CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells. - Wikidata - awgldk - TriplyDB

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.

http://www.wikidata.org/entity/Q40486239

about

The CRISPR/Cas9 system efficiently reverts the tumorigenic ability of BCR/ABL in vitro and in a xenograft model of chronic myeloid leukemia.Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery.Functional interrogation of non-coding DNA through CRISPR genome editing.The rising young scientist stars in China Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells.Reprogramming MHC specificity by CRISPR-Cas9-assisted cassette exchange.The state of gene therapy research in Africa, its significance and implications for the future.Developmental history and application of CRISPR in human disease.CRISPR/Cas9 system: a powerful technology for in vivo and ex vivo gene therapy.Adeno-Associated Virus Vectors and Stem Cells: Friends or Foes?CRISPR Editing Technology in Biological and Biomedical Investigation.Function and safety of lentivirus-mediated gene transfer for CSF2RA-deficiency.Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond.Genome editing in Drosophila melanogaster: from basic genome engineering to the multipurpose CRISPR-Cas9 system.CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice.CRISPR/CAS9 Technologies.Evolving Gene Therapy in Primary Immunodeficiency.Gene Therapy for β-Hemoglobinopathies.Genome editing: a robust technology for human stem cells.The CRISPR/Cas9 system: Their delivery, in vivo and ex vivo applications and clinical development by startups.Investigational drugs in phase I and phase II clinical trials for thalassemia.Ser/Thr protein kinase Bβ-NADPH oxidase 2 signaling in thromboinflammation.Technical considerations for the use of CRISPR/Cas9 in hematology research.CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors.Lentiviral vectors escape innate sensing but trigger p53 in human hematopoietic stem and progenitor cells.CRISPR/Cas9-Mediated CCR5 Ablation in Human Hematopoietic Stem/Progenitor Cells Confers HIV-1 Resistance In Vivo.Zebrafish In-Vivo Screening for Compounds Amplifying Hematopoietic Stem and Progenitor Cells: - Preclinical Validation in Human CD34+ Stem and Progenitor Cells.The Promise and Challenge of In Vivo Delivery for Genome Therapeutics.A directed evolution approach to select for novel Adeno-associated virus capsids on an HIV-1 producer T cell line.Synthetically modified guide RNA and donor DNA are a versatile platform for CRISPR-Cas9 engineering Multiplexed genetic engineering of human hematopoietic stem and progenitor cells using CRISPR/Cas9 and AAV6.Virus-Mediated Genome Editing via Homology-Directed Repair in Mitotic and Postmitotic Cells in Mammalian Brain.The changing landscape of gene editing in hematopoietic stem cells: a step towards Cas9 clinical translation.Gene therapy: Erasing sickle-cell disease.Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1.Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned.Combining Engineered Nucleases with Adeno-associated Viral Vectors for Therapeutic Gene Editing.Fetal haemoglobin induction in sickle cell disease.Pharmacological and molecular approaches for the treatment of β-hemoglobin disorders.A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease.

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CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.

http://www.wikidata.org/entity/Q40486239

description

2016 nî lūn-bûn

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2016年の論文

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2016年学术文章

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2016年学术文章

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2016年学术文章

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2016年學術文章

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2016年學術文章

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2016年學術文章

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name

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.

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type

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CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.

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prefLabel

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.

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CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells.

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Alec B Wilkens

http://www.w3.org/2001/XMLSchema#string

Anupama Narla

http://www.w3.org/2001/XMLSchema#string

Ayal Hendel

http://www.w3.org/2001/XMLSchema#string

Carmencita E Nicolas

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Daniel P Dever

http://www.w3.org/2001/XMLSchema#string

Gabriel Washington

http://www.w3.org/2001/XMLSchema#string

Kenneth I Weinberg

http://www.w3.org/2001/XMLSchema#string

Mara Pavel-Dinu

http://www.w3.org/2001/XMLSchema#string

Matthew H Porteus

http://www.w3.org/2001/XMLSchema#string

Nivi Saxena

http://www.w3.org/2001/XMLSchema#string

P2860

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

Identification of a hierarchy of multipotent hematopoietic progenitors in human cord blood

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

Towards a new era in medicine: therapeutic genome editing

Gene therapy: therapeutic gene causing lymphoma

Development and applications of CRISPR-Cas9 for genome engineering.

Genome editing. The new frontier of genome engineering with CRISPR-Cas9

Insertion of DNA sequences into the human chromosomal beta-globin locus by homologous recombination

Hematopoietic stem cell: self-renewal versus differentiation

Chimeric nucleases stimulate gene targeting in human cells

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384-389

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10.1038/NATURE20134

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7629

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539

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Andreas Reinisch

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2016-11-07T00:00:00Z

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1038845840

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27820943

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5898607

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