Stem Cell Based Exosomes: Are They Effective in Disease or Health?

Abstract

Exosomes are nano-sized vesicles involved in intercellular communication via delivery of molecules including lipids, nucleic acids, proteins, or other cellular components to distant or neighboring sites. Their ability to pass biological barriers, stability in physiological fluids without degradation, and distinctive affinity to target cells make exosomes very remarkable therapeutic vehicles. Virus-based approaches are some of the most widely used gene therapy methods; however, there are many issues need to be clarified such as high immunogenicity. Using of the exosomes procures the functional transfer of their cargo with minimal intervention from the immune system and it has been reported to be secure and well-tolerated. When the regenerative medicine is taken into consideration, stem cell-based approaches have been aimed to utilize but the general efficacy and safety profile of stem cell therapy has still not been enlightened. At this point, stem cell-derived exosomes exhibit a way to procure cell-free regenerative medicine with their unique characteristics. Exosomes are considered as appropriate and highly stable biological nano-vectors taking part in a wide variety of healthy and pathological processes for advanced targeted therapies. However, there are still crucial obstacles to achieve efficient isolation of large amount of specific and pure exosomes. Thus, large-scale exosome production under good manufacturing practice is required. The purpose of this review is to focus on stem cell-based exosomes for gene delivery and to introduce synthetic exosome-mimics as a potential alternative in the field of targeted gene therapies. Further, we aim to highlight the biobanking and large-scale manufacturing methods of exosomes.

Keywords: Biobanking; Exosome mimics; Exosomes; Gene delivery; Manufacturing; Stem cells.