Author: Fernando Aranda Vega
Publisher: Elsevier
ISBN: 0323994016
Category : Science
Languages : en
Pages : 326
Book Description
MRNA-Based Therapeutics, Volume 372 in the International Review of Cell and Molecular Biology series, covers topics surrounding the effect of different metabolic situations, their contribution to metabolic modulation, and their impact on tumor growth. Specific chapters in this release include New era of nucleic acid therapies: Clinical applications and perspectives, Messenger RNA as personalized therapy: time of truth for rare metabolic disease, Applications of Self-Replicating RNA, mRNA therapy in PKU, Advances in gene-editing technologies, mRNA delivery technologies: towards clinical translation, Advances in mRNA vaccines, and more. Provides the authority and expertise of leading contributors from an international board of authors Presents the latest release in International Review of Cell and Molecular Biology serials Updated release includes the latest information on MRNA-Based Therapeutics
MRNA-Based Therapeutics
Author:
Publisher: Academic Press
ISBN: 0323994091
Category : Science
Languages : en
Pages : 328
Book Description
MRNA-Based Therapeutics, Volume 372 in the International Review of Cell and Molecular Biology series, covers topics surrounding the effect of different metabolic situations, their contribution to metabolic modulation, and their impact on tumor growth. Specific chapters in this release include New era of nucleic acid therapies: Clinical applications and perspectives, Messenger RNA as personalized therapy: time of truth for rare metabolic disease, Applications of Self-Replicating RNA, mRNA therapy in PKU, Advances in gene-editing technologies, mRNA delivery technologies: towards clinical translation, Advances in mRNA vaccines, and more. - Provides the authority and expertise of leading contributors from an international board of authors - Presents the latest release in International Review of Cell and Molecular Biology serials - Updated release includes the latest information on MRNA-Based Therapeutics
Publisher: Academic Press
ISBN: 0323994091
Category : Science
Languages : en
Pages : 328
Book Description
MRNA-Based Therapeutics, Volume 372 in the International Review of Cell and Molecular Biology series, covers topics surrounding the effect of different metabolic situations, their contribution to metabolic modulation, and their impact on tumor growth. Specific chapters in this release include New era of nucleic acid therapies: Clinical applications and perspectives, Messenger RNA as personalized therapy: time of truth for rare metabolic disease, Applications of Self-Replicating RNA, mRNA therapy in PKU, Advances in gene-editing technologies, mRNA delivery technologies: towards clinical translation, Advances in mRNA vaccines, and more. - Provides the authority and expertise of leading contributors from an international board of authors - Presents the latest release in International Review of Cell and Molecular Biology serials - Updated release includes the latest information on MRNA-Based Therapeutics
Synthetic mRNA
Author: Robert E. Rhoads
Publisher: Humana
ISBN: 9781493936236
Category : Medical
Languages : en
Pages : 0
Book Description
This volume presents detailed laboratory protocols for in vitro synthesis of mRNA with favorable properties, its introduction into cells by a variety of techniques, and the measurement of physiological and clinical consequences such as protein replacement and cancer immunotherapy. Synthetic techniques are described for structural features in mRNA that provide investigational tools such as fluorescence emission, click chemistry, photo-chemical crosslinking, and that produce mRNA with increased stability in the cell, increased translational efficiency, and reduced activation of the innate immune response. Protocols are described for clinical applications such as large-scale transfection of dendritic cells, production of GMP-grade mRNA, redirecting T cell specificity, and use of molecular adjuvants for RNA vaccines. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Synthetic mRNA: Production, Introduction into Cells, and Physiological Consequences is a valuable and cutting-edge resource for both laboratory investigators and clinicians interested in this powerful and rapidly evolving technology.
