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Methods Mol Biol ; 2275: 227-245, 2021.
Artigo em Inglês | MEDLINE | ID: mdl-34118041

RESUMO

Genetic mutations and defects in mitochondrial DNA (mtDNA) are associated with certain types of mitochondrial dysfunctions, ultimately resulting in the emergence of a variety of human diseases. To achieve an effective mitochondrial gene therapy, it will be necessary to deliver therapeutic agents to the innermost mitochondrial space (the mitochondrial matrix), which contains the mtDNA pool. We recently developed a MITO-Porter, a liposome-based nanocarrier that delivers cargo to mitochondria via a membrane-fusion mechanism. In this chapter, we discuss the methodology used to deliver bioactive molecules to the mitochondrial matrix using a Dual Function (DF)-MITO-Porter, a liposome-based nanocarrier that delivers it cargo by means of a stepwise process, and an evaluation of mtDNA levels and mitochondrial activities in living cells. We also discuss mitochondrial gene silencing by the mitochondrial delivery of antisense RNA oligonucleotide (ASO) targeting mtDNA-encoded mRNA using the MITO-Porter system.


Assuntos
DNA Mitocondrial/genética , Mitocôndrias/genética , RNA Antissenso/farmacologia , RNA Mitocondrial/genética , DNA Mitocondrial/efeitos dos fármacos , Sistemas de Liberação de Medicamentos , Inativação Gênica , Células HeLa , Humanos , Lipossomos , Potencial da Membrana Mitocondrial/efeitos dos fármacos , Microscopia Confocal , Mutação , RNA Antissenso/química , RNA Mitocondrial/efeitos dos fármacos
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