Genetic therapies for neurological diseases

Area of Science:

• Neurology
• Genetics
• Biotechnology

Background:

• Genetic neurological diseases are often life-limiting.
• Current reviews often focus narrowly on specific gene therapy modalities.
• A comprehensive overview of diverse gene therapy strategies is needed.

Purpose of the Study:

• To provide a comprehensive review of gene therapy approaches for genetic neurological diseases.
• To consolidate knowledge across various therapeutic modalities.
• To discuss clinical applications and necessary expertise.

Main Methods:

• Literature review synthesizing preclinical and clinical data.
• Categorization of therapies including viral vectors, RNA-based treatments, antisense oligonucleotides, and gene editing.
• Inclusion of neurosurgical techniques and expertise requirements.

Main Results:

• Gene therapies show significant potential for treating neurological disorders.
• A wide array of genetic therapeutic strategies are under investigation and development.
• Successful clinical deployment requires specialized neurosurgical expertise.

Conclusions:

• Genetic therapies offer promising avenues for treating debilitating neurological conditions.
• A multidisciplinary approach integrating various gene therapy techniques and surgical expertise is crucial for clinical success.
• This review provides a foundational resource for understanding the landscape of gene therapy in neurology.