Advances in Nanomedicine for the Delivery of Therapeutic Nucleic Acids addresses several issues related to safe and effective delivery of nucleic acids (NAs) using nanoparticles. A further emphasis would be laid on the mechanism of delivery of NAs, the barriers encountered and the strategies adapted to combat them. An exhaustive account of the advantages as well shortcomings of all the delivery vectors being employed in delivery of various NAs will be provided. On final note the regulatory aspects of nanoparticles mediated NA would be discussed, with focus on their clinical relevance. The design and development of nucleic acid-based therapeutics for the treatment of diseases arising from genetic abnormalities has made significant progress over the past few years. NAs have been widely explored for the treatment of cancer and infectious diseases or to block cell proliferation and thereby caused diseases. Advances in synthetic oligonucleotide chemistry resulted in synthesis of NAs that are relatively stable in in vivo environments. However, cellular targeting and intracellular delivery of NAs still remains a challenge. Further development of NA-based therapeutics depends on the progress of safe and effective carriers for systemic administration. Nanomedicine has facilitated availability of vectors with diminished cytotoxicity and enhanced efficacy which are rapidly emerging as systems of choice. These vectors protect NAs from enzymatic degradation by forming condensed complexes along with targeted tissue and cellular delivery. During the past few years, a myriad reports have appeared reporting delivery of NAs mediated by nanoparticles. This book will provide an overview of nanoparticles being employed in the in vitro and in vivo delivery of therapeutically relevant NAs like DNA, siRNA, LNA, PNA, etc.

Gene therapy is emerging as a new class of therapeutics for the treatment of inherited and acquired diseases. However, poor cellular uptake and instability of DNA in the physiological milieu limits its therapeutic potential, hence a vector which can protect and efficiently transport DNA to the target cells must be developed. Nanotechnology-based non-viral vectors have been proposed as potential candidates. Various polymeric nanoparticles have been shown to be suitable, with high cellular uptake efficiencies and reduced cytotoxicity. These delivery vectors form condensed complexes with DNA which result in shielding against enzymatic degradation and enhanced cellular targeting. Advantages including easy manipulatibility, high stability, low cost and high payload, mean that nanoparticles from various polymers have been exploited. Gene therapy gives a systematic account of the many aspects of nanotechnology mediated gene therapy, from the preparation of nanoparticles to physicochemical characterization, and follows with applications in in vitro and in vivo models. This book emphasizes the various aspects of nanotechnology-based gene therapy, with initial chapters detailing the tools and techniques available for preparation and in vitro and in vivo characterization of nanoparticles. Later chapters provide exhaustive details on polymeric systems employed for gene therapy.
Provides an overview of nanotechnology applications in gene therapy, from preparation of nanoparticles to in vitro and in vivo studiesDetails the tools and techniques available for preparation, characterization and in vitro and in vivo study of nanoparticlesDetails the limitations of nanoparticle-mediated gene therapy and proposes ways in which they may be overcome

The Design and Development of Novel Drugs and Vaccines: Principles and Protocols presents both in silico methods and experimental protocols for vaccine and drug design and development, critically reviewing the most current research and emphasizing approaches and technologies that accelerate and lower the cost of product development. Sections review the technologies and approaches used to identify, characterize and establish a protein as a new drug and vaccine target, cover several molecular methods for in vitro studies of the desired target, and present various physiological parameters for in vivo studies. The book includes preclinical trials and research, along with information on FDA approval.

Gene silencing mediated through short interfering RNAs (siRNAs) is rapidly emerging as a new class of therapeutics for the treatment of inherited and acquired diseases. However, poor cellular uptake and instability in physiological conditions limits its therapeutic potential, hence there arises a need to develop a delivery system which can protect and efficiently transport siRNA to the target cells. Nanotechnology based non-viral vectors have been proposed as a potential alternate. Various polymeric nanoparticles have been shown as suitable delivery candidates with high cellular uptake efficiencies and advantage of reduced cytotoxicity. These delivery vectors form condensed complexes with siRNA which in turn provides protection to siRNA from enzymatic degradation and further leads to enhanced tissue and cellular targeting. Nanoparticles prepared from various polymers like polyethylenimine, chitosan have been largely exploited, as they bear several advantages such as, easy manipulatibility, high stability, low cost and high payload. Though numerous publications have appeared in the literature, there was still need to get more information under one roof, as book compilation which could provide a systematic account of all the aspects of nanotechnology mediated gene silencing right from the preparation of nanoparticles to in vitro and in vivo characterization followed by various applications in vitro and in vivo models. This book focuses on various aspects of nanotechnology based gene silencing. The initial chapters detail the techniques available for in vitro and in vivo characterization of nanoparticles. In the later chapters, exhaustive details about various polymeric systems employed for gene silencing has been accounted. The important topics discussed in the book include:- * Introduction to Nanotechnology * Physicochemical? Characterization of Nanoparticles * In Vitro and In Vivo Characterization of Nanoparticles * Concept and Barriers to Gene Silencing * Nanoparticles Mediated Targeted siRNA delivery * Polymers for siRNA Delivery * Chitosan * Polyethylenimine * Poly-L-lysine * Atelocollagen * Protamine * Dendrimers * Cyclodextrin * Poly (D, L-lactide-co-glycolide) * Nanomaterials for siRNA delivery; Keyword Index