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

• Main Authors: Yeon-Suk Yang (Author), Tadatoshi Sato (Author), Sachin Chaugule (Author), Hong Ma (Author), Jun Xie (Author), Guangping Gao (Author), Jae-Hyuck Shim (Author)
• Format: Book
• Published: Elsevier, 2024-03-01T00:00:00Z.
• Subjects: article
• Online Access: 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...