dCas9-VPR-mediated transcriptional activation of functionally equivalent genes for gene therapy
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E. Becirovic | S. Michalakis | L. M. Riedmayr | Sybille Böhm | Victoria Splith | Klara S. Hinrichsmeyer | Nina Karguth
[1] Timothy J. Hohman,et al. Protective genes and pathways in Alzheimer’s disease: moving towards precision interventions , 2021, Molecular Neurodegeneration.
[2] D. Grimm,et al. A universal protocol for isolating retinal ON bipolar cells across species via fluorescence-activated cell sorting , 2021, Molecular therapy. Methods & clinical development.
[3] S. Ramalingam,et al. Autosomal dominant retinitis pigmentosa with toxic gain of function: Mechanisms and therapeutics , 2020, European journal of ophthalmology.
[4] J. Wijnholds,et al. Novel Therapeutic Approaches for the Treatment of Retinal Degenerative Diseases: Focus on CRISPR/Cas-Based Gene Editing , 2020, Frontiers in Neuroscience.
[5] C. Wahl-Schott,et al. A gene therapy for inherited blindness using dCas9-VPR–mediated transcriptional activation , 2020, Science Advances.
[6] H. Blum,et al. Antisense Oligonucleotide- and CRISPR-Cas9-Mediated Rescue of mRNA Splicing for a Deep Intronic CLRN1 Mutation , 2020, Molecular therapy. Nucleic acids.
[7] F. Zhang,et al. CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors , 2020, Cell.
[8] Claire M. Brown,et al. Tutorial: guidance for quantitative confocal microscopy , 2020, Nature Protocols.
[9] K. Anderson,et al. Guidance for quantitative confocal microscopy , 2020, Nature Protocols.
[10] R. Samulski,et al. Engineering adeno-associated virus vectors for gene therapy , 2020, Nature Reviews Genetics.
[11] Hendrik Weisser,et al. Genome-wide investigation of gene-cancer associations for the prediction of novel therapeutic targets in oncology , 2020, Scientific Reports.
[12] Huanbin Zhou,et al. Targeted base editing in rice with CRISPR/ScCas9 system , 2020, Plant biotechnology journal.
[13] David R. Liu,et al. Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses , 2019, Nature Biomedical Engineering.
[14] David R. Liu,et al. Search-and-replace genome editing without double-strand breaks or donor DNA , 2019, Nature.
[15] Terence R Flotte,et al. Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here? , 2019, Annual review of virology.
[16] Xia Li,et al. Gain-of-Function Mutations: An Emerging Advantage for Cancer Biology. , 2019, Trends in biochemical sciences.
[17] S. Prescott,et al. A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene , 2019, Nature.
[18] Peter C. DeWeirdt,et al. Genetic screens in isogenic mammalian cell lines without single cell cloning , 2019, Nature Communications.
[19] Carel B. Hoyng,et al. Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina , 2019, Science Translational Medicine.
[20] G. Gao,et al. Adeno-associated virus vector as a platform for gene therapy delivery , 2019, Nature Reviews Drug Discovery.
[21] Navneet Matharu,et al. CRISPR-mediated activation of a promoter or enhancer rescues obesity caused by haploinsufficiency , 2019, Science.
[22] Silvio C. E. Tosatto,et al. InterPro in 2019: improving coverage, classification and access to protein sequence annotations , 2018, Nucleic Acids Res..
[23] P. Sieving,et al. Accessory heterozygous mutations in cone photoreceptor CNGA3 exacerbate CNG channel–associated retinopathy , 2018, The Journal of clinical investigation.
[24] M. Babu,et al. Human Diseases from Gain-of-Function Mutations in Disordered Protein Regions , 2018, Cell.
[25] Kang Zhang,et al. In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation. , 2018, Molecular therapy : the journal of the American Society of Gene Therapy.
[26] M. Mahfouz,et al. CRISPR base editors: genome editing without double-stranded breaks , 2018, The Biochemical journal.
[27] Maximilian Haeussler,et al. CRISPOR: intuitive guide selection for CRISPR/Cas9 genome editing experiments and screens , 2018, Nucleic Acids Res..
[28] Andreas Hierlemann,et al. Smart Cell Culture Systems: Integration of Sensors and Actuators into Microphysiological Systems. , 2018, ACS chemical biology.
[29] Michel Sadelain,et al. Gene therapy comes of age , 2018, Science.
[30] C. R. Esteban,et al. In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic Modulation , 2017, Cell.
[31] Nicole M. Gaudelli,et al. Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage , 2017, Nature.
[32] G. Aguirre,et al. Variabilities in retinal function and structure in a canine model of cone-rod dystrophy associated with RPGRIP1 support multigenic etiology , 2017, Scientific Reports.
