The power of CRISPR became definitively clear when the first CRISPR-based gene therapy, Casgevy (exa-cel), won regulatory approvals for the treatment of sickle cell disease. But CRISPR’s applications ...
Genetic disorders occur due to alterations in the primary genetic material—deoxyribonucleic acid (DNA)—of an organism.
“Through CRISPR RNA optimization, we achieved around 59% editing at the TTR locus in our in vitro experiments. In the mouse ...
Understanding human gene function in living organisms has long been hampered by fundamental differences between species.
Discover the innovative TECHNO platform for full-length gene humanisation, enhancing models for human gene function.
This session presents two linked talks on CRISPR–Cas9 model generation and sequence validation. Christopher Raymond will discuss how direct zygote editing in mice and rats reduces timelines and costs ...
AZoLifeSciences on MSN
Replacing large mouse gene regions with human DNA
Humanized mouse models are essential for investigating human gene function; however, the complete replacement of mouse genes ...
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