Mesenchymal stem cell-derived exosomes: Shaping the next era of Alzheimer’s disease treatment

Abstract

Alzheimer’s disease (AD) is a multifaceted neurodegenerative disease for which effective disease-modifying therapies are lacking. Mesenchymal stem cell-derived exosomes (MSC-Exos) have emerged as a promising therapeutic approach due to their unique biological functions and favorable biocompatibility. This review systematically explores the mechanism of action of MSC-Exos in AD therapy, including the removal of β-amyloid via the delivery of degradative enzymes, modulation of neuroinflammation, and promotion of neural regeneration. Meanwhile, this paper summarizes recent advances in preclinical and clinical studies, and analyzes the challenges in production standardization, safety assessment, and long-term efficacy validation of exosome therapies. Finally, several innovative strategies are proposed to enhance the therapeutic potential of MSC-Exos, including exosome functionalization and targeting optimization, gene editing techniques. This aims to promote the translation of exosomes from basic research to clinical application.

Keywords: Alzheimer’s disease; Mesenchymal stem cells; Exosomes; Precision medicine; Cell-free therapy

Core Tip: Mesenchymal stem cell-derived exosomes represent a promising therapeutic modality for Alzheimer’s disease, owing to their biological functions and biocompatibility. Mesenchymal stem cell-derived exosomes facilitate the clearance of β-amyloid by carrying degradative enzymes, modulate neuroinflammation, and promote neurorestoration. While preclinical and clinical data are encouraging, challenges such as production standardization, safety, and long-term efficacy remain. Strategies like exosome functionalization, targeting optimization, gene editing, and synthetic exosomes may enhance therapeutic potential and accelerate clinical translation.