Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. - Wikidata - awgldk - TriplyDB

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

http://www.wikidata.org/entity/Q34979802

about

Clinical development of gene therapy: results and lessons from recent successes Genome-editing Technologies for Gene and Cell Therapy Towards a new era in medicine: therapeutic genome editing Genome-editing technologies for gene correction of hemophilia Liver-targeted gene therapy: Approaches and challenges Therapeutic genome editing: prospects and challenges Gene therapy in an era of emerging treatment options for hemophilia B A universal system to select gene-modified hepatocytes in vivo Ultrasound-targeted hepatic delivery of factor IX in hemophiliac mice.Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy.Regenerative medicine: targeted genome editing in vivo.Hitting the target without pulling the trigger Cre Activated and Inactivated Recombinant Adeno-Associated Viral Vectors for Neuronal Anatomical Tracing or Activity Manipulation.In vivo genome editing of the albumin locus as a platform for protein replacement therapy Genome editing for inborn errors of metabolism: advancing towards the clinic.A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications.Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors.CRISPR-Cas9 Targeting of PCSK9 in Human Hepatocytes In Vivo-Brief Report.A genome editing primer for the hematologist In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA.Recombinant adeno-associated virus vectors in the treatment of rare diseases.A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.CRISPR/Cas9-mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse.Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success.TALEN/CRISPR-mediated engineering of a promoterless anti-viral RNAi hairpin into an endogenous miRNA locus.Obstacles and future of gene therapy for hemophilia.New approaches to gene and cell therapy for hemophilia.New and improved AAVenues: current status of hemophilia B gene therapy.Current Progress in Therapeutic Gene Editing for Monogenic Diseases Mechanistic basis for increased human gene targeting by promoterless vectors-roles of homology arms and Rad54 paralogs.Gene Insertion Into Genomic Safe Harbors for Human Gene Therapy.Viral vectors for therapy of neurologic diseases.Gene editing technology as an approach to the treatment of liver diseases.Human somatic cells deficient for RAD52 are impaired for viral integration and compromised for most aspects of homology-directed repair.Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond.Viral Vectors: The Road to Reducing Genotoxicity.In vivo blunt-end cloning through CRISPR/Cas9-facilitated non-homologous end-joining Improved methods of AAV-mediated gene targeting for human cell lines using ribosome-skipping 2A peptide.

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P2860

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

http://www.wikidata.org/entity/Q34979802

description

2014 nî lūn-bûn

@nan

2014 թուականի Հոկտեմբերին հրատարակուած գիտական յօդուած

@hyw

2014 թվականի հոտեմբերին հրատարակված գիտական հոդված

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2014年の論文

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2014年論文

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2014年論文

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2014年論文

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2014年論文

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2014年論文

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2014年论文

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name

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

@ast

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

@en

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

@nl

type

label

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

@ast

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

@en

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

@nl

prefLabel

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

@ast

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

@en

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.

@nl

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Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

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A Barzel

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F Zhang

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K Chu

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K M Gaensler

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L P Spector

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M H Porteus

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Y Shi

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P2860

Gene therapy with human and mouse T-cell receptors mediates cancer regression and targets normal tissues expressing cognate antigen

In vivo genome editing restores haemostasis in a mouse model of haemophilia

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.

High cleavage efficiency of a 2A peptide derived from porcine teschovirus-1 in human cell lines, zebrafish and mice

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses.

Quantifying genome-editing outcomes at endogenous loci with SMRT sequencing.

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo.

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7534

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Paul N. Valdmanis

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2014-10-29T00:00:00Z

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