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Current progress on microRNAs ‐based therapeutics in neurodegenerative diseases

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MicroRNAs (miRNAs)‐based therapy has recently emerged as a promising strategy in the treatments of neurodegenerative diseases. Thus, in this review, the most recent and important challenges and advances on the development of miRNA therapeutics for brain targeting are discussed. In particular, this review highlights current knowledge and progress in the field of manufacturing, recovery, isolation, purification, and analysis of these therapeutic oligonucleotides. Finally, the available miRNA delivery systems are reviewed and an analysis is presented in what concerns to the current challenges that have to be addressed to ensure their specificity and efficacy. Overall, it is intended to provide a perspective on the future of miRNA‐based therapeutics, focusing the biotechnological approach to obtain miRNAs. WIREs RNA 2017, 8:e1409. doi: 10.1002/wrna.1409

Schematic representation of microRNA‐based therapeutic approaches.
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Main barriers to successful in vivo delivery of microRNAs and mechanisms of transport across blood–brain barrier (BBB).
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Flowchart of the main unit operations to be considered during manufacturing process of microRNAs (miRNAs). It is shown a comparison between the most common methodologies for obtaining miRNA from cells, tissues, or Escherichia coli with the recombinant production methodology in Rhodovulum sulfidophilum. It is also indicated which are the impurities eliminated in each step.
[ Normal View | Magnified View ]

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RNA Processing > Processing of Small RNAs
Regulatory RNAs/RNAi/Riboswitches > Regulatory RNAs
RNA in Disease and Development > RNA in Disease

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