Nucleic Acid Therapeutics

Volume II: Research and Development
Erscheinungsjahr 2026
ISBN: 978-981-9246-33-5
Verlag: Springer

Buch, Englisch, 499 Seiten, Format (B × H): 155 mm x 235 mm

This book provides a systematic and interdisciplinary introduction to nucleic acid therapeutics (NATs)—a rapidly advancing class of biopharmaceuticals based on engineered DNA and RNA molecules. It features clinically validated and experimental technologies such as siRNA, mRNA, CRISPR, and RNA circuits, and offers insight into multitarget NAT design guided by polypharmacology principles. Special emphasis is placed on chemical modification, delivery systems, regulatory pathways, and synthetic biology tools. Readers will benefit from practical guidance, translational relevance, and conceptual clarity that bridge molecular mechanisms and therapeutic applications. The book is intended for researchers, clinicians, students, and professionals seeking to advance innovation in genomics-driven drug development and modern precision medicine.

This volume II focuses on the translational and developmental aspects of nucleic acid drugs, including chemical modifications, GMP-compliant manufacturing, and delivery technologies.

Zielgruppe

Research

Autoren/Hrsg.

Wang, Zhiguo

Zhang, Xiaofang

Wang, Jinghao

Fachgebiete

• Medizin | Veterinärmedizin Medizin | Public Health | Pharmazie | Zahnmedizin Medizinische Fachgebiete Pharmakologie, Toxikologie
• Medizin | Veterinärmedizin Medizin | Public Health | Pharmazie | Zahnmedizin Klinische und Innere Medizin
• Technische Wissenschaften Verfahrenstechnik | Chemieingenieurwesen | Biotechnologie Biotechnologie
• Naturwissenschaften Biowissenschaften Molekularbiologie

Inhaltsverzeichnis

Chapter 21. DNA-Based Gene Silencing and Knockdown: Antisense and shRNA.- Chapter 22. DNA-Based Gene Silencing and Knockdown: DNA-AGO and DNAzyme.- Chapter 23. Splicing Modulation.- Chapter 24. DNA-Based Gene Overexpression.- Chapter 25. DNA-Based Gene Activation and Silencing.- Chapter 26. Blockade of Protein–Protein or Protein–Ligand Interactions.- Chapter 27. Chemical Modifications I: Sugar Ring.- Chapter 28. Chemical Modifications II: Backbone.- Chapter 29. Chemical Modifications III: Nucleobase.- Chapter 30. Chemical Modifications IV: Terminal and Conjugation.- Chapter 31. Chemical Modifications V: Comparative Analysis and Combined Strategies.- Chapter 32. Overview of Nucleic Acid Drug Delivery Technologies.- Chapter 33. Emerging Delivery Technologies for Nucleic Acid Therapeutics: DNA Nanostructures and DNA Origami.- Chapter 34. Formulation Development and GMP Manufacturing of Nucleic Acid Therapeutics.- Chapter 35. Procedures and Regulatory Framework for The Development of Nucleic Acid Therapeutics.- Chapter 36. Radionuclide Labeling Strategies and Process Optimization for Nucleic Acid Therapeutics.- Chapter 37. Safety and Toxicology Issues of Nucleic Acid Therapeutics.- Chapter 38. Genetic and Epigenetic Therapy.- Chapter 39. Therapeutic Applications of Nucleic Acid Drugs: Disease Case Studies.- Chapter 40. AI and In Silico Tools in Nucleic Acid Drug Discovery.- Chapter 41. Combinatorial and Multimodal Nucleic Acid Therapies.- Chapter 42. Future Challenges and Outlook.

Über Autor(innen)

Zhiguo Wang is a Professor in the Department of Pharmacy, Director of the Guangdong Provincial Key Laboratory for Chronic Disease Interactive Network Research, Dean of MetAge Institute, and Chief Scientist, at the First Affiliated Hospital of Jinan University, China. His research focuses primarily on nucleic acid drug development and polypharmacology, particularly in the context of metabolism-related chronic diseases and aging. He earned his PhD in cardiovascular pharmacology from the Department of Pharmacology and Therapeutics at McGill University, Canada. Recognized as one of the pioneers in the field of polypharmacology, he was the first to propose the concept of microRNA interference. Over the past two decades, Professor Wang has developed multiple technological platforms for research and drug discovery involving miRNA, saRNA, antisense oligonucleotides, mRNA, and translatable circular RNA (circRNA), with an emphasis on their integration into multitarget therapeutic strategies. He has authored three monographs and four book chapters with Springer-Verlag/Springer Nature.

Xiaofang Zhang is a Research Scientist in the Department of Pharmacy at the First Affiliated Hospital of Jinan University and a core investigator at the Guangdong Provincial Key Laboratory for Chronic Disease Interactive Network Research & Guangzhou Key Laboratory of Basic and Translational Research for Chronic Diseases. Her research focuses on cardiovascular disease mechanisms and therapeutic development, with expertise in nucleic acid drug discovery (siRNA/saRNA/mRNA), polypharmacology-driven multitarget therapeutics, and preclinical validation for heart failure/sudden cardiac death, alongside early-stage nucleic acid drug development for oncology (hepatocellular/pancreatic cancer) and alopecia. She has designed and validated novel nucleic acid drugs demonstrating superior efficacy in animal models for cardiovascular indications and other diseases. She serves as Committee Member of the Youth Committee of Cardiovascular Pharmacology and the 2nd Committee of Drug Metabolism (Guangdong Pharmacological Society), having been secured leadership/participation in >20 competitive grants. Her achievements include 34 SCI publications in high-impact journals.

Jinghao Wang, Ph.D., is Professor and Chief Pharmacist in the Department of Pharmacy at the First Affiliated Hospital of Jinan University, concurrently serving as Director of the Joint Academician Laboratory and Guangzhou Key Laboratory of Basic and Translational Research for Chronic Diseases, and Executive Director of the Laboratory of Basic and Translational Research for Radiopharmaceuticals. His research focuses on cardiovascular pharmacology, drug discovery for chronic diseases, and radiopharmaceuticals. He earned his Ph.D. and M.S. in Pharmacology from Harbin Medical University under Academician Baofeng Yang. Professor Wang leads 11 research projects, and has authored over 15 SCI papers as first or corresponding author. He has edited 6 academic books and developed 9 expert consensus guidelines, while his honors include the Hospital Pharmacy Science Award (First Prize, Guangdong) and Outstanding Pharmacist recognition. He serves holds editorial and committee leadership roles in the Chinese Pharmacological Society and Guangdong Pharmaceutical Association.