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Genome-editing Technologies for Gene and Cell Therapy

Genome-editing Technologies for Gene and Cell Therapy

http://www.wikidata.org/entity/Q26769662

about

Remodeling and Control of Homologous Recombination by DNA Helicases and Translocases that Target Recombinases and Synapsis CRISPR-Cas9: from Genome Editing to Cancer Research Structures of malonic acid diamide/phospholipid composites and their lipoplexes.Integrase-Deficient Lentiviral Vector as an All-in-One Platform for Highly Efficient CRISPR/Cas9-Mediated Gene Editing Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery.CRISPR/Cas9 Editing of Murine Induced Pluripotent Stem Cells for Engineering Inflammation-Resistant Tissues.Cystic fibrosis lung environment and Pseudomonas aeruginosa infection.Genome surgery using Cas9 ribonucleoproteins for the treatment of age-related macular degeneration.Functional interrogation of non-coding DNA through CRISPR genome editing.Gene Editing for Treatment of Neurological Infections.Genome-wide specificities of CRISPR-Cas Cpf1 nucleases in human cells.CRISPR interference (CRISPRi) for gene regulation and succinate production in cyanobacterium S. elongatus PCC 7942.Mutant CAG Repeats Effectively Targeted by RNA Interference in SCA7 Cells.Programmable Genome Editing Tools and their Regulation for Efficient Genome Engineering.Reprogramming MHC specificity by CRISPR-Cas9-assisted cassette exchange.COL7A1 Editing via CRISPR/Cas9 in Recessive Dystrophic Epidermolysis Bullosa.Research on genodermatoses using novel genome-editing tools.Specific Destruction of HIV Proviral p17 Gene in T Lymphoid Cells Achieved by the Genome Editing Technology.Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects.CRISPR-Cas9 technology and its application in haematological disorders.Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.RNA-based therapies for genodermatoses.Gene Editing of Human Hematopoietic Stem and Progenitor Cells: Promise and Potential Hurdles.CRISPR/Cas9: a historical and chemical biology perspective of targeted genome engineering.Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics Targeting CPS1 in the treatment of Carbamoyl phosphate synthetase 1 (CPS1) deficiency, a urea cycle disorder.Therapeutic gene editing: delivery and regulatory perspectives Photoactivated cells link diagnosis and therapy.Healing a Heart Through Genetic Intervention.Current status and perspectives of chimeric antigen receptor modified T cells for cancer treatment.Optochemical Control of Biological Processes in Cells and Animals.Molecular basis of β thalassemia and potential therapeutic targets.Leber congenital amaurosis/early-onset severe retinal dystrophy: clinical features, molecular genetics and therapeutic interventions.Therapeutic Gene Editing Safety and Specificity.Hit-and-run programming of therapeutic cytoreagents using mRNA nanocarriers.Scalpel or Straitjacket: CRISPR/Cas9 Approaches for Muscular Dystrophies.Rhesus iPSC Safe Harbor Gene-Editing Platform for Stable Expression of Transgenes in Differentiated Cells of All Germ Layers.Clinical Applications of Genome Editing to HIV Cure.Sendai virus, an RNA virus with no risk of genomic integration, delivers CRISPR/Cas9 for efficient gene editing Genome engineering: a new approach to gene therapy for neuromuscular disorders.

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P2860

Genome-editing Technologies for Gene and Cell Therapy

http://www.wikidata.org/entity/Q26769662

description

2016 nî lūn-bûn

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2016 թուականի Մարտին հրատարակուած գիտական յօդուած

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2016 թվականի մարտին հրատարակված գիտական հոդված

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2016年の論文

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2016年論文

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2016年論文

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2016年論文

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2016年論文

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2016年論文

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2016年论文

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name

Genome-editing Technologies for Gene and Cell Therapy

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Genome-editing Technologies for Gene and Cell Therapy

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Genome-editing Technologies for Gene and Cell Therapy

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Genome-editing Technologies for Gene and Cell Therapy

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Genome-editing Technologies for Gene and Cell Therapy

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Genome-editing Technologies for Gene and Cell Therapy

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Genome-editing Technologies for Gene and Cell Therapy

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Genome-editing Technologies for Gene and Cell Therapy

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Genome-editing Technologies for Gene and Cell Therapy

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Molecular Therapy

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Genome-editing Technologies for Gene and Cell Therapy

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Charles A Gersbach

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Morgan L Maeder

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Creative Commons Attribution 4.0 International

P2860

Discovery and Functional Characterization of Diverse Class 2 CRISPR-Cas Systems

CRISPR Provides Acquired Resistance Against Viruses in Prokaryotes

Targeted gene therapy of xeroderma pigmentosum cells using meganuclease and TALEN™

Assembly of designer TAL effectors by Golden Gate cloning

Mutations altering the cleavage specificity of a homing endonuclease

Toward a code for the interactions of zinc fingers with DNA: selection of randomized fingers displayed on phage

Building zinc fingers by selection: toward a therapeutic application

Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain

RNA-guided human genome engineering via Cas9

TAL nucleases (TALNs): hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain

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430-46

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468461

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10.1038/MT.2016.10

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3

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24

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290379273

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26755333

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biomedical investigative technique

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4786923

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