Frontiers in neuroscienceAug 22, 2026

Turning brain cells into neurons to treat neurological diseases: uses, challenges, and future possibilities

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗OA ↗

Updated Aug 23, 2026

Abstract

Direct neuronal reprogramming may provide a promising alternative for neuronal replacement in neurodegenerative diseases.

Neurological diseases often result from the irreversible loss of terminally differentiated neurons.
Induced pluripotent stem cells face limitations due to risks like tumor formation and immune rejection.
Directly converting non-neuronal cells into functional neurons could bypass these issues.
Key transcription factors are being investigated for their role in transforming glial cells into neurons.
Contamination issues in adeno-associated virus delivery systems could affect therapy outcomes.
Future research may focus on expanding reprogramming targets and improving techniques for regulation.

Simplified

Neurological diseases, often caused by irreversible loss of terminally differentiated neurons, present considerable challenges to treatment due to the limited regenerative capacity of these neurons. Although induced pluripotent stem cells hold promise for neuronal regeneration, their clinical application is constrained by risks, including tumorigenicity, incomplete neuronal maturation, and immune rejection. Recent advancements in direct neuronal reprogramming, which bypasses the intermediate pluripotent stage by directly converting non-neuronal cells into functional neurons, offer a compelling alternative for in situ neuronal replacement in neurodegenerative diseases. Key transcription factors, such as NeuroD1, Ascl1, Sox2, as well as CRISPR activation (CRISPRa) of NGN2 and ISL1, have been explored to convert glial cells into neurons. However, several challenges remain. This review discusses the current applications of direct neuronal reprogramming technology in several neurological diseases. We further highlight the potential contamination issues in adeno-associated virus (AAV) delivery systems and propose a code of conduct to avoid artifacts and pitfalls. Finally, we point out future directions for expanding direct reprogramming targets, integrating organoid-based disease modeling, and advancing reprogramming regulation techniques.

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Turning brain cells into neurons to treat neurological diseases: uses, challenges, and future possibilities

Abstract

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