Meganucleases and other tools for targeted genome engineering: perspectives and challenges for gene therapy.
about
Customizing the genome as therapy for the β-hemoglobinopathiesTowards a new era in medicine: therapeutic genome editingInduced Pluripotency and Gene Editing in Disease Modelling: Perspectives and ChallengesApplication of genomics-assisted breeding for generation of climate resilient crops: progress and prospectsModelling human disease with pluripotent stem cellsQuantifying on- and off-target genome editingHoming endonucleases: DNA scissors on a mission5'-Cytosine-Phosphoguanine (CpG) Methylation Impacts the Activity of Natural and Engineered MeganucleasesStructural mechanisms of the degenerate sequence recognition by Bse634I restriction endonucleaseCRISPR-Cas systems for editing, regulating and targeting genomesQuantifying genome-editing outcomes at endogenous loci with SMRT sequencing.Genome-Edited T Cell Therapies.Control of catalytic efficiency by a coevolving network of catalytic and noncatalytic residues.Improved modeling of side-chain--base interactions and plasticity in protein--DNA interface designChromosomal context and epigenetic mechanisms control the efficacy of genome editing by rare-cutting designer endonucleases.Genome engineering empowers the diatom Phaeodactylum tricornutum for biotechnology.Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.MAR elements and transposons for improved transgene integration and expressionEfficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9TALE-PvuII fusion proteins--novel tools for gene targeting.Single-stranded γPNAs for in vivo site-specific genome editing via Watson-Crick recognition.Efficient design of meganucleases using a machine learning approach.Homing endonucleases: from genetic anomalies to programmable genomic clippersRapid prototyping of microbial cell factories via genome-scale engineering.Rescue of DNA-PK Signaling and T-Cell Differentiation by Targeted Genome Editing in a prkdc Deficient iPSC Disease Model.Using Group II Introns for Attenuating the In Vitro and In Vivo Expression of a Homing EndonucleaseTargeted In Situ Gene Correction of Dysfunctional APOE Alleles to Produce Atheroprotective Plasma ApoE3 Protein.CRISPR-Cas9-mediated genome editing and guide RNA design.Controlled delivery of β-globin-targeting TALENs and CRISPR/Cas9 into mammalian cells for genome editing using microinjectionTerminal deoxynucleotidyl transferase requires KU80 and XRCC4 to promote N-addition at non-V(D)J chromosomal breaks in non-lymphoid cellsDifferential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells.Quantitative analysis of TALE-DNA interactions suggests polarity effects.The genetic basis of severe combined immunodeficiency and its variants.A genome editing primer for the hematologistDiscovery of Nigri/nox and Panto/pox site-specific recombinase systems facilitates advanced genome engineeringCRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells.Reprogramming homing endonuclease specificity through computational design and directed evolution.Programmable Genome Editing Tools and their Regulation for Efficient Genome Engineering.The Development of TALE Nucleases for Biotechnology.Redesign of extensive protein-DNA interfaces of meganucleases using iterative cycles of in vitro compartmentalization.
P2860
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P2860
Meganucleases and other tools for targeted genome engineering: perspectives and challenges for gene therapy.
description
2011 nî lūn-bûn
@nan
2011年の論文
@ja
2011年論文
@yue
2011年論文
@zh-hant
2011年論文
@zh-hk
2011年論文
@zh-mo
2011年論文
@zh-tw
2011年论文
@wuu
2011年论文
@zh
2011年论文
@zh-cn
name
Meganucleases and other tools ...... d challenges for gene therapy.
@ast
Meganucleases and other tools ...... d challenges for gene therapy.
@en
type
label
Meganucleases and other tools ...... d challenges for gene therapy.
@ast
Meganucleases and other tools ...... d challenges for gene therapy.
@en
prefLabel
Meganucleases and other tools ...... d challenges for gene therapy.
@ast
Meganucleases and other tools ...... d challenges for gene therapy.
@en
P2093
P2860
P921
P1433
P1476
Meganucleases and other tools ...... d challenges for gene therapy.
@en
P2093
Frédéric Pâques
George Silva
Guillermo Montoya
Julianne Smith
Laurent Poirot
Philippe Duchateau
Roman Galetto
P2860
P356
10.2174/156652311794520111
P407
P577
2011-02-01T00:00:00Z