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Nucleic Acids and Precision Therapies for Neuroprotection
About this book
Neurodegenerative disorders are rising worldwide, yet most approved drugs merely temper symptoms. Nucleic Acids and Precision Therapies for Neuroprotection addresses this therapeutic shortfall directly, tracing how antisense oligonucleotides, RNA interference agents, mRNA platforms, aptamers, and CRISPR-class editors are redefining what "treatable" means for Alzheimer's, Parkinson's, ALS, Huntington's disease, and Multiple Sclerosis.
Rather than cataloguing modalities in isolation, the book lays out an end-to-end translational roadmap from target identification and sequence optimisation to blood–brain barrier traversal, immunogenicity management, scalable manufacturing, and ethical oversight. Delivery science (including viral, nanoparticle, and hybrid vectors), AI-enabled biomarker discovery, adaptive trial architectures, and shifting global regulatory frameworks are viewed as interlocking elements of a single development pathway.
By aligning disparate scientific "dialects" into a shared, practice-oriented language, Nucleic Acids and Precision Therapies for Neuroprotection offers what the field currently lacks: a cohesive guide for moving nucleic acid therapeutics from concept to clinic with precision. Researchers, clinicians, regulators, and industry teams will find pragmatic frameworks, comparative tables, and decision points that shorten the path between mechanistic insight and patient impact without sacrificing rigour or transparency.
KEY FEATURES:
- Integrates molecular design, delivery engineering, clinical trial strategy, and regulatory science into one end-to-end translational workflow for nucleic acid therapeutics in neurology
- Delivers disease-specific playbooks (AD, PD, ALS, HD, MS) that map genotype/phenotype data to actionable targets, modality selection, and delivery routes
- Embeds AI/ML-driven biomarker discovery, patient stratification, and manufacturability analytics alongside ethical, access, and cost considerations
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Information
Table of contents
- Cover Page
- Half Title page
- Title Page
- Copyright Page
- Contents
- Foreword
- Preface
- Acknowledgments
- About the Author
- 1 Introduction to Neurodegenerative Disorders
- 2 Traditional Therapies for Neurodegenerative Diseases
- 3 Nucleic Acid Therapies for Neurodegenerative Disorders
- 4 Antisense and RNA–Interference Strategies for Neurodegeneration
- 5 Aptamer and mRNA Platforms for Receptor Targeting and Protein Replacement
- 6 Genome Editing and Next-Generation Nucleic-Acid Tools
- 7 Precision Medicine and Nucleic Acid Therapies for Neurodegenerative Disorders
- 8 Challenges in the Development and Application of Nucleic Acid Therapies for Neurodegenerative Disorders
- 9 Approaches for the Delivery of Nucleic Acid Therapies in Managing Neurodegenerative Disorders
- 10 AI and Nucleic Acid Therapeutics in Neurodegenerative Disease
- 11 Clinical Trials of Nucleic Acid Therapies for Neurodegenerative Disorders
- 12 Regulatory Considerations for Nucleic Acid Therapies in Neurodegenerative Disorders
- 13 Future Perspectives and Challenges in Nucleic Acid Therapies for Neurodegenerative Disorders
- Index
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