Peripheral Nerve Repair & Regeneration2025
Engineered AAV Capsids Mediate Transduction of Murine Neurofibroma and Sciatic Nerve
Abou Haidar E, Prabhakar S, Cheah PS, Hanlon KS, Espinoza P, Crain AV, Patel N, Radcliff GW, Cheng M, Hernández IC, Minderler S, de la Cruz D, Ng C, da Hora CC, Charest A, Stemmer-Rachamimov A, Jowett N, Breakefield XO, Maguire CA
Gene Ther
What Is This Study About?
This study engineered novel viral capsids (AAV) that can deliver genes directly to tumors and nerves associated with neurofibromatosis. The engineered vectors successfully transduced both neurofibroma tissue and sciatic nerve in mice.
Key Findings
- Engineered AAV capsids achieved targeted gene delivery to peripheral nerve tumors
- Successfully transduced both neurofibroma tissue and healthy sciatic nerve in mice
- Opens potential for gene therapy approaches to nerve-associated tumors
What This Means for Patients
This research lays groundwork for potential gene therapies targeting nerve tumors, which could offer less invasive treatment options for patients with neurofibromatosis.
Citation & Links
Citation: Abou Haidar E, Prabhakar S, et al. Gene Ther. 2025 Jun 10.
PMID: 40494929