Biblio
A homozygous variant in INTS11 links mitosis and neurogenesis defects to a severe neurodevelopmental disorder. Cell Rep. 2023;42(12):113445.
Targeted addition of mini-dystrophin into rDNA locus of Duchenne muscular dystrophy patient-derived iPSCs. Biochem Biophys Res Commun. 2021;545:40-45.
ssODN-Mediated In-Frame Deletion with CRISPR/Cas9 Restores FVIII Function in Hemophilia A-Patient-Derived iPSCs and ECs. Mol Ther Nucleic Acids. 2019;17:198-209.
. Targeting of the Human F8 at the Multicopy rDNA Locus in Hemophilia A Patient-Derived iPSCs Using TALENickases. Biochem Biophys Res Commun. 2016.