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Open Access 01-12-2024 | Gene Therapy in Oncology | Review Article

Taking the knife to neurodegeneration: a review of surgical gene therapy delivery to the CNS

Authors: Andrea Perera, Olivier Brock, Aminul Ahmed, Chris Shaw, Keyoumars Ashkan

Published in: Acta Neurochirurgica | Issue 1/2024

Abstract

Gene supplementation and editing for neurodegenerative disorders has emerged in recent years as the understanding of the genetic mechanisms underlying several neurodegenerative disorders increases. The most common medium to deliver genetic material to cells is via viral vectors; and with respect to the central nervous system, adeno-associated viral (AAV) vectors are a popular choice. The most successful example of AAV-based gene therapy for neurodegenerative disorders is Zolgensma© which is a transformative intravenous therapy given to babies with spinal muscular atrophy. However, the field has stalled in achieving safe drug delivery to the central nervous system in adults for which treatments for disorders such as amyotrophic lateral sclerosis are desperately needed. Surgical gene therapy delivery has been proposed as a potential solution to this problem. While the field of the so-called regenerative neurosurgery has yielded pre-clinical optimism, several challenges have emerged. This review seeks to explore the field of regenerative neurosurgery with respect to AAV-based gene therapy for neurodegenerative diseases, its progress so far and the challenges that need to be overcome.

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Title: Taking the knife to neurodegeneration: a review of surgical gene therapy delivery to the CNS
Authors: Andrea Perera
Olivier Brock
Aminul Ahmed
Chris Shaw
Keyoumars Ashkan
Publication date: 01-12-2024
Publisher: Springer Vienna
Keywords: Gene Therapy in Oncology
Amyotrophic Lateral Sclerosis
Published in: Acta Neurochirurgica / Issue 1/2024
Print ISSN: 0001-6268
Electronic ISSN: 0942-0940
DOI: https://doi.org/10.1007/s00701-024-06028-8

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Taking the knife to neurodegeneration: a review of surgical gene therapy delivery to the CNS

Abstract

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

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