Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles.
Publication/Presentation Date
8-1-2022
Abstract
Fibrodysplasia ossificans progressiva (FOP) is a rare and devastating genetic disease, in which soft connective tissue is converted into heterotopic bone through an endochondral ossification process. Patients succumb early as they gradually become trapped in a second skeleton of heterotopic bone. Although the underlying genetic defect is long known, the inherent complexity of the disease has hindered the discovery of effective preventions and treatments. New developments in the gene therapy field have motivated its consideration as an attractive therapeutic option for FOP. However, the immune system's role in FOP activation and the as-yet unknown primary causative cell, are crucial issues which must be taken into account in the therapy design. While gene therapy offers a potential therapeutic solution, more knowledge about FOP is needed to enable its optimal and safe application.
Volume
33
Issue
15-16
First Page
782
Last Page
788
ISSN
1557-7422
Published In/Presented At
Eekhoff, E. M. W., de Ruiter, R. D., Smilde, B. J., Schoenmaker, T., de Vries, T. J., Netelenbos, C., Hsiao, E. C., Scott, C., Haga, N., Grunwald, Z., De Cunto, C. L., di Rocco, M., Delai, P. L. R., Diecidue, R. J., Madhuri, V., Cho, T. J., Morhart, R., Friedman, C. S., Zasloff, M., Pals, G., … Micha, D. (2022). Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles. Human gene therapy, 33(15-16), 782–788. https://doi.org/10.1089/hum.2022.023
Disciplines
Business Administration, Management, and Operations | Health and Medical Administration | Management Sciences and Quantitative Methods
PubMedID
35502479
Department(s)
Administration and Leadership
Document Type
Article