# Exosome treatment | Symptoms, Causes, Tests and Treatment | OSANG

> Exosomes are key molecules that mediate the therapeutic effects of stem cells, and are attracting attention as 'cell-free therapy' that overcomes the limitations of cell…

- Official page: https://osns.co.kr/en/encyclopedia/exosome-therapy
- Organization: OSANG Neurosurgery

## Page content

Regenerative Medicine

## Exosome treatment

Exosome Therapy · Z79.899

Exosome therapy is a regenerative medicine technique that uses exosomes, nano-sized (30-150 nm) extracellular vesicles secreted by cells, for treatment. It induces regenerative, anti-inflammatory, and neuroprotective effects by delivering growth factors, miRNA, and proteins to target tissues without direct cell transplantation.

## At a glance

Exosomes are key molecules that mediate the therapeutic effects of stem cells, and are attracting attention as 'cell-free therapy' that overcomes the limitations of cell transplantation. Mesenchymal stem cell-derived exosomes (MSC-exosomes) are the most studied, and their nerve regeneration, angiogenesis, and anti-inflammatory effects have been confirmed in preclinical studies. It has the ability to cross the blood-brain barrier and has great potential as a treatment for central nervous system diseases. Clinical application research is ongoing, and standardized manufacturing and quality control are key challenges for clinical translation.

- 01Definition and Overview

- 02Composition and biological properties of exosomes

- 03Mesenchymal stem cell-derived exosome (MSC-exosome)

- 04Nervous system applied research

- 05Manufacturing and standardization challenges

- 06Comparison: Exosomes vs Stem Cell Therapy

## Definition and Overview

Exosomes are nano-sized (30-150 nm) extracellular vesicles (EV) that originate from intracellular multivesicular bodies (MVBs) and are released outside the cell when fused with the cell membrane. It is secreted by all cells and functions as an important means of intercellular signaling, including proteins, miRNAs, mRNA, DNA, and lipids.

Exosome therapy is a method of inducing tissue regeneration, anti-inflammatory, and neuroprotective effects by administering exosomes for therapeutic purposes into the body. After research showed that much of the therapeutic effect of stem cells is mediated through exosomes secreted rather than the cells themselves, exosomes are attracting attention as a 'cell-free therapy' that uses exosomes without cell transplantation.

## Composition and biological properties of exosomes

Exosomes are vesicles surrounded by a phospholipid bilayer membrane, and their interior contains hundreds to thousands of proteins (surface markers such as CD63, CD81, and CD9), nucleic acids (miRNA, mRNA, ncRNA), and lipids.

The ability to cross the blood-brain barrier (BBB) is an important property of exosomes. Exosomes can be delivered across the BBB to the central nervous system through their small size and special surface molecules, showing potential for treating brain diseases that are difficult to access with existing drugs.

Exosomes show low immunogenicity, resulting in low immune response even during allogenic administration. This increases treatment accessibility by eliminating the need for autologous cell collection.

## Mesenchymal stem cell-derived exosome (MSC-exosome)

Exosomes secreted by mesenchymal stem cells (MSCs) are the most studied therapeutic exosomes. MSC-exosomes exhibit anti-inflammatory, immunomodulatory, neurotrophic, and angiogenic effects similar to those of parental cells (MSCs).

In a myocardial protection study, MSC-exosomes significantly reduced myocardial ischemia-reperfusion injury, showing a similar effect to direct MSC transplantation.

In a stroke model, intravenous administration of MSC-exosomes resulted in recovery of neurological function, angiogenesis, and increased neuroplasticity. This effect is related to the mechanism by which miRNAs, such as miR-133b in exosomes, regulate the expression of nerve growth-related genes.

## Nervous system applied research

A phase 1 clinical study of MSC-exosomes in post-stroke rehabilitation is ongoing. Preclinical research data is also accumulating in traumatic brain injury (TBI), spinal cord injury, ALS, and Parkinson's disease. The possibility of promoting recovery through delivery of neurotrophic factors in peripheral nerve damage and autonomic nerve dysfunction is also being studied. The possibility of special exosomes (derived from IFNγ-stimulated dendritic cells) to induce myelin regeneration in multiple sclerosis has been reported.

## Manufacturing and standardization challenges

The key tasks for clinical translation of exosome therapy are standardized manufacturing and quality control. The characteristics vary depending on the exosome isolation method (ultracentrifugation, size exclusion chromatography, precipitation reagent), and the source of the manufactured cells, culture conditions, and storage method affect efficacy. The Minimal Information for Studies of Extracellular Vesicles (MISEV) guidelines have been proposed to standardize studies.

## Comparison: Exosomes vs Stem Cell Therapy

The advantages of exosome therapy include a low risk of pulmonary embolism, immune rejection, and tumor formation due to cell transplantation, ease of cryopreservation and long-term storage, the ability to cross the blood-brain barrier, and the possibility of developing a standardized formulation. Disadvantages include lack of manufacturing standardization, mass production costs, and lack of clinical evidence.

## Frequently asked questions

### Q01What are exosomes?

Exosomes are very small, nano-sized vesicles secreted by cells. They are thousands of times smaller than cells, with a diameter of 30 to 150 nm. Proteins, genetic information (miRNA, mRNA), and lipids made in the cell are packaged into these vesicles and transferred to other cells. It is an important means by which cells communicate with each other and regulate their functions, and has been likened to an ‘intercellular postman’.

### Q02How is exosome therapy different from stem cell therapy?

Stem cell therapy is a method of injecting cells directly into the body, while exosome therapy is a 'cell-free treatment' that extracts and administers only exosomes secreted from stem cells. The therapeutic effects of stem cells are often due to signaling through exosomes rather than the cells themselves. Exosomes have little immune rejection, are easy to store frozen, and can pass through the blood-brain barrier, so they have great potential in treating central nervous system diseases. However, standardization challenges still remain.

### Q03How do exosomes help in nervous system regeneration?

Mesenchymal stem cell-derived exosomes contain nerve growth factors (BDNF, NGF, etc.) to promote neuronal survival and growth. It also contains anti-inflammatory factors, which reduce the inflammatory response at the site of nerve damage. It has the ability to cross the blood-brain barrier and can be transmitted directly to the central nervous system. In animal experiments, the effect of exosome administration after stroke was reported to promote recovery of nerve function and vascular regeneration.

### Q04To what extent is exosome therapy currently used clinically?

Exosome therapy is currently producing many positive results in preclinical studies (animal experiments), and phase 1 and 2 clinical studies are in progress for various diseases such as stroke, ALS, diabetic foot ulcers, and hair loss. Some clinics perform exosome injections for anti-aging, nerve regeneration, and pain control purposes, but since most are in the clinical research stage, you must consult with a specialist to determine suitability until sufficient evidence on effectiveness and safety is established.

### Q05What are the side effects of exosome treatment?

The side effects reported to date are known to be generally safe, with only mild pain, redness, and temporary fever at the injection site. However, long-term safety data are not yet sufficient. Effectiveness and safety may vary depending on the source of the exosome (from which cell), manufacturing method, and dosage. It is important to use quality controlled products from certified medical institutions.

## Related articles

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- stem cell therapy

- Extracorporeal shock wave therapy

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