[33] Livia S. Carvalho,et al. Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting , 2017, Front. Neurosci..
[34] David Cowburn,et al. A promiscuous split intein with expanded protein engineering applications , 2017, Proceedings of the National Academy of Sciences.
[35] C. Wahl-Schott,et al. Peripherin-2 and Rom-1 have opposing effects on rod outer segment targeting of retinitis pigmentosa-linked peripherin-2 mutants , 2017, Scientific Reports.
[36] J. Sahel,et al. Insight into the mechanisms of enhanced retinal transduction by the engineered AAV2 capsid variant ‐7m8 , 2016, Biotechnology and bioengineering.
[37] Dacheng Ma,et al. Integration and exchange of split dCas9 domains for transcriptional controls in mammalian cells , 2016, Nature Communications.
[38] T. Lancet. Fit to serve , 2016, The Lancet.
[39] Prashant Mali,et al. A multifunctional AAV–CRISPR–Cas9 and its host response , 2016, Nature Methods.
[40] Jean Bennett,et al. Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial , 2016, The Lancet.
[41] Predrag Radivojac,et al. The Loss and Gain of Functional Amino Acid Residues Is a Common Mechanism Causing Human Inherited Disease , 2016, PLoS Comput. Biol..
[42] C. Wahl-Schott,et al. AAV Vectors for FRET-Based Analysis of Protein-Protein Interactions in Photoreceptor Outer Segments , 2016, Front. Neurosci..
[43] David R. Liu,et al. Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage , 2016, Nature.
[44] E. Ayuso,et al. Practical utilization of recombinant AAV vector reference standards: focus on vector genomes titration by free ITR qPCR , 2016, Molecular therapy. Methods & clinical development.
[45] M. Biel,et al. In Vivo Analysis of Disease-Associated Point Mutations Unveils Profound Differences in mRNA Splicing of Peripherin-2 in Rod and Cone Photoreceptors , 2016, PLoS genetics.
[46] Eric J. Topol,et al. Protective alleles and modifier variants in human health and disease , 2015, Nature Reviews Genetics.
[47] Feng Zhang,et al. Orthogonal gene knock out and activation with a catalytically active Cas9 nuclease , 2015, Nature Biotechnology.
[48] G. Church,et al. Cas9 gRNA engineering for genome editing, activation and repression , 2015, Nature Methods.
[49] Wolfgang Wurst,et al. Development of an intein-mediated split–Cas9 system for gene therapy , 2015, Nucleic acids research.
[50] Yuta Nihongaki,et al. Photoactivatable CRISPR-Cas9 for optogenetic genome editing , 2015, Nature Biotechnology.
[51] S. E. Barker,et al. Long-term effect of gene therapy on Leber's congenital amaurosis. , 2015, The New England journal of medicine.
[52] Feng Zhang,et al. A split-Cas9 architecture for inducible genome editing and transcription modulation , 2015, Nature Biotechnology.
[53] Ron Weiss,et al. Highly-efficient Cas9-mediated transcriptional programming , 2014, Nature Methods.
[54] Alexandro E. Trevino,et al. Genome-scale transcriptional activation by an engineered CRISPR-Cas9 complex , 2014, Nature.
[55] L. Vandenberghe,et al. Adeno-associated virus: fit to serve. , 2014, Current opinion in virology.
[56] Max A. Horlbeck,et al. Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation , 2014, Cell.
[57] A. Wlodawer,et al. Nature's recipe for splitting inteins. , 2014, Protein engineering, design & selection : PEDS.
[58] Lief E. Fenno,et al. Targeting cells with single vectors using multiple-feature Boolean logic , 2014, Nature Methods.
[59] G. Church,et al. CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering , 2013, Nature Biotechnology.
[60] Christopher M. Vockley,et al. RNA-guided gene activation by CRISPR-Cas9-based transcription factors , 2013, Nature Methods.
[61] Morgan L. Maeder,et al. CRISPR RNA-guided activation of endogenous human genes , 2013, Nature Methods.
[62] Eli J. Fine,et al. DNA targeting specificity of RNA-guided Cas9 nucleases , 2013, Nature Biotechnology.
[63] Luke A. Gilbert,et al. CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes , 2013, Cell.
[64] T. Lamb,et al. Evolution of phototransduction, vertebrate photoreceptors and retina , 2013, Progress in Retinal and Eye Research.
[65] Deniz Dalkara,et al. In Vivo–Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous , 2013, Science Translational Medicine.
[66] B. A. French,et al. Adeno-associated virus serotype 9 efficiently targets ischemic skeletal muscle following systemic delivery , 2013, Gene Therapy.
[67] W. Hauswirth,et al. A comprehensive review of retinal gene therapy. , 2013, Molecular therapy : the journal of the American Society of Gene Therapy.
