Self-Derived Exosomes as a Novel Gene Therapy

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.

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