Stem cell-derived extracellular vesicles for rare diseases: A clinical-bottleneck-to-mechanism framework for translation.
A new framework matches stem cell-derived particles to rare disease mechanisms, guiding which treatments to develop first.
This review proposes a clinically driven translational framework for stem cell-derived extracellular vesicles in rare diseases, matching each disease category to its dominant pathological barrier and appropriate EV function/delivery strategy. The framework addresses manufacturing, regulatory, and off-target risk challenges that currently limit rare disease EV translation.
What the study was
- Study design
- Narrative review with framework proposal
- Population
- Rare disease patients (autoimmune, neurodegenerative, skeletal, metabolic)
- Category
- Drug Development
- Maturity
- Exploratory
- Journal
- Bioscience Trends
Why it surfaced
Novel translational framework for EV therapy in rare diseases; addresses multiple rare disease categories with matched biological rationale. Found via T9 fallback query.
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