Lethal Osteogenesis Imperfecta Resulting from a Single Nucleotide Change in One Human proα 1(I) Collagen Allele

Transfer of proα2(I) cDNA into cells of

Transfer of proα2(I) cDNA into cells of a murine model of human Osteogenesis Imperfecta restores synthesis of type I collagen comprised of α1(I) and α2(I) heterotrimers in vitro and in vivo

✍ Christopher Niyibizi; Patrick Smith; Zhibao Mi; Charlotte L. Phillips; Paul Robb 📂 Article 📅 2001 🏛 John Wiley and Sons 🌐 English ⚖ 197 KB

## Abstract The __oim__ mouse is a model of human Osteogenesis Imperfecta (OI) that has deficient synthesis of proα2(I) chains. Cells isolated from __oim__ mice synthesize α1(I) collagen homotrimers that accumulate in tissues. To explore the feasibility of gene therapy for OI, a murine proα2(I) cDN