Your new post is loading...
According to a study conducted on primates by Intellia Therapeutics, CRISPR/Cas9 would work better than conventional genetic therapy for increasing the activity of factor IX (FIX), the blood clotting protein that is missing in people with hemophilia B. Researchers have shown that CRISPR/Cas9-mediated gene insertion can lead to higher levels of FIX activity over time compared to conventional gene therapy that uses adeno-associated viral vectors (AAVs) to deliver modified genes to cells. According to the company, a six-week study demonstrated a successful increase in FIX activity, at or above normal limits, in the blood of non-human primates. FIX activity levels can be refined by adjusting several parameters, such as CRISPR/Cas9 doses. Also, in a mouse model of liver regeneration, the companies’ scientists demonstrated that unlike conventional gene therapy, CRISPR/Cas9 can allow gene constructs to remain active inside liver cells even as they divide and expand in order to restore the tissue that was lost. The researchers took part of the animals' livers to induce tissue regeneration. After 14 days, the activity levels of the artificial FIX fell by 85% with the gene delivered using standard gene therapy. In contrast, when CRISPR / Cas9 was used, FIX activity levels remained stable for at least 45 days.
