Associated Papers
Development of Engineered-U1 snRNA Therapies: Current Status.
Gonçalves M et al.
International Journal of Molecular Sciences, 2023
Prime Editing for Human Gene Therapy: Where Are We Now?
Godbout K et al.
Cells, 2023
A Compensatory U1snRNA Partially Rescues FAH Splicing and Protein Expression in a Splicing-Defective Mouse Model of Tyrosinemia Type I.
Balestra D et al.
International Journal of Molecular Sciences, 2020
Gene and Base Editing as a Therapeutic Option for Cystic Fibrosis-Learning from Other Diseases.
Mention K et al.
Genes, 2019
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