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- W1720268051 endingPage "1196" @default.
- W1720268051 startingPage "1183" @default.
- W1720268051 abstract "Introduction: Friedreich ataxia (FRDA) is a progressive neurodegenerative disease affecting ∼ 1/50,000 people. The disease is caused by guanine–adenine–adenine repeat expansions in the frataxin gene, which results in frataxin deficiency. Cardiac complications occur in a majority of patients and are the leading cause of death in FRDA. Presently, there are no therapeutic agents to slow the disease progression. Recently, gene and cell therapies have been proposed as future treatments for addressing the debilitating cardiac features of the disease.Areas covered: This review describes potential gene and cell therapy approaches to treating cardiac complications in FRDA. Cell therapies include induced pluripotent stem cells and bone marrow-derived mesenchymal stem cells. Gene therapy options consist of lentiviral, herpes simplex virus and adeno-associated virus (AAV) vectors for gene transfer, with AAV-frataxin vectors reaching late preclinical stages of testing.Expert opinion: Gene and cell therapies are progre..." @default.
- W1720268051 created "2016-06-24" @default.
- W1720268051 creator A5002108062 @default.
- W1720268051 creator A5010542220 @default.
- W1720268051 creator A5038264883 @default.
- W1720268051 creator A5049007341 @default.
- W1720268051 creator A5055857472 @default.
- W1720268051 date "2015-08-31" @default.
- W1720268051 modified "2023-10-18" @default.
- W1720268051 title "Prospects of gene and cell therapy for managing cardiac complications in Friedreich ataxia" @default.
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