Abstract
No curative treatment of OXPHOS disorders is currently available, despite great progress in our understanding of the molecular bases of these diseases. We review available and experimental therapeutic approaches that fall into the following categories:
(a) Palliative therapy; (b) removal of noxious metabolites; (c) administration of artificial electron acceptors; (d) administration of metabolites and cofactors; (e) administration of oxygen radical scavengers and f-gene therapy.
Progress in each of these approaches provides some glimmer of hope for the future, although much work remains to be done.
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Sengers, R.C.A., Trijbels, J.M.F., Boelen, C.C.A., Morava, E., Smeitink, J.A.M. (2004). Therapeutic Options in OXPHOS Disorders. In: Oxidative Phosphorylation in Health and Disease. Medical Intelligence Unit. Springer, Boston, MA. https://doi.org/10.1007/0-387-26992-4_10
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DOI: https://doi.org/10.1007/0-387-26992-4_10
Publisher Name: Springer, Boston, MA
Print ISBN: 978-0-306-48232-8
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