AI Article Synopsis
- Osteopetrosis presents a challenging medical issue due to the lack of effective and safe treatments for its various forms.
- Recent research advancements in understanding the disease's causes and the development of specialized models are aiding the creation of targeted therapies.
- Innovative experimental approaches being explored include gene correction in stem cells, gene therapy, protein replacement, and other methods to improve treatment outcomes for osteopetrosis.
Article Abstract
The medical treatment of osteopetrosis is an ongoing clinical problem. There are no effective and safer therapeutic approaches for all its forms. However, recent discoveries concerning the etiology and the pathogenesis of osteopetrosis, the development of dedicated cellular and animal models, and the advent of new technologies are paving the way for the development of targeted and safer therapies for both lethal and milder osteopetrosis. This review summarizes the huge effort and successes made by researchers to identify and develop new experimental approaches with this objective, such as the use of non-genotoxic myeloablation, gene correction of inducible Pluripotent Stem Cells (iPSCs), lentiviral-based gene therapy, protein replacement, prenatal treatment, osteoclast precursors transplantation and RNA Interference.
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| http://dx.doi.org/10.1016/j.bone.2022.116567 | DOI Listing |
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