This unique volume in our Drugs and Pharmaceutical Sciences series covers the development of gene therapy today, the technology involved, clinical applications of siRNA, non-viral vector-based mRNA delivery using nanotechnology, and RNA based vaccines for treating the infectious diseases. It also presents the current application of the CRISPR/Cas9 gene-editing technique which has revolutionized genome editing and which was awarded the 2020 Nobel Prize in Chemistry. Several new drug delivery systems are explored for the applications of gene therapy. These are found to be useful in treating chronic illnesses, including cancer and infectious diseases. Key Features: Overview of the development of gene therapy Provides the most up to date information on the development of gene therapy, from the technology involved to gene correction and genome editing Presents CRISPR gene therapy recent trends and applications Discusses siRNA, mRNA, and DNA plasmids

This volume examines the advantages and limitations of the major gene delivery systems and offers guidelines to select the most appropriate viral or synthetic delivery system for specific therapeutic applications. It discusses advances in the design, optimization, and adaptation of gene delivery systems for the treatment of cancerous, cardiovascula

1 D.V. Schaffer, W. Zhou: Gene Therapy as Future Human Therapeutics.- 2 J. Heidel, S. Mishra, M.E. Davis: Molecular Conjugates.- 3 M. Manthorpe, P. Hobart, G. Hermanson, M. Ferrari, A. Geall, B. Goff, A. Rolland: Plasmid Vaccines and Therapeutics: From Design to Applications.- 4 S.R. Little, R. Langer: Non-Viral Delivery of Cancer Genetic Vaccines.- 5 J.C. Grieger, R.J. Samulski: Adeno-Associated Virus as a Gene Therapy Vector: Vector Development, Production and Clinical Applications.- 6 J.H. Yu, D.V. Schaffer: Advanced Targeting Strategies for Murine Retroviral and Adeno-Associated Viral Vectors.- 7 N. Loewen, E.M. Poeschla: Lentiviral Vectors.- 8 N.E. Altaras, J.G. Aunins, R.K. Evans, A. Kamen, J.O. Konz, J.J. Wolf: Production and Formulation of Adenovirus Vectors.-

Here, front-line researchers in the booming field of nanobiotechnology describe the most promising approaches for bioinspired drug delivery, encompassing small molecule delivery, delivery of therapeutic proteins and gene delivery. The carriers surveyed include polymeric, proteinaceous and lipid systems on the nanoscale, with a focus on their adaptability for different cargoes and target tissues. Thanks to the broad coverage of carriers as well as cargoes discussed, every researcher in the field will find valuable information here.

Gene delivery is a transport of genes of therapeutic values into the chromosomes of the cells or tissues which can be targeted to replace the faulty genes. In last two decades lot of research efforts are dedicated to gene delivery for therapeutic applications. Today gene therapy is promising approach in treatment of genetic diseases including mitochondrial related diseases like blindness, muscular dystrophy, cystic fibrosis, and some cancers. Gene Delivery Systems: Nano Delivery Technologies observes the exploration of nanotechnology for gene therapy and gene delivery. Written by prominent authors in the field, this book covers various aspects of gene delivery including challenges in delivering gene therapy, advances in genome editing, RNA-based gene therapy, Green nanoparticles for oligonucleotide delivery. Additional features include" Provides the most up to date information on the development of gene therapy, from the technology involved to gene correction and genome editing. Includes knowledge of the current application of CRISPR/Cas9 gene-editing technique; an approach that has recently been given the Noble Prize. Examines the development of mRNA vaccines for Covid -19 in challenging pandemic scenario Discusses siRNA, mRNA, and DNA plasmids.

A practical resource for everyone involved in the gene therapy field and in the design of effective gene delivery systems, this volume presents an overview and update of recent advances in the field of non-viral methods for the in vivo transfer of therapeutic genes to biological targets using conventional routes of administration. Methods to contro

To treat disease or correct genetic disorders using gene therapy, the most suitable vehicle must be able to deliver genes to the appropriate tissues and cells in the body in a specific as well as safe and effective manner. While viruses are the most popular vehicles to date, their disadvantages include toxicity, limited size of genes they can carry

Polysaccharide-Based Nanocomposites for Gene Delivery and Tissue Engineering presents quantitative background on new polysaccharide nanocomposites in a clear and logical way, highlighting the most exciting applications in gene delivery and tissue engineering and their progress. The book focuses on the different types of polysaccharide nanocomposites for gene delivery and tissue engineering and covers polysaccharide hydrogels for tissue engineering and polysaccharide magnetic nanocomposites for gene delivery. Chapters cover various nanocomposites presented in twenty-one separate chapters. This book will be of great interest to all those researching the development and applications of polysaccharide-based nanocomposites for modeling. As polysaccharide-based nanocomposites promise cutting-edge applications in gene delivery and tissue engineering, with their development at the forefront of modern medicine, this book is a welcome title on this exciting science. - Presents quantitative background on new polysaccharide nanocomposites for advanced medicine - Focuses on polysaccharide nanocomposites in relation to gene delivery and tissue engineering - Highlights the most exciting, leading-edge applications in gene delivery and tissue engineering - Covers polysaccharide hydrogels for tissue engineering and magnetic nanocomposites for gene delivery - Offers a logical and useful presentation of polysaccharide nanocomposites organized first by application and then by nanocomposite

In the quest for innovative drug delivery systems attempting to meet the unmet needs in pharmaceutical space, research has taken a much more complicated path that poses a significant challenge for translation. Despite the progress made with novel materials, polyesters still remain at the helm of drug delivery technologies. This book provides a single source of reference of polyester drug delivery systems that covers a broad spectrum of materials design, manufacturing techniques, and applications.

The concept of smart drug delivery vehicles involves designing and preparing a nanostructure (or microstructure) that can be loaded with a cargo, this can be a therapeutic drug, a contrast agent for imaging, or a nucleic acid for gene therapy. The nanocarrier serves to protect the cargo from degradation by enzymes in the body, to enhance the solubility of insoluble drugs, to extend the circulation half-life, and to enhance its penetration and accumulation at the target site. Importantly, smart nanocarriers can be designed to be responsive to a specific stimulus, so that the cargo is only released or activated when desired. In this volume we cover smart nanocarriers that respond to externally applied stimuli that usually involve application of physical energy. This physical energy can be applied from outside the body and can either cause cargo release, or can activate the nanostructure to be cytotoxic, or both. The stimuli covered include light of various wavelengths (ultraviolet, visible or infrared), temperature (increased or decreased), magnetic fields (used to externally manipulate nanostructures and to activate them), ultrasound, and electrical and mechanical forces. Finally we discuss the issue of nanotoxicology and the future scope of the field.