| First Author | Santiago-Fernández O | Year | 2019 |
| Journal | Nat Med | Volume | 25 |
| Issue | 3 | Pages | 423-426 |
| PubMed ID | 30778239 | Mgi Jnum | J:285478 |
| Mgi Id | MGI:6393058 | Doi | 10.1038/s41591-018-0338-6 |
| Citation | Santiago-Fernandez O, et al. (2019) Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome. Nat Med 25(3):423-426 |
| abstractText | CRISPR/Cas9-based therapies hold considerable promise for the treatment of genetic diseases. Among these, Hutchinson-Gilford progeria syndrome, caused by a point mutation in the LMNA gene, stands out as a potential candidate. Here, we explore the efficacy of a CRISPR/Cas9-based approach that reverts several alterations in Hutchinson-Gilford progeria syndrome cells and mice by introducing frameshift mutations in the LMNA gene. |
