RNA Therapeutics
The Evolving Landscape of RNA Therapeutics
- 1st Edition - April 13, 2022
- Latest edition
- Editors: Paloma H. Giangrande, Vittorio de Franciscis, John J. Rossi
- Language: English
RNA Therapeutics: The Evolving Landscape of RNA Therapeutics provides a comprehensive overview of RNA therapeutic modalities, from bench-to-bedside, with an emphasis on the incre… Read more
RNA Therapeutics: The Evolving Landscape of RNA Therapeutics provides a comprehensive overview of RNA therapeutic modalities, from bench-to-bedside, with an emphasis on the increasingly impactful areas of gene therapy, oligonucleotide therapeutics, gene editing and delivery. International leaders in the field examine RNA-based therapeutics tools that have been developed to-date to modulate cellular processes such as transcription, translation and protein function. Approved RNA-based therapies and lessons learned from failed therapies are discussed in-depth, as are evolving advances in RNA biochemical analysis, and similar advances that are enabling clinical application of RNA-based therapies.
Later sections discuss delivery technologies, remaining hurdles in research and translation, the therapy development process from the lab to the clinic, and novel RNA-based therapies currently in development.
- Features leading experts in the field of RNA therapeutics, spanning all classes of RNA therapies
- Provides a detailed examination of approved RNA therapies and lessons learned from failed therapeutics
- Covers all aspects of therapeutic discovery and preclinical development, as well as clinical translation, manufacturing and regulatory aspects
Active researchers in biochemistry, human genetics, molecular biology, cell biology, pharmacology; researchers in industry and pharma. Students of biochemical sciences; clinicians across disease specialties
SECTION 1 The world of RNA therapeutics: Available RNA tools to modulate cellular processes
1. Targeting chromatin: Transcriptional gene activation (saRNA)
Jon Voutila and Nagy A Habib
2. Argonaute and TNRC6, partners in RNAi
David R. Corey, Krystal Courtney Johnson, and Samantha Johnson
3. Targeted RNA therapeutics for treatment of cancer and immunomodulation
Zhuoran Zhang, Yu-Lin Su, Chunsong Yu, Marice Alcantara, Daniel Rossi, and Marcin Kortylewski
4. Extrahepatic delivery of RNA to immune cells
Inbal Hazan-Halevy, Dalit Landesman-Milo, Edo Kon, Niels Dammes, and Dan Peer
SECTION 2 Learning from history
5. Aptamer-based protein inhibitors
Haixiang Yu, James Frederiksen, and Bruce A. Sullenger
6. Lessons learned from developing an oligonucleotide drug for a rare disease
Patricia Soblechero-Martín and Virginia Arechavala-Gomeza
SECTION 3 Delivery
7. Nucleoside modifications of in vitro transcribed mRNA to reduce immunogenicity and improve translation of prophylactic and therapeutic antigens
Mohamad-Gabriel Alameh and Drew Weissman
8. SOMAmer reagents and the SomaScan platform: Chemically modified aptamers and their applications in therapeutics, diagnostics, and proteomics
Daniel J. Schneider, Sean A. Lynch, Amy D. Gelinas, Rachel M. Ostroff, John C. Rohloff, Preston Williams, Nebojsa Janjic, and Daniel W. Drolet
SECTION 4 From bench to bedside
9. CMC and regulatory aspects of oligonucleotide therapeutics
Thomas M. Rupp and Hagen Cramer
10. CMC: Regulatory landscape
Marc M. Lemaitre
11. CMC and manufacturing
Christopher Lincoln
SECTION 5 From bench to bedside
12. RNA delivery for cancer gene therapy
Selin Oncul, Paola Amero, Cristian Rodriguez-Aguayo, Anil K. Sood, George A. Calin, and Gabriel Lopez-Berestein
13. Combinatorial RNA therapies in cancer immunotherapy: Challenges and directions
Kevin Holm, Jessica Alluin, Min-Sun Song, Jiehua Zhou, and John Rossi
- Edition: 1
- Latest edition
- Published: April 13, 2022
- Language: English
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Paloma H. Giangrande
Dr. Paloma Giangrande is the VP of Biology at Wave Life Sciences, Cambridge, MA and Adjunct Associate Professor of Internal Medicine at the University of Iowa, Iowa City, IA. Her career has been dedicated to the investigation and development of RNA-based therapeutic approaches (including RNA aptamers, siRNAs, and mRNA) for cancer, cardiovascular disease, rare diseases, and critical illness. As a consequence, she has developed a deep understanding of these technologies, diseases, and associated disease mechanisms. She is also an internationally recognized expert in oligonucleotide therapeutics and delivery. Towards this end, she was the first to demonstrate that RNA ligands (aptamers) can be used to deliver therapeutic siRNAs to target cells. Her 60+ publications and 9+ patents are a testament to this body of work and her commitment to the development of novel RNA-based therapies for many diseases.
Affiliations and expertise
Associate Professor of Internal Medicine, University of Iowa, Iowa City, IA; Director Rare Diseases, ModernaTx Inc, Cambridge, MA, USAVd
Vittorio de Franciscis
Dr. Vittorio de Franciscis is Senior Collaborator at the CNR Institute of Genetic and Biomedical Research (IRGB) in Milan Italy. For more than ten years, the interest of the research has focused on translating the understanding of the molecular basis of neoplastic transformation in the design of selective therapies based on the use of therapeutic RNAs (aptamers, siRNAs, miRNAs). In his laboratory, they demonstrated the possibility of generating aptamers that bind to high affinity and inhibit transmembrane receptors involved in cancer progression (including, EGFR, AXL, PDGFR, IR, Eph). For the selection of aptamers they have developed a variant of the SELEX technology that makes use of living cells as a complex target. They have recently shown that such RNA aptamers against RTK can be used as targeting moieties for the selective delivery of therapeutic miRNAs and anti-miRs, against tumor cells and tissues.
Affiliations and expertise
Senior Collaborator, Research Director, CNR, Institute of Experimental Endocrinology and Oncology, Naples, ItalyJR
John J. Rossi
Affiliations and expertise
City of Hope's Beckman Research Institute, Duarte, California, U.S.A.Read RNA Therapeutics on ScienceDirect