Article

Amelioration of hemophilia B through CRISPR/Cas9 induced homology-independent targeted integration

2021-03-19

Abstract excerpt

Site-specific integration of exogenous gene through genome editing is a promising strategy for gene therapy. However, homology-directed repair (HDR) only occurring in proliferating cells is inefficient especially in vivo . To investigate the efficacy of Cas9-induced homology-independent targeted integration (HITI) strategy for gene therapy, a rat hemophilia B model was generated and employed. Through HITI, a DNA...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 54915738-ff7c-5c58-9c1e-eec0911bf65c
DOI: 10.1101/2021.03.18.435908

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Amelioration of hemophilia B through CRISPR/Cas9 induced homology-independent targeted integrationDOI 10.1101/2021.03.18.435908Read

Amelioration of hemophilia B through CRISPR/Cas9 induced homology-independent targeted integrationSemantic proximity Read

CRISPR-Cas9-Mediated In Vivo Gene Integration at the Albumin Locus Recovers Hemostasis in Neonatal and Adult Hemophilia B MiceSemantic proximity Read

CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice.Semantic proximity Read

Viral Vector-Based Delivery of CRISPR/Cas9 and Donor DNA for Homology-Directed Repair in an In Vitro Model for Canine Hemophilia BSemantic proximity Read

Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIIISemantic proximity Read

CRISPR-Mediated In Situ Introduction or Integration of F9-Padua in Human iPSCs for Gene Therapy of Hemophilia B.Semantic proximity Read

Inherent hepatocytic heterogeneity determines expression and retention of edited F9 alleles post-AAV/CRISPR infusion.Semantic proximity Read

CRISPR/Cas9 Edition of the F9 Gene in Human Mesenchymal Stem Cells for Hemophilia B TherapySemantic proximity Read

In vivo LNP-CRISPR Approaches for the Treatment of HemophiliaSemantic proximity Read

Low-dose AAV-CRISPR-mediated liver-specific knock-in restored hemostasis in neonatal hemophilia B mice with subtle antibody responseSemantic proximity Read

Challenges and opportunities when transitioning from in vivo gene replacement to in vivo CRISPR/Cas9 therapies – a spotlight on hemophiliaSemantic proximity Read

Homology-directed gene-editing approaches for hematopoietic stem and progenitor cell gene therapySemantic proximity Read