New Publication in Cell: AniView Supports Mitochondrial Transfer Therapy Research

New progress has been made in organelle therapy based on mitochondrial capsule delivery systems for the treatment of Parkinson’s disease and other mitochondrial dysfunction-related disorders.

Researchers Xingguo Liu and Qi Long from the Guangzhou Medical University, in collaboration with the Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Sciences and multiple partner institutions, have made new advances in organelle therapy research. The related findings have been published in Cell (IF = 42.5, top-tier journal).

This study provides new insights into erythrocyte membrane encapsulated mitochondrial delivery systems (mitochondrial capsules) for efficient mitochondrial transplantation, improved cellular energy restoration, and functional recovery in multiple disease models including Parkinson’s disease, mitochondrial DNA depletion syndrome, and Leigh syndrome.

Mitochondria, as the “powerhouses” of the cell, play a crucial role in maintaining cellular fate and human health. Mitochondrial dysfunction is the root cause of many major diseases, including neurodegenerative disorders, diabetes, liver diseases, eye diseases, and aging. Among them, mitochondrial diseases are a unique class of genetic disorders caused by abnormalities in nuclear-encoded mitochondrial proteins or mutations in mitochondrial DNA (mtDNA). These diseases affect more than 0.02% of the population, and current treatments can only alleviate symptoms without correcting the underlying biochemical or genetic defects. Although gene therapy and gene-editing technologies have made progress in treating mitochondrial diseases, their application is still limited by issues such as poor compatibility of editing systems, limited efficiency, off-target effects, and ethical concerns. Other diseases associated with mitochondrial dysfunction, including neurodegenerative diseases, ischemia-reperfusion injury, metabolic disorders, and aging, also lack effective mitochondrial repair strategies. Therefore, direct transplantation of healthy mitochondria to replace or repair damaged ones is considered a highly promising therapeutic strategy, especially for diseases caused by mtDNA mutations. However, efficient delivery of exogenous mitochondria into cells and tissues remains a major unsolved challenge. Although natural intercellular mitochondrial transfer exists and “three-parent baby” technology has achieved mitochondrial replacement at the embryonic level, these approaches cannot treat already affected patients. Previous studies of free mitochondrial transplantation have shown promise in animal models and early clinical research, but a more efficient and broadly applicable delivery strategy is urgently needed to realize its therapeutic potential.

To address this challenge, the authors developed a high-efficiency mitochondrial delivery system based on red blood cell membrane encapsulation. Healthy mitochondria were isolated from donors and encapsulated within a membrane layer to form “mitochondrial capsules”, which were then delivered into recipient cells. This strategy significantly enhanced intracellular ATP production, increased mtDNA levels, and reduced mtDNA mutations, thereby restoring impaired cellular functions. Ultimately, this therapy was applied in mouse models and successfully alleviated symptoms of multiple mitochondrial dysfunction–related diseases, including Parkinson’s disease, mitochondrial DNA depletion syndrome, and Leigh syndrome.

Figure 1. A new approach for disease treatment via mitochondrial transplantation

Experiments using Aniview

To evaluate the in vivo delivery efficiency and tissue specificity of the mitochondrial capsules, mitochondrial luciferase signals in transgenic mice were monitored using the AniView multimodal in vivo imaging system from Bioligh Biotechnology. Whole-body imaging showed strong bioluminescence signals in the abdominal region of the capsule-treated group, significantly higher than those of the control group, indicating enhanced mitochondrial activity in vivo (Figure 2). In brain-specific imaging, only mice injected with mitochondrial capsules exhibited high-intensity luminescence in the brain, whereas no signal was detected in the free mitochondrial group (Figure 3). This demonstrates that the capsule system not only functions systemically but also has the ability to cross physiological barriers such as the blood–brain barrier and efficiently deliver functional mitochondria to the brain.

Figure 2. Effects of mitochondrial capsules (+mito capsule) on in vivo bioluminescent signals in mt-Akaluc mice

Figure 3. Brain targeting capability of mitochondrial capsules (mito capsule)

DOI: 10.1016/j.cell.2026.02.023