In vivo genome editing restores haemostasis in a mouse model of haemophilia - Wikidata - wikimedia - TriplyDB

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

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

http://www.wikidata.org/entity/Q28241725

about

Cas9 as a versatile tool for engineering biology RNA-guided human genome engineering via Cas9 Clinical development of gene therapy: results and lessons from recent successes In Vivo Delivery Systems for Therapeutic Genome Editing Customizing the genome as therapy for the β-hemoglobinopathies Genome-editing Technologies for Gene and Cell Therapy Towards a new era in medicine: therapeutic genome editing From Gene Targeting to Genome Editing: Transgenic animals applications and beyond Enabling functional genomics with genome engineering Engineered Viruses as Genome Editing Devices Designed nucleases for targeted genome editing The gene therapy journey for hemophilia: are we there yet?Hematopoietic stem cell engineering at a crossroads Human pluripotent stem cells for modelling human liver diseases and cell therapy Progress and prospects of engineered sequence-specific DNA modulating technologies for the management of liver diseases Animal models of hemophilia Current translational and clinical practices in hematopoietic cell and gene therapy Genome-editing technologies for gene correction of hemophilia Genome editing: the road of CRISPR/Cas9 from bench to clinic CRISPR-Cas9 System as a Versatile Tool for Genome Engineering in Human Cells The potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challenges Genetically engineered mouse models in oncology research and cancer medicine The application of genome editing in studying hearing loss Gene therapy for 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 Pleiotropy in complex traits: challenges and strategies A universal system to select gene-modified hepatocytes in vivo ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering Usher syndrome: Hearing loss, retinal degeneration and associated abnormalities.Rescue of bilirubin-induced neonatal lethality in a mouse model of Crigler-Najjar syndrome type I by AAV9-mediated gene transfer Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo Oligonucleotide-based strategies to combat polyglutamine diseases.CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice.ENU Mutagenesis in the Mouse.Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.Gene transfer in the liver using recombinant adeno-associated virus The scientific quest for lasting youth: prospects for curing aging.CRISPR/Cas9 for genome editing: progress, implications and challenges.

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

http://www.wikidata.org/entity/Q28241725

description

2011 nî lūn-bûn

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2011 թուականի Յունիսին հրատարակուած գիտական յօդուած

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2011 թվականի հունիսին հրատարակված գիտական հոդված

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2011年の論文

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2011年論文

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2011年論文

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2011年論文

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2011年論文

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2011年論文

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2011年论文

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name

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

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

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

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

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

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

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

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

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

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

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Anand S Bhagwat

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David E Paschon

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Edward J Rebar

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Hojun Li

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Jonathan D Finn

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Lacramiora Ivanciu

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Michael C Holmes

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Nirav Malani

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Samuel L Murphy

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P2860

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

Safety and efficacy of gene transfer for Leber's congenital amaurosis

Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

Effect of gene therapy on visual function in Leber's congenital amaurosis

Genome editing with engineered zinc finger nucleases

Selection of target sites for mobile DNA integration in the human genome

Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases

Chimeric nucleases stimulate gene targeting in human cells

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1288242

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10.1038/NATURE10177

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7355

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475

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Frederic D. Bushman

Philip D Gregory

Xavier M Anguela

Katherine A High

Virginia Haurigot

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2011-06-26T00:00:00Z

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51250606

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1041862601

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21706032

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