[68] R. Klein,et al. The advent of AAV9 expands applications for brain and spinal cord gene delivery , 2012, Expert opinion on biological therapy.
[69] V. Kefalov. Rod and Cone Visual Pigments and Phototransduction through Pharmacological, Genetic, and Physiological Approaches* , 2011, The Journal of Biological Chemistry.
[70] C. Rudolph,et al. Adeno-associated virus serotype 9-mediated pulmonary transgene expression: effect of mouse strain, animal gender and lung inflammation , 2011, Gene Therapy.
[71] M. Lock,et al. Efficient serotype-dependent release of functional vector into the culture medium during adeno-associated virus manufacturing. , 2010, Human gene therapy.
[72] Zhijian Wu,et al. Effect of genome size on AAV vector packaging. , 2010, Molecular therapy : the journal of the American Society of Gene Therapy.
[73] W. Hauswirth,et al. High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.
[74] James M. Wilson,et al. Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro. , 2009, Molecular therapy : the journal of the American Society of Gene Therapy.
[75] Jürgen Cox,et al. A practical guide to the MaxQuant computational platform for SILAC-based quantitative proteomics , 2009, Nature Protocols.
[76] H. Sweeney,et al. Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat. , 2008, Human gene therapy.
[77] B. Wang,et al. Protein trans-splicing as a means for viral vector-mediated in vivo gene therapy. , 2008, Human gene therapy.
[78] Thomas D. Schmittgen,et al. Analyzing real-time PCR data by the comparative CT method , 2008, Nature Protocols.
[79] K. Yau,et al. Quantal noise from human red cone pigment , 2008, Nature Neuroscience.
[80] Y. Kan,et al. AAV serotype 1 mediates more efficient gene transfer to pig myocardium than AAV serotype 2 and plasmid , 2008, The journal of gene medicine.
[81] K. Yau,et al. Signaling Properties of a Short-Wave Cone Visual Pigment and Its Role in Phototransduction , 2007, The Journal of Neuroscience.
[82] K. Nakatani,et al. Physiological properties of rod photoreceptor cells in green-sensitive cone pigment knock-in mice. , 2007, The Journal of general physiology.
[83] 易美济. 科学仪器公司Thermo Fisher Scientific正式起航 , 2007 .
[84] B. Hyman,et al. Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain , 2006, Neuroscience.
[85] L. Davies,et al. Long-term persistence of gene expression from adeno-associated virus serotype 5 in the mouse airways , 2006, Gene Therapy.
[86] Amy E Palmer,et al. Measuring calcium signaling using genetically targetable fluorescent indicators , 2006, Nature Protocols.
[87] Amos Bairoch,et al. ScanProsite: detection of PROSITE signature matches and ProRule-associated functional and structural residues in proteins , 2006, Nucleic Acids Res..
[88] O. Danos,et al. Long-term expression and repeated administration of AAV type 1, 2 and 5 vectors in skeletal muscle of immunocompetent adult mice , 2006, Gene Therapy.
[89] Lili Wang,et al. Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.
[90] Jianming Xu. Preparation, culture, and immortalization of mouse embryonic fibroblasts. , 2005, Current protocols in molecular biology.
[91] Bing Wang,et al. Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart , 2005, Nature Biotechnology.
[92] P. Reier,et al. Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system. , 2004, Molecular therapy : the journal of the American Society of Gene Therapy.
[93] James M. Allen,et al. Systemic delivery of genes to striated muscles using adeno-associated viral vectors , 2004, Nature Medicine.
[94] M. Naash,et al. Expression of cone-photoreceptor-specific antigens in a cell line derived from retinal tumors in transgenic mice. , 2004, Investigative ophthalmology & visual science.
[95] John A. Stanturf,et al. Where are we and how did we get here , 2003 .
[96] G. Kobinger,et al. Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model. , 2001, Human molecular genetics.
[97] James M. Wilson,et al. Gene Therapy Vectors Based on Adeno-Associated Virus Type 1 , 1999, Journal of Virology.
[98] Z. Hu,et al. Protein trans-splicing by a split intein encoded in a split DnaE gene of Synechocystis sp. PCC6803. , 1998, Proceedings of the National Academy of Sciences of the United States of America.
[99] T. Dryja,et al. Digenic retinitis pigmentosa due to mutations at the unlinked peripherin/RDS and ROM1 loci. , 1994, Science.
[100] M. Al-Ubaidi,et al. Bilateral retinal and brain tumors in transgenic mice expressing simian virus 40 large T antigen under control of the human interphotoreceptor retinoid-binding protein promoter , 1992, The Journal of cell biology.
[101] P. Sharp,et al. Recombinant retroviruses encoding simian virus 40 large T antigen and polyomavirus large and middle T antigens , 1986, Molecular and cellular biology.