via Cas9Q24598394
Multiplex genome engineering using CRISPR/Cas systemsQ24609428
Mutations in the CEP290 (NPHP6) gene are a frequent cause of Leber congenital amaurosisQ24678616
RNA-programmed genome editing in human cellsQ28044562
Spectrum of NPHP6/CEP290 mutations in Leber congenital amaurosis and delineation of the associated phenotypeQ28292007
Centrosomal-ciliary gene CEP290/NPHP6 mutations result in blindness with unexpected sparing of photoreceptors and visual brain: implications for therapy of Leber congenital amaurosisQ28305398
Precise gene modification mediated by TALEN and single-stranded oligodeoxynucleotides in human cellsQ28541724
Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificityQ29615792
DNA targeting specificity of RNA-guided Cas9 nucleasesQ29615793
Retinitis pigmentosaQ29616538
CRISPR RNA-guided activation of endogenous human genesQ29617072
Embryonic stem cell trials for macular degeneration: a preliminary reportQ29620714
Generation, purification and transplantation of photoreceptors derived from human induced pluripotent stem cellsQ33526318
A linkage survey of 20 dominant retinitis pigmentosa families: frequencies of the nine known loci and evidence for further heterogeneityQ33679975
Effect of genome size on AAV vector packagingQ33730405
Minimum requirements for efficient transduction of dividing and nondividing cells by feline immunodeficiency virus vectorsQ33814215
Differential macular morphology in patients with RPE65-, CEP290-, GUCY2D-, and AIPL1-related Leber congenital amaurosisQ33845544
Allele-specific genome editing and correction of disease-associated phenotypes in rats using the CRISPR-Cas platformQ33851650
Transplantation of adult mouse iPS cell-derived photoreceptor precursors restores retinal structure and function in degenerative miceQ33895271
In vivo genome editing using Staphylococcus aureus Cas9Q34043628
Humanized Mice Reveal Differential Immunogenicity of Cells Derived from Autologous Induced Pluripotent Stem CellsQ34044685
The cellular fate of mutant rhodopsin: quality control, degradation and aggresome formation.Q34135152
Blindness in children: control priorities and research opportunitiesQ34345377
GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleasesQ34454104
High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects.Q34507554
Retinal repair by transplantation of photoreceptor precursorsQ34580415
Efficient generation of retinal progenitor cells from human embryonic stem cells.Q35036440
Adult donor rod photoreceptors integrate into the mature mouse retinaQ35221330
Enhanced homology-directed human genome engineering by controlled timing of CRISPR/Cas9 deliveryQ35247829
The nature of dominant mutations of rhodopsin and implications for gene therapyQ35566986
Enhanced differentiation and delivery of mouse retinal progenitor cells using a micropatterned biodegradable thin-film polycaprolactone scaffoldQ39568788
Use of a synthetic xeno-free culture substrate for induced pluripotent stem cell induction and retinal differentiationQ40883512
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 dystrophiesQ41737723
Transplantation of human embryonic stem cell-derived photoreceptors restores some visual function in Crx-deficient miceQ41927703
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 cellsQ48410413
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
AbstractQ57784395
Preservation of the Inner Retina in Retinitis PigmentosaQ64117612
Preservation of inner retinal responses in the aged Royal College of Surgeons rat. Evidence against glutamate excitotoxicity in photoreceptor degenerationQ72016842
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 degenerationQ35877231
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 recognitionQ36398694
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 targetingQ36442476
ssODN-mediated knock-in with CRISPR-Cas for large genomic regions in zygotesQ36529479
Evaluation of TCR Gene Editing Achieved by TALENs, CRISPR/Cas9, and megaTAL NucleasesQ36675286
A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle CellsQ36782735
Nucleotide-resolution DNA double-strand break mapping by next-generation sequencingQ36834533
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 cellsQ37045850
Subretinal implantation of retinal pigment epithelial cells derived from human embryonic stem cells: improved survival when implanted as a monolayerQ37053158
Patient-specific iPSC-derived photoreceptor precursor cells as a means to investigate retinitis pigmentosaQ37126951
cGMP production of patient-specific iPSCs and photoreceptor precursor cells to treat retinal degenerative blindnessQ37137455
Search for a correlation between telomere length and severity of retinitis pigmentosa due to the dominant rhodopsin Pro23His mutationQ37140625
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 prospectsQ37355359
Human retinal progenitor cell transplantation preserves visionQ37622521
Autosomal recessive retinitis pigmentosa due to ABCA4 mutations: clinical, pathologic, and molecular characterization.Q37716568
Recent advances in targeted genome engineering in mammalian systemsQ38025056
Stem cells for investigation and treatment of inherited retinal diseaseQ38197508
Approaches to cell delivery: substrates and scaffolds for cell therapyQ38204642
Genome-wide translocation sequencing reveals mechanisms of chromosome breaks and rearrangements in B cellsQ38626707
Two-photon polymerization for production of human iPSC-derived retinal cell grafts.Q38738347
P4510describes a project that usesCRISPR-Cas methodQ17310682
P433issue9
P921main subjectCas9Q16965677
CRISPR-Cas methodQ17310682
CRISPRQ412563
P304page(s)1999-2013
P577publication date2017-06-12
P1433published inMolecular TherapyQ15762400
P1476titleUsing CRISPR-Cas9 to Generate Gene-Corrected Autologous iPSCs for the Treatment of Inherited Retinal Degeneration
P478volume25