Publisher: Humana
ISBN: 9781493936236
Category : Medical
Languages : en
Pages : 0
Book Description
This volume presents detailed laboratory protocols for in vitro synthesis of mRNA with favorable properties, its introduction into cells by a variety of techniques, and the measurement of physiological and clinical consequences such as protein replacement and cancer immunotherapy. Synthetic techniques are described for structural features in mRNA that provide investigational tools such as fluorescence emission, click chemistry, photo-chemical crosslinking, and that produce mRNA with increased stability in the cell, increased translational efficiency, and reduced activation of the innate immune response. Protocols are described for clinical applications such as large-scale transfection of dendritic cells, production of GMP-grade mRNA, redirecting T cell specificity, and use of molecular adjuvants for RNA vaccines. Written in the highly successful Methods in Molecular Biology series format, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step laboratory protocols, and tips on troubleshooting and avoiding known pitfalls. Synthetic mRNA: Production, Introduction into Cells, and Physiological Consequences is a valuable and cutting-edge resource for both laboratory investigators and clinicians interested in this powerful and rapidly evolving technology.
Cardiac Regeneration
Author: Masaki Ieda
Publisher: Springer
ISBN: 3319561065
Category : Medical
Languages : en
Pages : 277
Book Description
This Volume of the series Cardiac and Vascular Biology offers a comprehensive and exciting, state-of-the-art work on the current options and potentials of cardiac regeneration and repair. Several techniques and approaches have been developed for heart failure repair: direct injection of cells, programming of scar tissue into functional myocardium, and tissue-engineered heart muscle support. The book introduces the rationale for these different approaches in cell-based heart regeneration and discusses the most important considerations for clinical translation. Expert authors discuss when, why, and how heart muscle can be salvaged. The book represents a valuable resource for stem cell researchers, cardiologists, bioengineers, and biomedical scientists studying cardiac function and regeneration.
Publisher: Springer
ISBN: 3319561065
Category : Medical
Languages : en
Pages : 277
Book Description
This Volume of the series Cardiac and Vascular Biology offers a comprehensive and exciting, state-of-the-art work on the current options and potentials of cardiac regeneration and repair. Several techniques and approaches have been developed for heart failure repair: direct injection of cells, programming of scar tissue into functional myocardium, and tissue-engineered heart muscle support. The book introduces the rationale for these different approaches in cell-based heart regeneration and discusses the most important considerations for clinical translation. Expert authors discuss when, why, and how heart muscle can be salvaged. The book represents a valuable resource for stem cell researchers, cardiologists, bioengineers, and biomedical scientists studying cardiac function and regeneration.
RNA Therapeutics
Author: Mouldy Sioud
Publisher: Humana Press
ISBN: 9781607616641
Category : Science
Languages : en
Pages : 527
Book Description
Central to the synthesis of proteins, the performance of catalysis, and many other physiological processes, the aberrant expression of which can be linked to human diseases including cancers, RNA has proven to be key target for therapeutics as well as a tool for therapy. In RNA Therapeutics: Function, Design, and Delivery, expert contributors from a broad spectrum of scientific backgrounds highlight the roles that messenger RNAs and small RNAs can play in biology and medicine. While covering the five major RNA-based drugs, namely the use of ribozymes to cleave and/or correct mRNA transcript, the use of siRNA for targeted silencing of gene transcripts, the use of aptamers, like short RNA molecules, for neutralizing the protein functions, the use mRNA-transfected DCs to activate immune system against tumor cells, as well as the use of RNA to reprogram T and/or DC cell function, this extensive volume brings together the fields of coding (mRNA) and non-coding RNA such as ribozymes, RNAse P, siRNAs, and miRNAs into one convenient source. Written in the highly successful Methods in Molecular BiologyTM series format, the cutting-edge protocol chapters contain introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and practical tips on troubleshooting and avoiding known pitfalls. Also, the book contains several excellent reviews for teaching purposes. Authoritative and comprehensive, RNA Therapeutics: Function, Design, and Delivery provides key models and tools which will assist researchers in increasing our understanding of RNA functions, modifications, and their involvement in diseases in order to lead to the design of vital new RNA-based therapeutics.
