Nucleic Acid Therapeutics
Volume II: Research and Development
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Beschreibung
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.
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.
Integrates molecular biology, chemistry, delivery and clinical science across disciplines Highlights emerging technologies like LNPs, CRISPR variants, and AI-based design tools Provides disease-specific applications with global regulatory and translational relevance
Autor*in
Zhiguo Wang
Themen in »Nucleic Acid Therapeutics«
Nucleic Acid Therapeutics Multitarget drugs Antisense Oligonucleotides Splice Modulation mRNA Therapeutics Regulatory and Translational Pathways