Transient expression of an adenine base editor corrects the Hutchinson-Gilford progeria syndrome mutation and improves the skin phenotype in mice
oleh: Daniel Whisenant, Kayeong Lim, Gwladys Revêchon, Haidong Yao, Martin O. Bergo, Piotr Machtel, Jin-Soo Kim, Maria Eriksson
| Format: | Article |
|---|---|
| Diterbitkan: | Nature Portfolio 2022-06-01 |
Deskripsi
Base editing to treat diseases is progressing but tissue delivery and progenitor cells correction are challenging. Here, the authors show sustained effects and propagation of mutation-corrected progenitors by transient adenine base editor expression, improving the skin phenotype of HGPS mice.