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Author: Saghir Akhtar Publisher: CRC Press ISBN: 1351366130 Category : Medical Languages : en Pages : 393
Book Description
With contributions from leading experts, this book is the first to focus solely on addressing the problems and reviewing the strategies currently being used to improve the delivery of antisense nucleic acids. Important delivery issues, such as improving biological stability, improving cell-specific targeting and cellular uptake, manipulating subcellular distribution and producing liposomal delivery systems for antisense agents are comprehensively covered in this volume. This book links review-type articles with contributions that contain exciting never-before-published data on the cellular delivery of oligonucleotides. It stimulates reading for both established researchers and newcomers to the antisense field.
Author: Saghir Akhtar Publisher: CRC Press ISBN: 1351366130 Category : Medical Languages : en Pages : 393
Book Description
With contributions from leading experts, this book is the first to focus solely on addressing the problems and reviewing the strategies currently being used to improve the delivery of antisense nucleic acids. Important delivery issues, such as improving biological stability, improving cell-specific targeting and cellular uptake, manipulating subcellular distribution and producing liposomal delivery systems for antisense agents are comprehensively covered in this volume. This book links review-type articles with contributions that contain exciting never-before-published data on the cellular delivery of oligonucleotides. It stimulates reading for both established researchers and newcomers to the antisense field.
Author: Saghir Akhtar Publisher: CRC Press ISBN: 9781138558571 Category : Languages : en Pages : 318
Book Description
With contributions from leading experts, this book is the first to focus solely on addressing the problems and reviewing the strategies currently being used to improve the delivery of antisense nucleic acids. Important delivery issues, such as improving biological stability, improving cell-specific targeting and cellular uptake, manipulating subcellular distribution and producing liposomal delivery systems for antisense agents are comprehensively covered in this volume. This book links review-type articles with contributions that contain exciting never-before-published data on the cellular delivery of oligonucleotides. It stimulates reading for both established researchers and newcomers to the antisense field.
Author: Nicolay Ferrari Publisher: John Wiley & Sons ISBN: 1119070309 Category : Medical Languages : en Pages : 563
Book Description
A comprehensive review of contemporary antisense oligonucleotides drugs and therapeutic principles, methods, applications, and research Oligonucleotide-based drugs, in particular antisense oligonucleotides, are part of a growing number of pharmaceutical and biotech programs progressing to treat a wide range of indications including cancer, cardiovascular, neurodegenerative, neuromuscular, and respiratory diseases, as well as other severe and rare diseases. Reviewing fundamentals and offering guidelines for drug discovery and development, this book is a practical guide covering all key aspects of this increasingly popular area of pharmacology and biotech and pharma research, from the basic science behind antisense oligonucleotides chemistry, toxicology, manufacturing, to safety assessments, the design of therapeutic protocols, to clinical experience. Antisense oligonucleotides are single strands of DNA or RNA that are complementary to a chosen sequence. While the idea of antisense oligonucleotides to target single genes dates back to the 1970's, most advances have taken place in recent years. The increasing number of antisense oligonucleotide programs in clinical development is a testament to the progress and understanding of pharmacologic, pharmacokinetic, and toxicologic properties as well as improvement in the delivery of oligonucleotides. This valuable book reviews the fundamentals of oligonucleotides, with a focus on antisense oligonucleotide drugs, and reports on the latest research underway worldwide. • Helps readers understand antisense molecules and their targets, biochemistry, and toxicity mechanisms, roles in disease, and applications for safety and therapeutics • Examines the principles, practices, and tools for scientists in both pre-clinical and clinical settings and how to apply them to antisense oligonucleotides • Provides guidelines for scientists in drug design and discovery to help improve efficiency, assessment, and the success of drug candidates • Includes interdisciplinary perspectives, from academia, industry, regulatory and from the fields of pharmacology, toxicology, biology, and medicinal chemistry Oligonucleotide-Based Drugs and Therapeutics belongs on the reference shelves of chemists, pharmaceutical scientists, chemical biologists, toxicologists and other scientists working in the pharmaceutical and biotechnology industries. It will also be a valuable resource for regulatory specialists and safety assessment professionals and an important reference for academic researchers and post-graduates interested in therapeutics, antisense therapy, and oligonucleotides.
