Article
|
Open Access
Featured
-
-
Article |
AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice
The feasibility of adeno-associated-virus-delivered nonsense suppressor tRNAs operating on premature termination codons (AAV-NoSTOP) is explored to restore gene function, using a mouse model of mucopolysaccharidosis type I for proof of concept.
- Jiaming Wang
- , Yue Zhang
- & Dan Wang
-
Article |
Transposon-encoded CRISPR–Cas systems direct RNA-guided DNA integration
A programmable transposase integrates donor DNA at user-defined genomic target sites with high fidelity, revealing a new approach for genetic engineering that obviates the need for DNA double-strand breaks and homologous recombination.
- Sanne E. Klompe
- , Phuc L. H. Vo
- & Samuel H. Sternberg