Publisher: Humana Press
ISBN: 9781607616641
Category : Science
Languages : en
Pages : 527
Book Description
Central to the synthesis of proteins, the performance of catalysis, and many other physiological processes, the aberrant expression of which can be linked to human diseases including cancers, RNA has proven to be key target for therapeutics as well as a tool for therapy. In RNA Therapeutics: Function, Design, and Delivery, expert contributors from a broad spectrum of scientific backgrounds highlight the roles that messenger RNAs and small RNAs can play in biology and medicine. While covering the five major RNA-based drugs, namely the use of ribozymes to cleave and/or correct mRNA transcript, the use of siRNA for targeted silencing of gene transcripts, the use of aptamers, like short RNA molecules, for neutralizing the protein functions, the use mRNA-transfected DCs to activate immune system against tumor cells, as well as the use of RNA to reprogram T and/or DC cell function, this extensive volume brings together the fields of coding (mRNA) and non-coding RNA such as ribozymes, RNAse P, siRNAs, and miRNAs into one convenient source. Written in the highly successful Methods in Molecular BiologyTM series format, the cutting-edge protocol chapters contain introductions to their respective topics, lists of the necessary materials and reagents, step-by-step, readily reproducible laboratory protocols, and practical tips on troubleshooting and avoiding known pitfalls. Also, the book contains several excellent reviews for teaching purposes. Authoritative and comprehensive, RNA Therapeutics: Function, Design, and Delivery provides key models and tools which will assist researchers in increasing our understanding of RNA functions, modifications, and their involvement in diseases in order to lead to the design of vital new RNA-based therapeutics.
MRNA-Based Therapeutics
Author: Fernando Aranda Vega
Publisher: Elsevier
ISBN: 0323994016
Category : Science
Languages : en
Pages : 326
Book Description
MRNA-Based Therapeutics, Volume 372 in the International Review of Cell and Molecular Biology series, covers topics surrounding the effect of different metabolic situations, their contribution to metabolic modulation, and their impact on tumor growth. Specific chapters in this release include New era of nucleic acid therapies: Clinical applications and perspectives, Messenger RNA as personalized therapy: time of truth for rare metabolic disease, Applications of Self-Replicating RNA, mRNA therapy in PKU, Advances in gene-editing technologies, mRNA delivery technologies: towards clinical translation, Advances in mRNA vaccines, and more. Provides the authority and expertise of leading contributors from an international board of authors Presents the latest release in International Review of Cell and Molecular Biology serials Updated release includes the latest information on MRNA-Based Therapeutics
Publisher: Elsevier
ISBN: 0323994016
Category : Science
Languages : en
Pages : 326
Book Description
MRNA-Based Therapeutics, Volume 372 in the International Review of Cell and Molecular Biology series, covers topics surrounding the effect of different metabolic situations, their contribution to metabolic modulation, and their impact on tumor growth. Specific chapters in this release include New era of nucleic acid therapies: Clinical applications and perspectives, Messenger RNA as personalized therapy: time of truth for rare metabolic disease, Applications of Self-Replicating RNA, mRNA therapy in PKU, Advances in gene-editing technologies, mRNA delivery technologies: towards clinical translation, Advances in mRNA vaccines, and more. Provides the authority and expertise of leading contributors from an international board of authors Presents the latest release in International Review of Cell and Molecular Biology serials Updated release includes the latest information on MRNA-Based Therapeutics
Exploring Novel Clinical Trial Designs for Gene-Based Therapies
Author: National Academies of Sciences, Engineering, and Medicine
Publisher: National Academies Press
ISBN: 0309672988
Category : Medical
Languages : en
Pages : 127
Book Description
Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.
Publisher: National Academies Press
ISBN: 0309672988
Category : Medical
Languages : en
Pages : 127
Book Description
Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.
