Biblio
Preclinical evaluation of CRISPR-based therapies for Noonan syndrome caused by deep-intronic variants. Mol Ther Nucleic Acids. 2024;35(1):102123.
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Generation of homozygous CRISPRa human induced pluripotent stem cell (hiPSC) lines for sustained endogenous gene activation. Stem Cell Res. 2020;48:101944.
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