M. Rapoport, D. Marcus, A. Saada, T. Erlich, R. Hadad, H. Greif, M. Lichtenstein, H. Lorberboum-Galski, Mitochondrion, Volume 24, Supplement, September 2015, Page S35, ISSN 1567-7249, doi:10.1016/j.mito.2015.07.096.
The approach is to fuse the Mitochondrial targeting Sequence (MTS), with the delivery peptide TAT [HIV-transactivator of transcription (TAT) peptide]. This novel approach has been tested using different mitochondrial proteins implicated in mitochondrial human diseases: Lipoamide Dehydrogenase (LAB), C6ORF66 and Frataxin, and have been evaluated in vitro, in patients' cells and in vivo, in mouse models. In patient's cells and in mice tissues, including the brain, AT-MTS-Mitochondrial fusion proteins arrive at the cells and their mitochondria rapidly and efficiently, getting an improvement of the mitochondrial functions and life span in animal models.
Thursday, September 17, 2015
Repurposing riluzole to treat hereditary cerebellar ataxia
Heather Wood; Nature Reviews Neurology (2015) doi:10.1038/nrneurol.2015.161 Published online 15 September 2015
Given the limited availability of new therapies for neurological disease, repurposing of existing drugs is an approach that is being increasingly explored. A randomized controlled trial, conducted in Italy provides evidence that this drug could also be beneficial in patients with hereditary cerebellar ataxia, spinocerebellar ataxia and Friedreich ataxia.
Given the limited availability of new therapies for neurological disease, repurposing of existing drugs is an approach that is being increasingly explored. A randomized controlled trial, conducted in Italy provides evidence that this drug could also be beneficial in patients with hereditary cerebellar ataxia, spinocerebellar ataxia and Friedreich ataxia.
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