Therapeutic Oligonucleotides
Author: Jens Kurreck
Publisher: Royal Society of Chemistry
ISBN: 0854041168
Category : Medical
Languages : en
Pages : 362
Book Description
This book provides a compelling overall update on current status of RNA interference
Publisher: Royal Society of Chemistry
ISBN: 0854041168
Category : Medical
Languages : en
Pages : 362
Book Description
This book provides a compelling overall update on current status of RNA interference
RNA Technologies and Their Applications
Author: Volker A. Erdmann
Publisher: Springer Science & Business Media
ISBN: 3642121683
Category : Medical
Languages : en
Pages : 460
Book Description
RNA technologies are the driving forces of modern medicine and biotechnology. They combine the fields of biochemistry, chemistry, molecular biology, cell biology, physics, nanotechnology and bioinformatics. The combination of these topics is set to revolutionize the medicine of tomorrow. After more than 15 years of extensive research in the field of RNA technologies, the first therapeutics are ready to reach the first patients. Thus we are witnessing the birth of a very exciting time in the development of molecular medicine, which will be based on the methods of RNA technologies. This volume is the first of a series. It covers various aspects of RNA interference and microRNAs, although antisense RNA applications, hammerhead ribozyme structure and function as well as non-coding RNAs are also discussed. The authors are internationally highly respected experts in the field of RNA technologies.
Publisher: Springer Science & Business Media
ISBN: 3642121683
Category : Medical
Languages : en
Pages : 460
Book Description
RNA technologies are the driving forces of modern medicine and biotechnology. They combine the fields of biochemistry, chemistry, molecular biology, cell biology, physics, nanotechnology and bioinformatics. The combination of these topics is set to revolutionize the medicine of tomorrow. After more than 15 years of extensive research in the field of RNA technologies, the first therapeutics are ready to reach the first patients. Thus we are witnessing the birth of a very exciting time in the development of molecular medicine, which will be based on the methods of RNA technologies. This volume is the first of a series. It covers various aspects of RNA interference and microRNAs, although antisense RNA applications, hammerhead ribozyme structure and function as well as non-coding RNAs are also discussed. The authors are internationally highly respected experts in the field of RNA technologies.
Drugs
Author: Rick Ng
Publisher: John Wiley & Sons
ISBN: 1118210700
Category : Science
Languages : en
Pages : 368
Book Description
"Concise and easy to read, the book quickly introduces basic concepts, then moves on to discuss target selection and the drug discovery process for both small and large molecular drugs." —Doody's Reviews, May 2009 "The second edition of a book that offers a user-friendly step-by-step introduction to all the key processes involved in bringing a drug to the market, including the performance of preclinical trials." —Chemistry World, February 2009 The new edition of this best-selling book continues to offer a user-friendly, step-by-step introduction to all the key processes involved in bringing a drug to the market, including the performance of pre-clinical studies, the conduct of human clinical trials, regulatory controls, and even the manufacturing processes for pharmaceutical products. Concise and easy to read, the book quickly introduces basic concepts, then moves on to discuss target selection and the drug discovery process for both small and large molecular drugs. This second edition features many key enhancements, including Key Points, Chapter Summary, and Review Questions in each chapter, Answers to Review Questions provided in a book-end appendix, and one or two carefully selected "mini" case studies in each chapter. Richly illustrated throughout with over ninety figures and tables, this important book also includes helpful listings of current FDA and European guidelines and a special section on regulatory authority and processes in China. It is an indispensable resource for pharmaceutical industry and academic researchers, pharmaceutical managers and executives, healthcare clinicians, policymakers, regulators, and lobbyists with an interest in drug development. It is also an excellent textbook for students in pharmacy, science, and medicine courses.