Reverse relations

cites work (P2860)
Q90298681Allele-Specific CRISPR-Cas9 Genome Editing of the Single-Base P23H Mutation for Rhodopsin-Associated Dominant Retinitis Pigmentosa
Q90481753Allele-specific genome targeting in the development of precision medicine
Q88627116CRISPR GENOME SURGERY IN THE RETINA IN LIGHT OF OFF-TARGETING
Q52430638CRISPR-Cas9-Based Genome Editing of Human Induced Pluripotent Stem Cells.
Q90298722CRISPR-Cas9-Mediated Correction of the 1.02 kb Common Deletion in CLN3 in Induced Pluripotent Stem Cells from Patients with Batten Disease
Q55091758Cellular regeneration strategies for macular degeneration: past, present and future.
Q64937510Correction of NR2E3 Associated Enhanced S-cone Syndrome Patient-specific iPSCs using CRISPR-Cas9.
Q52568713Feeder-free differentiation of cells exhibiting characteristics of corneal endothelium from human induced pluripotent stem cells.
Q100512500Gene therapy and gene correction: targets, progress, and challenges for treating human diseases
Q92276489Genome Editing as a Treatment for the Most Prevalent Causative Genes of Autosomal Dominant Retinitis Pigmentosa
Q92464626Genome Editing in Patient iPSCs Corrects the Most Prevalent USH2A Mutations and Reveals Intriguing Mutant mRNA Expression Profiles
Q55000684Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy.
Q58554380Human eye conditions: insights from the fly eye
Q90152961Leber Congenital Amaurosis (LCA): Potential for Improvement of Vision
Q64275334Mechanisms Underlying the Visual Benefit of Cell Transplantation for the Treatment of Retinal Degenerations
Q92988365Molecular Therapies for Inherited Retinal Diseases-Current Standing, Opportunities and Challenges
Q59354224Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector
Q89115884NAD+ and sirtuins in retinal degenerative diseases: A look at future therapies
Q90027776Optimizing Donor Cellular Dissociation and Subretinal Injection Parameters for Stem Cell-Based Treatments
Q99417940Precise allele-specific genome editing by spatiotemporal control of CRISPR-Cas9 via pronuclear transplantation
Q92301681Progress in Gene Therapy for Rhodopsin Autosomal Dominant Retinitis Pigmentosa
Q88957422Regenerating Eye Tissues to Preserve and Restore Vision
Q55387849Spliceosome-Mediated Pre-mRNA trans-Splicing Can Repair CEP290 mRNA.
Q45873271The molecular and cellular basis of rhodopsin retinitis pigmentosa reveals potential strategies for therapy
Q45874106The pigmented epithelium, a bright partner against photoreceptor degeneration
Q89791404Transplantation of iPSC-TM stimulates division of trabecular meshwork cells in human eyes

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