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## Meet the editor

Dr. Irina Vlasova-St. Louis obtained her MD and Ph.D. from Ural State Medical Academy, Russia. She completed her postdoctoral training at the University of Minnesota, USA, and a laboratory fellowship sponsored by the Lymphoma Research Foundation. She served as an assistant professor at the University of Minnesota, Department of Medicine, providing expertise in several biological disciplines including infectious diseases,

immunology, genomics, and bioinformatics. During the COVID-19 pandemic, Dr. Vlasova-St. Louis led genomic surveillance of circulating SARS-CoV-2 variants of concern in Nevada, USA. Currently, she is a genomics specialist at the Newborn Screening Department, Texas Department of State Health Services, USA. She is sponsored by federal grants from the Association of Public Health Laboratories and Centers for Disease Control and Prevention. She is a part of the Johns Hopkins University Advanced Academic Program: Individualized Genomics and Health.

### Contents



Preface

*"It's time for scientists to shout about RNA therapies"*

*RNA Therapeutics - History, Design, Manufacturing, and Applications* addresses recent advances in RNA-based drug discovery and commercialization. The design and development of RNA drugs have resulted in several distinct classes of treatment known as nucleic acid therapies. Chapter 1: "Introductory Chapter: RNA Drugs Development and Commercialization" describes and discusses these therapies, which include an impressive list of mRNA, SARNA, miRNA, siRNA, RNA analogs, ribozymes, anti-

The COVID-19 pandemic spurred a burst of development of mRNA vaccines, which culminated in rapid mRNA vaccine testing and approval for use in humans. Chapter 2 "Prophylactic Ribonucleic Acid Vaccines to Combat RNA Viral Infections in Humans" discusses these vaccines, while Chapter 3 "Ribozymes as Therapeutic Agents against

Applications of RNA therapeutics range from infectious disease prophylaxis to treating cancer and chronic conditions, improving organ transplant outcomes, and correcting inherited mutations. Chapter 4: "A New Era of RNA Personalized Vaccines for Cancer and Cancer-Causing Infectious Diseases" discusses RNA therapeutics for various types of malignancies originatingfrom chronic viral infections or somatic mutations. Chapter 5: "Perspective Chapter: RNA Therapeutics for Cancers" reviews distinct classes of RNA therapeutics as well as examines the challenges in RNA drug

engineering, delivery, and improvement of pharmacological effectiveness.

health and thus hold great promise to modernize health care.

The optimization of RNA-based therapeutics enables countless opportunities in our pursuit of achieving the goals of individualized medicine. This is particularly applicable to rare genetic disorders for which RNA drugs may provide a cure. As such, Chapter 6: "RNA Interference Applications for Machado-Joseph Disease" discusses applications of RNA interferences for a rare neurodegenerative disease caused by abnormal expansion of trinucleotide repeats in non-coding regions of RNAs.

RNA therapeutics have significantly impacted medicine, economy, and overall public

sense oligonucleotides, and CRISPR-based drugs.

Infectious Diseases" discusses therapeutic ribozymes.

*Lorna Harries (Nature 574, S15. 2019)*
