CRISPR/Cas9-mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse.
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Genome-editing technologies for gene correction of hemophiliaThe CRISPR/Cas9 system efficiently reverts the tumorigenic ability of BCR/ABL in vitro and in a xenograft model of chronic myeloid leukemia.Long-Term Assessment of AAV-Mediated Zinc Finger Nuclease Expression in the Mouse Brain.CRISPR/Cas9 targeting events cause complex deletions and insertions at 17 sites in the mouse genome.CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice.Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success.The CRB1 Complex: Following the Trail of Crumbs to a Feasible Gene Therapy Strategy.Gene therapy research in Asia.Recent Advances in CRISPR-Cas9 Genome Editing Technology for Biological and Biomedical Investigations.CRISPR/Cas9 system: a powerful technology for in vivo and ex vivo gene therapy.CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice.CRISPR-Cas9 technology and its application in haematological disorders.In vivo and in vitro disease modeling with CRISPR/Cas9.2017 Clinical trials update: Innovations in hemophilia therapy.A Prospective Treatment Option for Lysosomal Storage Diseases: CRISPR/Cas9 Gene Editing Technology for Mutation Correction in Induced Pluripotent Stem Cells.Immunity to CRISPR Cas9 and Cas12a therapeutics.Novel therapies and current clinical progress in hemophilia A.Gene Therapy for Hemophilia.[CRISPR-Cas system as molecular scissors for gene therapy].Nuclease-Mediated Gene Therapies for Inherited Metabolic Diseases of the Liver.Cas9-nickase-mediated genome editing corrects hereditary tyrosinemia in rats.Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9.Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9.Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 system.Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX.CRISPR/Cas9: the Jedi against the dark empire of diseases.Delivering CRISPR: a review of the challenges and approaches
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CRISPR/Cas9-mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse.
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article científic
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article scientifique
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articolo scientifico
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artigo científico
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bilimsel makale
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scientific article published on 10 March 2016
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
CRISPR/Cas9-mediated somatic c ...... eliorates hemophilia in mouse.
@en
CRISPR/Cas9-mediated somatic c ...... eliorates hemophilia in mouse.
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type
label
CRISPR/Cas9-mediated somatic c ...... eliorates hemophilia in mouse.
@en
CRISPR/Cas9-mediated somatic c ...... eliorates hemophilia in mouse.
@nl
prefLabel
CRISPR/Cas9-mediated somatic c ...... eliorates hemophilia in mouse.
@en
CRISPR/Cas9-mediated somatic c ...... eliorates hemophilia in mouse.
@nl
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CRISPR/Cas9-mediated somatic c ...... eliorates hemophilia in mouse.
@en
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Honghui Han
Liren Wang
Wenqing Lu
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P304
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10.15252/EMMM.201506039
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2016-03-10T00:00:00Z