Article

Improving the efficiency of liver targeting rAAV-mediated homologous recombination using ribonucleotide reductase inhibitors

2020-12-10

Abstract excerpt

<title>Abstract</title> Recombinant adeno-associated viral (rAAV) vectors continue to gain popularity for <italic>in vivo</italic> therapeutic gene delivery. Homologous recombination-based gene therapy using rAAV (AAV-HR) without nucleases has several advantages over classical gene therapy, especially when targeting the liver in neonatal/pediatric populations due to its potential for permanent sustained transg...

Topics

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

Identifiers and source

Literature Corpus work: b120fcd0-849b-5c17-94e2-0d94409ad71b
DOI: 10.21203/rs.3.rs-106148/v1

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Improving the efficiency of liver targeting rAAV-mediated homologous recombination using ribonucleotide reductase inhibitorsDOI 10.21203/rs.3.rs-106148/v1Read

In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential geneSemantic proximity Read

Ex Vivo Hepatocyte Reprograming Promotes Homology‐Directed DNA Repair to Correct Metabolic Disease in Mice After TransplantationSemantic proximity Read

Ribonucleotide Reductase: A Critical Enzyme for Cancer Chemotherapy and Antiviral AgentsSemantic proximity Read

Inhibitors of the Cancer Target Ribonucleotide Reductase, Past and PresentSemantic proximity Read

MicroRNA-122-mediated liver detargeting enhances the tissue specificity of cardiac genome editingSemantic proximity Read

Genetic manipulation using hepatocyte-targeting adeno-associated viral vectors has minimal off-target effectsSemantic proximity Read

Homologous recombination is required for AAV-mediated gene targeting.Semantic proximity Read

AAV8 Gene Therapy Rescues the Newborn Phenotype of a Mouse Model of Crigler-Najjar.Semantic proximity Read

Adenovirus-mediated Cre deletion of floxed sequences in primary mouse cells is an efficient alternative for studies of gene deletion.Semantic proximity Read

Hepatitis B Virus Activates Deoxynucleotide Synthesis in Nondividing Hepatocytes by Targeting the R2 GeneSemantic proximity Read

Highly Efficient Lentiviral Vector-Mediated Transduction of Nondividing, Fully Reimplantable Primary HepatocytesSemantic proximity Read

Potent siRNA Inhibitors of Ribonucleotide Reductase Subunit RRM2 Reduce Cell Proliferation In vitro and In vivoSemantic proximity Read