Self-Derived Exosomes as a Novel Gene Therapy
Description
This template was adapted from the original submission. Edits were made to enhance scientific accuracy, optimal usability and/or to meet industry-leading design standards for science communication.
This template illustrates the main steps to obtain self-derived exosomes for use as a novel gene therapy approach. Exosomes are small, cell-derived vesicles containing microRNA (miRNA) and small interfering RNA (siRNA) that are capable of genetic exchange between cells. Exosome can be derived from a patient's differentiated hematopoietic stem cells and used for tissue-targeted cargo delivery through the expression of tissue-specific peptides. By loading miRNA or siRNA of the targeted gene, exosomes can selectively regulate gene expression.
Acknowledgements
References
Alvarez-Erviti, L., et al.. (2011) Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes. Nat Biotechnol. https://doi.org/10.1038/nbt.1807
Mathiyalagan, P., et al.. (2017) Exosomes-Based Gene Therapy for MicroRNA Delivery. Methods Mol Biol. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5502074/
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