Associated Papers
Prime editing optimized RTT permits the correction of the c.8713C>T mutation in DMD gene.
Happi Mbakam C et al.
Molecular Therapy. Nucleic Acids, 2022
A cryptic splice-altering KCNQ1 variant in trans with R259L leading to Jervell and Lange-Nielsen syndrome.
Torrado M et al.
Npj Genomic Medicine, 2021
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