Targeting Trafficking in Drug Development by Alfredo Ulloa-Aguirre & Ya-Xiong Tao

Author:Alfredo Ulloa-Aguirre & Ya-Xiong Tao
Language: eng
Format: epub
Publisher: Springer International Publishing, Cham

5 Regenerative Bone Medicine for Treatment of Syndromes Linked to Defects in Type I Procollagen Secretion

For a number of protein traffic-related disorders, clinical presentation occurs at early stages of life, including both prenatal and paediatric stages (Aridor and Hannan 2000). This feature is particularly evident in skeletal and craniofacial syndromes where the effects of type I PC deficit begin during foetal life and often present with abnormal skeletal development, shortened bones or multiple fractures and malformations (Jobling et al. 2014). Therefore, appropriate strategies may attempt to treat the foetus before additional pathology occurs. To date, because no cures have been developed for the treatment of skeletal disorders, the field of OI has sought regenerative approaches to enhance bone formation and counteract bone fragility. Such schemes have become a requirement since the only pharmacological treatment available for these patients, bisphosphonate therapy, cannot correct the primary defect of inadequate collagen production (Bembi et al. 1997).

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