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| Content Provider | World Health Organization (WHO)-Global Index Medicus |
|---|---|
| Author | Gaj, Thomas Schaffer, David V. |
| Description | Author Affiliation: Gaj T ( Department of Chemical and Biomolecular Engineering, University of California, Berkeley, California 94720.); Schaffer DV ( Department of Chemical and Biomolecular Engineering, University of California, Berkeley, California 94720.) |
| Abstract | The CRISPR-Cas9 system has emerged as a highly versatile platform for introducing targeted genome modifications into mammalian cells and model organisms. However, fully capitalizing on the therapeutic potential for this system requires its safe and efficient delivery into relevant cell types. Adeno-associated virus (AAV) vectors are a clinically promising class of engineered gene-delivery vehicles capable of safely infecting a broad range of dividing and nondividing cell types, while also serving as a highly effective donor template for homology-directed repair. Together, CRISPR-Cas9 and AAV technologies have the potential to accelerate both basic research and clinical applications of genome engineering. Here, we present a step-by-step protocol for AAV-mediated delivery of CRISPR-Cas systems into mammalian cells. Procedures are given for the preparation of high-titer virus capable of achieving a diverse range of genetic modifications, including gene knockout and integration. |
| File Format | HTM / HTML |
| ISSN | 19403402 |
| Issue Number | 11 |
| Journal | Cold Spring Harbor Protocols |
| Volume Number | 2016 |
| e-ISSN | 15596095 |
| Language | English |
| Publisher | Cold Spring Harbor Laboratory Press |
| Publisher Date | 2016-11-01 |
| Publisher Place | United States |
| Access Restriction | One Nation One Subscription (ONOS) |
| Subject Keyword | Discipline Clinical Laboratory Techniques |
| Content Type | Text |
| Resource Type | Article |
| Subject | Biochemistry, Genetics and Molecular Biology |
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