Nomlabofusp, a Fusion Protein of Human Frataxin and a Cell Penetrant Peptide, Delivers Mature and Fu...
Nomlabofusp, a Fusion Protein of Human Frataxin and a Cell Penetrant Peptide, Delivers Mature and Functional Frataxin into Mitochondria
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Cham: Springer International Publishing
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Language
English
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Cham: Springer International Publishing
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Contents
Friedreich’s ataxia is a rare, progressive, genetic disorder, the root cause of which is a significant deficiency in the mitochondrial protein frataxin. Frataxin is ubiquitously expressed, but its deficiency results in a variety of debilitating symptoms, with disease severity, rate of progression and age of onset inversely correlating with tissue f...
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Full title
Nomlabofusp, a Fusion Protein of Human Frataxin and a Cell Penetrant Peptide, Delivers Mature and Functional Frataxin into Mitochondria
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TN_cdi_proquest_miscellaneous_3181809820
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_3181809820
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ISSN
1550-7416
E-ISSN
1550-7416
DOI
10.1208/s12248-025-01054-5