Rahim, Ahad A;
Kurian, Manju A;
Zhou, Haiyan;
Ferguson, Ross;
Tabrizi, Sarah J;
Lignani, Gabriele;
Aquilina, Kristian;
(2025)
Genetic therapies for neurological diseases.
Pharmacological Reviews
, Article 100093. 10.1016/j.pharmr.2025.100093.
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Abstract
Often, gene therapy reviews concentrate upon specific therapeutic modalities – particularly either viral vector-mediated or a non-viral approach. Here, we draw together a comprehensive array of knowledge across the field of genetic therapy for genetic neurological disease. The sections on preclinical and clinical application of viral vectors are followed by sections on RNA-based therapies and then by antisense oligonucleotide approaches also in preclinical and clinical settings. We present a separate section on gene editing strategies and conclude with a section elaborating upon the neurosurgical techniques and the expertise required for clinical application of many of these technologies.
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