AAV-based gene editing of type 1 collagen mutation to treat osteogenesis imperfecta

• Egile Nagusiak: Yeon-Suk Yang (Egilea), Tadatoshi Sato (Egilea), Sachin Chaugule (Egilea), Hong Ma (Egilea), Jun Xie (Egilea), Guangping Gao (Egilea), Jae-Hyuck Shim (Egilea)
• Formatua: Liburua
• Argitaratua: Elsevier, 2024-03-01T00:00:00Z.
• Gaiak: article
• Sarrera elektronikoa: Connect to this object online.

Osteogenesis imperfecta (OI) is a genetic disorder characterized by bone fragility, low bone mass, fractures, and extraskeletal manifestations. Since OI is commonly caused by single-nucleotide mutation(s) in the COL1A1 or COL1A2 genes encoding type I collagens, we developed a genome-editing strategy...