Publisher: John Wiley & Sons
ISBN: 1118210700
Category : Science
Languages : en
Pages : 368
Book Description
"Concise and easy to read, the book quickly introduces basic concepts, then moves on to discuss target selection and the drug discovery process for both small and large molecular drugs." —Doody's Reviews, May 2009 "The second edition of a book that offers a user-friendly step-by-step introduction to all the key processes involved in bringing a drug to the market, including the performance of preclinical trials." —Chemistry World, February 2009 The new edition of this best-selling book continues to offer a user-friendly, step-by-step introduction to all the key processes involved in bringing a drug to the market, including the performance of pre-clinical studies, the conduct of human clinical trials, regulatory controls, and even the manufacturing processes for pharmaceutical products. Concise and easy to read, the book quickly introduces basic concepts, then moves on to discuss target selection and the drug discovery process for both small and large molecular drugs. This second edition features many key enhancements, including Key Points, Chapter Summary, and Review Questions in each chapter, Answers to Review Questions provided in a book-end appendix, and one or two carefully selected "mini" case studies in each chapter. Richly illustrated throughout with over ninety figures and tables, this important book also includes helpful listings of current FDA and European guidelines and a special section on regulatory authority and processes in China. It is an indispensable resource for pharmaceutical industry and academic researchers, pharmaceutical managers and executives, healthcare clinicians, policymakers, regulators, and lobbyists with an interest in drug development. It is also an excellent textbook for students in pharmacy, science, and medicine courses.
Peptide-based Drug Discovery
Author: Ved Srivastava
Publisher: Royal Society of Chemistry
ISBN: 1782627324
Category : Medical
Languages : en
Pages : 589
Book Description
With potentially high specificity and low toxicity, biologicals offer promising alternatives to small-molecule drugs. Peptide therapeutics have again become the focus of innovative drug development efforts backed up by a resurgence of venture funds and small biotechnology companies. What does it take to develop a peptide-based medicine? What are the key challenges and how are they overcome? What are emerging therapeutics for peptide modalities? This book answers these questions with a holistic story from molecules to medicine, combining the themes of design, synthesis and clinical applications of peptide-based therapeutics and biomarkers. Chapters are written and edited by leaders in the field from industry and academia and they cover the pharmacokinetics of peptide therapeutics, attributes necessary for commercially successful metabolic peptides, medicinal chemistry strategies for the design of peptidase-resistant peptide analogues, disease classes for which peptide therapeutic are most relevant, and regulatory issues and guidelines. The critical themes covered provide essential background information on what it takes to develop peptide-based medicine from a chemistry perspective and views on the future of peptide drugs. This book will be a valuable resource not only as a reference book for the researcher engaged in academic and pharmaceutical setting, from basic research to manufacturing and from organic chemistry to biotechnology, but also a valuable resource to graduate students to understand discovery and development process for peptide-based medicine.
Publisher: Royal Society of Chemistry
ISBN: 1782627324
Category : Medical
Languages : en
Pages : 589
Book Description
With potentially high specificity and low toxicity, biologicals offer promising alternatives to small-molecule drugs. Peptide therapeutics have again become the focus of innovative drug development efforts backed up by a resurgence of venture funds and small biotechnology companies. What does it take to develop a peptide-based medicine? What are the key challenges and how are they overcome? What are emerging therapeutics for peptide modalities? This book answers these questions with a holistic story from molecules to medicine, combining the themes of design, synthesis and clinical applications of peptide-based therapeutics and biomarkers. Chapters are written and edited by leaders in the field from industry and academia and they cover the pharmacokinetics of peptide therapeutics, attributes necessary for commercially successful metabolic peptides, medicinal chemistry strategies for the design of peptidase-resistant peptide analogues, disease classes for which peptide therapeutic are most relevant, and regulatory issues and guidelines. The critical themes covered provide essential background information on what it takes to develop peptide-based medicine from a chemistry perspective and views on the future of peptide drugs. This book will be a valuable resource not only as a reference book for the researcher engaged in academic and pharmaceutical setting, from basic research to manufacturing and from organic chemistry to biotechnology, but also a valuable resource to graduate students to understand discovery and development process for peptide-based medicine.