Pharmacological and biological therapeutic strategies for osteogenesis imperfecta

R Marom, YC Lee, I Grafe, B Lee - American Journal of Medical …, 2016 - Wiley Online Library
Osteogenesis imperfecta (OI) is a connective tissue disorder characterized by bone fragility,
low bone mass, and bone deformities. The majority of cases are caused by autosomal …

Osteogenesis imperfecta: prospects for molecular therapeutics

A Forlino, JC Marini - Molecular genetics and metabolism, 2000 - Elsevier
… of the mutant α2(I) collagen allele in type IV Osteogenesis Imperfecta fibroblasts. A molecular
approach to therapeutics of dominant negative disorders. J Clin Invest 97:448–454, 1996. …

Pathophysiology and therapeutic options in osteogenesis imperfecta: an update

E Brizola, TM Felix, JR Shapiro - Research and reports in …, 2016 - Taylor & Francis
Osteogenesis imperfecta (OI) is a rare clinically and genetically heterogeneous systemic
disorder of bone and connective tissue characterized by bone fragility and physical findings …

Treatment options for osteogenesis imperfecta

R Besio, A Forlino - Expert Opinion on Orphan Drugs, 2015 - Taylor & Francis
Introduction: Osteogenesis imperfecta (OI) is a heritable disease characterized by bone
fragility. A wide range of clinical severity and heterogeneity of the molecular defects is reported. …

New frontiers for dominant osteogenesis imperfecta treatment: gene/cellular therapy approaches

R Besio, A Forlino - Advances in Regenerative Biology, 2015 - Taylor & Francis
… 1 Gene therapy strategies to target mutant collagen transcripts. (a) Antisense
oligodeoxyribonucleotides (ODNs) are single-stranded DNA molecules that upon their entry into a …

Osteogenesis imperfecta in childhood: treatment strategies

RHH Engelbert, HEH Pruijs, FA Beemer… - Archives of physical …, 1998 - Elsevier
Osteogenesis imperfecta (01) is a skeletal disorder of remarkable clinical variability
characterized by bone fragility, osteopenia, variable degrees of short stature, and progressive …

Current and emerging treatments for the management of osteogenesis imperfecta

E Monti, M Mottes, P Fraschini, PC Brunelli… - Therapeutics and …, 2010 - Taylor & Francis
Osteogenesis imperfecta (OI) is the most common bone genetic disorder and it is
characterized by bone brittleness and various degrees of growth disorder. Clinical severity varies …

[HTML][HTML] Management of osteogenesis imperfecta

SH Ralston, MS Gaston - Frontiers in endocrinology, 2020 - frontiersin.org
Osteogenesis imperfecta is the term used to describe a group of inherited disorders … As
new genes for osteogenesis imperfecta have been discovered a new classification system has …

[HTML][HTML] Osteogenesis imperfecta: current and prospective therapies

M Botor, A Fus-Kujawa, M Uroczynska, KL Stepien… - Biomolecules, 2021 - mdpi.com
… the OI phenotype may be a more promising therapeutic strategy than correction of the structural
… A summary of the current experimental strategies for OI therapy is presented in Figure 3. …

New perspectives on osteogenesis imperfecta

A Forlino, WA Cabral, AM Barnes… - Nature Reviews …, 2011 - nature.com
… for osteogenesis imperfecta as a collagen-related disorder. The more prevalent autosomal
dominant forms of osteogenesis imperfecta are … of dominant osteogenesis imperfecta include …