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

YS Yang, T Sato, S Chaugule, H Ma, J Xie… - … Therapy-Nucleic Acids, 2024 - cell.com
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 …

[HTML][HTML] Restoration of Osteogenesis by CRISPR/Cas9 Genome Editing of the Mutated COL1A1 Gene in Osteogenesis Imperfecta

H Jung, YA Rim, N Park, Y Nam, JH Ju - Journal of Clinical Medicine, 2021 - mdpi.com
Osteogenesis imperfecta (OI) is a genetic disease characterized by bone fragility and
repeated fractures. The bone fragility associated with OI is caused by a defect in collagen …

[HTML][HTML] Generation of a heterozygous COL1A1 (c. 3969_3970insT) osteogenesis imperfecta mutation human iPSC line, MCRIi001-A-1, using CRISPR/Cas9 editing

HH Far, YN Patria, A Motazedian, AG Elefanty… - Stem Cell Research, 2019 - Elsevier
To develop a disease model for the human 'brittle bone'disease, osteogenesis imperfecta,
we have used gene editing to produce a facsimile of the patient heterozygous COL1A1 …

[HTML][HTML] The use of simultaneous reprogramming and gene correction to generate an osteogenesis imperfecta patient COL1A1 c. 3936 G> T iPSC line and an …

S Howden, HH Far, A Motazedian, AG Elefanty… - Stem Cell Research, 2019 - Elsevier
To develop a disease model for the human 'brittle bone'disease, osteogenesis imperfecta,
we used a simultaneous reprogramming and CRISPR-Cas9 genome editing method to …

[HTML][HTML] Gene targeting of mutant COL1A2 alleles in mesenchymal stem cells from individuals with osteogenesis imperfecta

JR Chamberlain, DR Deyle, U Schwarze, P Wang… - Molecular Therapy, 2008 - cell.com
Mesenchymal stem cells (MSCs) are adult cells with the capacity to differentiate into multiple
cell types, including bone, fat, cartilage, and muscle cells. In order to effectively utilize …

[HTML][HTML] CRISPR/Cas9 editing to generate a heterozygous COL2A1 p. G1170S human chondrodysplasia iPSC line, MCRIi019-A-2, in a control iPSC line, MCRIi019-A

LHW Kung, L Sampurno, KM Yammine, A Graham… - Stem Cell Research, 2020 - Elsevier
To develop an in vitro disease model of a human chondrodysplasia, we used CRISPR/Cas9
gene editing to generate a heterozygous COL2A1 exon 50 c. 3508 GGT> TCA (p. G1170S) …

[HTML][HTML] Normal collagen and bone production by gene-targeted human osteogenesis imperfecta iPSCs

DR Deyle, IF Khan, G Ren, PR Wang, J Kho… - Molecular Therapy, 2012 - cell.com
Osteogenesis imperfecta (OI) is caused by dominant mutations in the type I collagen genes.
In principle, the skeletal abnormalities of OI could be treated by transplantation of patient …

[HTML][HTML] CRISPR-Cas9 targeting of MMP13 in human chondrocytes leads to significantly reduced levels of the metalloproteinase and enhanced type II collagen …

CI Seidl, TA Fulga, CL Murphy - Osteoarthritis and Cartilage, 2019 - Elsevier
Objective To investigate the efficacy of CRISPR-Cas9 mediated editing in human
chondrocytes, and to develop a genome editing approach relevant to cell-based repair …

Targeting gene therapy for osteogenesis imperfecta

DJ Prockop - New England Journal of Medicine, 2004 - Mass Medical Soc
Targeting Gene Therapy for Osteogenesis Imperfecta Page 1 The new england journal of
medicine n engl j med 350;22 www.nejm.org may 27, 2004 2302 clinical implications of basic …

Potential therapeutic strategies for osteoarthritis via CRISPR/Cas9 mediated gene editing

R Vlashi, X Zhang, H Li, G Chen - Reviews in Endocrine and Metabolic …, 2024 - Springer
Osteoarthritis (OA) is an incapacitating and one of the most common physically degenerative
conditions with an assorted etiology and a highly complicated molecular mechanism that to …