Work

3. Development of a Cas9 Protein Delivery System with Lentiviral Vectors for RNA-Guided Genome Editing

Year: 2016

Type: article

Abstract: Gene correction is an ideal gene therapy strategy for hereditary disease, including sickle cell disease. Recently, the CRISPR/Cas9 system was developed to allow site-specific DNA breakage, which can enhance homologous recombination-based gene correction with template DNA. However, efficient delivery remains crucial for successful genome editing. In this work, we sought to develop a new gene/protein delivery system with lentiviral vectors for efficient genome editing. We first used an HIV-1 based lentiviral vector system to deliver the CRISPR/Cas9 system including guide RNAs and the Cas9 endonuclease since efficient delivery in various cells, including hematopoietic stem cells, has previously been established.

Source: Molecular Therapy

DOI: 10.1016/s1525-0016(16)32812-x

Authors: Naoya Uchida, Anna Shvygin, Luke Skala, Lydia Raines, Josiah Ballantine, John Tisdale

Institution: National Institutes of Health

Language: English

Topic: CRISPR and Genetic Engineering

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $4,500