RNA Therapeutics

The Evolving Landscape of RNA Therapeutics

Häftad, Engelska, 2022

Av Paloma H. Giangrande, Vittorio de Franciscis, John J. Rossi, USA) Giangrande, Paloma H. (Associate Professor of Internal Medicine, University of Iowa, Iowa City, IA; Director Rare Diseases, ModernaTx Inc, Cambridge, MA, Italy) de Franciscis, Vittorio (Senior Collaborator, Research Director, CNR, Institute of Experimental Endocrinology and Oncology, Naples, U.S.A.) Rossi, John J. (City of Hope's Beckman Research Institute, Duarte, California, Vittorio de Franciscis

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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

Produktinformation

Utgivningsdatum2022-04-13
Mått191 x 235 x 32 mm
Vikt970 g
FormatHäftad
SpråkEngelska
Antal sidor472
FörlagElsevier Science
ISBN9780128215951

Tillhör följande kategorier

Biologi inom Naturvetenskap och teknik
Biokemi inom Naturvetenskap och teknik

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. 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.

SECTION 1 The world of RNA therapeutics: Available RNA tools to modulate cellular processes1. Targeting chromatin: Transcriptional gene activation (saRNA)2. Argonaute and TNRC6, partners in RNAi3. Targeted RNA therapeutics for treatment of cancer and immunomodulation4. Extrahepatic delivery of RNA to immune cellsSECTION 2 Learning from history5. Aptamer-based protein inhibitors6. Lessons learned from developing an oligonucleotide drug for a rare diseaseSECTION 3 Delivery7. Nucleoside modifications of in vitro transcribed mRNA to reduce immunogenicity and improve translation of prophylactic and therapeutic antigens8. SOMAmer reagents and the SomaScan platform: Chemically modified aptamers and their applications in therapeutics, diagnostics, and proteomicsSECTION 4 From bench to bedside9. CMC and regulatory aspects of oligonucleotide therapeutics10. CMC: Regulatory landscape11. CMC and manufacturingSECTION 5 From bench to bedside12. RNA delivery for cancer gene therapy13. Combinatorial RNA therapies in cancer immunotherapy: Challenges and directions