Genterapi

Polymers and Nanomaterials for Gene Therapy provides the latest information on gene therapy, a topic that has attracted significant attention over the past two decades for the treatment of inherited and acquired genetic diseases. Major research efforts are currently focused on designing suitable carrier vectors that compact and protect oligonucleotides for gene therapy.

The book explores the most recent developments in the field of polymer science and nanotechnology, and how these advancements have helped in the design of advanced materials. Non-viral vector systems, including cationic lipids, polymers, dendrimers, peptides and nanoparticles, are potential routes for compacting DNA for systemic delivery. However, unlike viral analogues that have no difficulty in overcoming cellular barriers and immune defense mechanisms, non-viral gene carriers consistently exhibit significant reduced transfection efficiency due to numerous extra- and intracellular obstacles. Therefore, biocompatibility and potential for large-scale production make these compounds increasingly attractive for gene therapy.

This book contains chapters on the engineering of polymers and nanomaterials for gene therapy, and how they can form complexes with DNA and avoid both in vitro and in vivo barriers. Other chapters describe in vitro, ex vivo, in vivo gene therapy studies, and the current issues affecting non-viral gene therapy.

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 for Viral Infections provides a comprehensive review of the broader field of nucleic acid and its use in treating viral infections. The text bridges the gap between basic science and important clinical applications of the technology, providing a systematic, integrated review of the advances in nucleic acid-based antiviral drugs and the potential advantages of new technologies over current treatment options.

Coverage begins with the fundamentals, exploring varying topics, including harnessing RNAi to silence viral gene expression, antiviral gene editing, viral gene therapy vectors, and non-viral vectors.

Subsequent sections include detailed coverage of the developing use of gene therapy for the treatment of specific infections, the principles of rational design of antivirals, and the hurdles that currently face the further advancement of gene therapy technology.

**Selected for 2026 Doody's Core Titles as an Essential Purchase in Clinical Genetics**For decades, Emery and Rimoin’s Principles and Practice of Medical Genetics and Genomics has served as the ultimate resource for clinicians integrating genetics and genomics into medical practice. With detailed coverage in contributions from more than 250 of the world’s most trusted authorities in medical genetics and a series of 11 volumes available for individual sale, the seventh edition of this classic reference includes the latest information on seminal topics such as prenatal diagnosis, genome sequencing, public health genetics, and genetic counseling. Volumes 3-11 cover diagnosis, management, and treatment of both rare and common disorders in all organ systems. Students, medical providers, and researchers all benefit from the latest information about the role of the genome in health and disease. This comprehensive yet practical resource emphasizes theory and research fundamentals across the full spectrum of applications to medicine.In this volume, leading physicians and researchers thoroughly examine medical genetics and genomics as applied to endocrine, skin, connective tissue and skeletal disorders, with emphasis on understanding the genetic mechanisms underlying these conditions, diagnostic approaches, and treatment methods that make use of current genomic technologies and translational studies.With regular advances in genomic technologies propelling precision medicine into the clinic, Emery and Rimoin’s Principles and Practice of Medical Genetics and Genomics, seventh edition, bridges the gap between high-level molecular genetics and genomics and practical application and serves as an invaluable clinical tool for the health professionals and researchers.

How tiny variations in our personal DNA can determine how we look, how we behave, how we get sick, and how we get well.

News stories report almost daily on the remarkable progress scientists are making in unraveling the genetic basis of disease and behavior. Meanwhile, new technologies are rapidly reducing the cost of reading someone''s personal DNA (all six billion letters of it). Within the next ten years, hospitals may present parents with their newborn''s complete DNA code along with her footprints and APGAR score. In Genetic Twists of Fate, distinguished geneticists Stanley Fields and Mark Johnston help us make sense of the genetic revolution that is upon us.

Fields and Johnston tell real life stories that hinge on the inheritance of one tiny change rather than another in an individual''s DNA: a mother wrongly accused of poisoning her young son when the true killer was a genetic disorder; the screen siren who could no longer remember her lines because of Alzheimer''s disease; and the president who was treated with rat poison to prevent another heart attack. In an engaging and accessible style, Fields and Johnston explain what our personal DNA code is, how a few differences in its long list of DNA letters makes each of us unique, and how that code influences our appearance, our behavior, and our risk for such common diseases as diabetes or cancer.