Abstract
CRISPR-based genome-editing technologies, including nuclease editing, base editing, and prime editing, have recently revolutionized the development of therapeutics targeting disease-causing mutations. To advance the assessment and development of genome editing tools, a robust mouse model is valuable, particularly for evaluating in vivo activity and delivery strategies. In this study, we successfully generated a knock-in mouse line carrying the Traffic Light Reporter design known as TLR-multi-Cas variant 1 (TLR-MCV1). We comprehensively validated the functionality of this mouse model for both in vitro and in vivo nuclease and prime editing. The TLR-MCV1 reporter mouse represents a versatile and powerful tool for expediting the development of editing technologies and their therapeutic applications.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 570-582 |
| Number of pages | 13 |
| Journal | CRISPR Journal |
| Volume | 6 |
| Issue number | 6 |
| DOIs | |
| State | Published - Dec 1 2023 |
| Externally published | Yes |
Bibliographical note
Publisher Copyright:© Mary Ann Liebert, Inc.