RNAi Therapeutic Delivery by Exosomes

  • Samira Lakhal
  • Samir El Andaloussi
  • Aisling J. O’Loughlin
  • Jinghuan Li
  • Matthew M. J. Wood
Part of the Advances in Delivery Science and Technology book series (ADST)


Since the discovery of short interfering RNAs (siRNAs), their potential as a therapeutic platform has been widely recognized. However, clinical translation has been stalled by inefficient delivery in vivo. While some success has been achieved with cationic lipids and lipid-like materials for therapeutic RNAi delivery to liver, delivery across the blood–brain barrier (BBB) to the central nervous system for the treatment of neurological disorders such as Parkinson’s, Alzheimer’s, and Huntington’s disease remains a challenge. To address the problem of inefficient delivery across the BBB, our laboratory exploited one of nature’s mechanisms for intercellular communication, named exosomes. They are a class of membrane vesicles derived from the endolysosomal compartment implicated in cell–cell communication by shuttling various proteins, lipids, and RNAs between cells. We have developed a method to target exosomes with brain-specific peptides and subsequently load them with siRNA for targeted delivery to brain. This chapter aims at providing an insight into membrane vesicle-mediated RNA delivery and how these vectors can be utilized for RNAi therapy.


Membrane Vesicle Recipient Cell Human Brain Microvascular Endothelial Cell Drug Delivery Vehicle Rabies Virus Glycoprotein 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.


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Copyright information

© Controlled Release Society 2013

Authors and Affiliations

  • Samira Lakhal
    • 1
  • Samir El Andaloussi
    • 1
  • Aisling J. O’Loughlin
    • 1
  • Jinghuan Li
    • 1
  • Matthew M. J. Wood
    • 1
  1. 1.Department of Physiology, Anatomy and GeneticsUniversity of OxfordOxfordUK

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