Author: Stanley T. Crooke Publisher: CRC Press ISBN: 1439832501 Category : Medical Languages : en Pages : 848
Book Description
Extensively revised and updated, Antisense Drug Technology: Principles, Strategies, and Applications, Second Edition reflects the logarithmic progress made in the past four years of oligonucleotide-based therapies, and, in particular, antisense therapeutics and research. Interpreting lessons learned from the clinical trials of first generati
Author: Cy A. Stein Publisher: Wiley-Liss ISBN: Category : Medical Languages : en Pages : 556
Book Description
Applied Antisense Oligonucleotide Technology provides the basic concepts as well as the practical concerns associated with the use of antisense oligonucleotides to modify gene expression. Chapters include oligonucleotide chemistry, DNA triplex formation, delivery mechanisms, pharmocokinetics, toxicity, oligonucleotides.
Author: Amy Elizabeth Arnold Publisher: ISBN: Category : Languages : en Pages :
Book Description
Antisense therapeutics, including antisense oligonucleotides (AONs) and small interfering ribonucleic acids (siRNAs), are powerful tools for regulating genes, making them a promising therapy for diseases such as cancer where oncogenic genes are over-expressed. The delivery of antisense therapeutics to target cells presents a significant challenge due to the many barriers a nucleic acid must face in order to reach the cytoplasm where it exerts its effects. In this thesis, I explored multiple strategies for delivery of AONs and siRNAs, focusing on targeting the desired cell population, inducing endocytosis, and facilitating endosomal escape. This was done within the context of glioblastoma (GBM), and specifically the glioblastoma stem cells (GSCs), an aggressive subpopulation of GBM cells that are involved in resistance, migration, and recurrence. Antisense oligonucleotides against a relevant GBM gene were conjugated to an antibody engineered to target CD44, a cell surface receptor which is highly expressed on GSCs. Using this system, we demonstrated functional targeting, endocytosis, and gene knockdown in the GSCs, leading to a morphological change in the cells. This represented the first time an antibody-oligonucleotide conjugate was used to target the GSC population. We were challenged with a lack of endosomal escape when using the antibody delivery platform, so we next looked at using a protein with a native endosomal escape mechanism to facilitate oligonucleotide delivery. For the second strategy, I conjugated attenuated diphtheria toxin (aDT), a protein which escapes the endolysosomal pathway, to siRNAs against relevant gene targets involved in GSC proliferation and invasion. Using this aDT-siRNA conjugate, we could downregulate genes of interest in the glioblastoma stem cells, leading to significant changes in cell viability and the invasive capacity of these cells. This is the first diphtheria toxin-based siRNA delivery vehicle and represents a platform technology for siRNA- and AON-based therapies.
Author: Patel Jayvadan Publisher: LAP Lambert Academic Publishing ISBN: 9783659201158 Category : Languages : en Pages : 100
Book Description
Over the past 2 decades, the antisense oligonucleotide technology has emerged as a valid approach to selectively modulate gene expression. By adhering to a strict set of specific rules, ongoing in vitro studies using antisense oligonucleotides permit the characterization of new targets and new potential therapeutic compounds. The number of in vitro experiments has increased continuously, and this has led to numerous therapeutic trials, a few of which now appear preliminarily to be positive. However, the optimal use of antisense oligonucleotides in the treatment of disease requires the resolution of problems relating to effective design, enhanced biological activity, and efficient target delivery. These issues are currently being actively addressed and will hopefully continue to shed light on ways to increase therapeutic efficacy and specificity.
Author: Saghir Akhtar
Author: Saghir Akhtar
Author: Saghir Akhtar
Author: Gregory Gregoriadis
Author: Nicolay Ferrari
Author: Jens Kurreck
Author: Stanley T. Crooke
Author: Cy A. Stein
Author: Amy Elizabeth Arnold
Author: Patel Jayvadan
Author: Felicia Codrea