Mahato, Biraj ; Jash, Sukanta ; Adhya, Samit (2011) RNA-mediated restoration of mitochondrial function in cells harboring a Kearns Sayre Syndrome mutation Mitochondrion, 11 (4). pp. 564-574. ISSN 15677249
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Official URL: http://doi.org/10.1016/j.mito.2011.03.006
Related URL: http://dx.doi.org/10.1016/j.mito.2011.03.006
Abstract
Mutations in mitochondrial DNA (mtDNA) generate multi-system disorders due to failure of ATP production. A cybrid containing a 1.9-kb mtDNA deletion from a patient with Kearns Sayre Syndrome is respiration-defective and grows glycolytically. When treated with a ribonucleoprotein (RNP) complex of polycistronic RNA 1 (pcRNA1) containing mtDNA-encoded genes and a multi-subunit carrier complex R8, full-length pcRNA1 was transported to mitochondria. Translation of the pcRNA1-encoded mRNAs was observed in mitochondria from RNP-treated cells. Respiration of the cybrid was rescued to approximately 90% of normal within hours, switching the cells to aerobic growth. These findings have implications for the development of effective mitochondrial gene therapy.
| Item Type: | Article |
|---|---|
| Source: | Copyright of this article belongs to Elsevier B.V. |
| Keywords: | Mitochondrial deletion, RNA, RNA Import complex, Kearns Sayre Syndrome, Cybrid, Gene therapy |
| ID Code: | 129204 |
| Deposited On: | 22 Nov 2022 10:53 |
| Last Modified: | 22 Nov 2022 10:53 |
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