In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene.

• Tác giả: Gang Bao, Christine Beeton, Karl-Dimiter Bissig, Mingming Cao, Adam Castoreno, Tyler Chickering, Marcel A Chuecos, Marco De Giorgi, Alexandria M Doerfler, Mia N Furgurson, Jun Han, Ayrea Hurley, Sarah Hyde, Vasant Jadhav, Jong Kim, William R Lagor, Stephanie Lefebvre, Rich Gang Li, M Cecilia Ljungberg, James F Martin, Patrick Miller, So Hyun Park, Kalyani R Patel, June Qin, Lavanya Saxena, Mark K Schlegel, Christopher J Walkey, Kelly Wassarman, Ivan Zlatev
• Ngôn ngữ: eng
• Thông tin xuất bản: United States : Science translational medicine , 2025
• Bộ sưu tập: NCBI
• ID: 179291

Tóm tắt

Homology-directed repair (HDR)-based genome editing is an approach that could permanently correct a broad range of genetic diseases. However, its utility is limited by inefficient and imprecise DNA repair mechanisms in terminally differentiated tissues. Here, we tested Repair Drive, a platform technology for selectively expanding HDR-corrected hepatocytes in adult mice in vivo. Repair Drive involves transient conditioning of the liver by knocking down an essential gene,