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Muscular Dystrophy Therapeutics
This detailed book presents a comprehensive collection of state-of-the-art protocols on muscular dystrophy therapeutics, covering therapeutics using antisense oligonucleotides, gene replacement, genome editing, small molecules, stem cells, and antibodies. Written by leaders in the field, the volume explores techniques that are currently in use and are starting an exciting therapeutic revolution in muscular dystrophy. As a part of the highly successful Methods in Molecular Biology series, chapters include introductions to their respective topics, lists of the necessary materials and reagents, step-by-step and readily reproducible laboratory protocols, as well as tips on troubleshooting and avoiding known pitfalls. Authoritative and practical, Muscular Dystrophy Therapeutics: Methods and Protocols serves as an ideal resource to inspire readers and provide tips, strategies, and advice to develop new therapeutic technologies for this group of diseases.
Muscle Gene Therapy
Muscle disease represents an important health threat to the general population. There is essentially no cure. Gene therapy holds great promise to correct the genetic defects and eventually achieve full recovery in these diseases. Significant progresses have been made in the field of muscle gene therapy over the last few years. The development of novel gene delivery vectors has substantially enhanced specificity and efficiency of muscle gene delivery. The new knowledge on the immune response to viral vectors has added new insight in overcoming the immune obstacles. Most importantly, the field has finally moved from small experimental animal models to human patients. This book will bring together the leaders in the field of muscle gene transfer to provide an updated overview on the progress of muscle gene therapy. It will also highlight important clinical applications of muscle gene therapy.
All Our Families
A provocation to reclaim our disability lineage in order to profoundly reimagine the possibilities for our relationship to disability, kinship, and carework Disability is often described as a tragedy, a crisis, or an aberration, though 1 in 5 people worldwide have a disability. Why is this common human experience rendered exceptional? In All Our Families, disability studies scholar Jennifer Natalya Fink argues that this originates in our families. When we cut a disabled member out of the family story, disability remains a trauma as opposed to a shared and ordinary experience. This makes disability and its diagnosis traumatic and exceptional. Weaving together stories of members of her own fam...
Parvoviruses
This comprehensive reference work brings together for the first time information on every aspect of the parvoviruses in a single volume. It presents the new system of parvovirus classification, as agreed by the International Committee for the Taxonomy of Viruses (ICTV), and includes cutting edge information on the virology, molecular and cellular biology, immunology and clinical manifestations of infection with each known and proposed member of the family Parvoviridae. The book also describes the rapidly expanding basic and clinical science which underpins gene therapy applications using helper-dependent and helper-independent parvoviruses. The respected editorial group has drawn together renowned contributors from around the world to produce what will undoubtedly become the standard reference on the subject. It is essential reading for clinical or research virologists and microbiologists, infectious disease specialists, and public health specialists and laboratories.
Viral Vectors for Gene Therapy
Viral Vectors for Gene Therapy: Methods and Protocols consists of 30 ch- ters detailing the use of herpes viruses, adenoviruses, adeno-associated viruses, simple and complex retroviruses, including lentiviruses, and other virus systems for vector development and gene transfer. Chapter cont- butions provide perspective in the use of viral vectors for applications in the brain and in the central nervous system. Viral Vectors for Gene Therapy: Methods and Protocols contains step-by-step methods for successful rep- cation of experimental procedures, and should prove useful for both experienced investigators and newcomers in the field, including those beginning graduate study or undergoing postdo...
Gene Therapy in Lung Disease
Presents up-to-date summaries of recently completed and ongoing clinical trials. With writings from more than 35 internationally renowned experts, Gene Therapy in Lung Disease unlocks the biological mysteries of infection immunity cytokine behavior fibrosis and illustrates the use of gene the
Cardiac Cell and Gene Transfer
Heart disease is the leading cause of death in developed countries. Recent experimental advances featuring cellular, molecular, and genetic tools and technologies offer the potential for new therapeutic strategies directed toward remediation of inherited and acquired heart diseases. Whether these recent basic science advances will ultimately translate to clinical efficacy for patients with heart disease is unknown and is important to ascertain. Cardiac Cell and Gene Transfer: Principles, Protocols, and Applications is designed to provide the reader with up-to-date coverage of a myriad of specific methodo- gies and protocols for gene and cell transfer to the myocardium. Each chapter features ...
Gene Therapy Methods
This volume in the prestigious Methods in Enzymology series discusses methods currently used in preclinical and clinical gene therapy. Subjects covered in this book, such as the use of adeno-associated virus delivery for treatment of Parkinson's disease, are topical and are presented in the methods-oriented style popularized by this series. Discusses methods currently used in preclinical and clinical gene therapy Covers the use of adeno-associated virus delivery for treatment of Parkinson's disease
Retinal Degenerative Diseases
This is the proceedings of the XIIIth International Symposium on Retinal Degenerations, which will be held in Emeishan, Sichuan, China on September 18 - 23, 2008. The themes will include "Molecular and genetic mechanisms in photoreceptor degeneration", "Age-related macular degeneration", "New diagnostic techniques for retinal degenerations". "Neuroprotection in the prevention of retinal degeneration", "Gene therapy and the correction of gene defects", as well as other emerging topics that may develop over the next few months.
Approaches to Blocking the Immune Response to Gene Transfer with Viral Vectors
Viral vectors are superior tools for gene therapy and as a genetic vaccine platform because viruses have evolved to efficiently infect and transfer their genomes to cells. Several impressive successes in viral vector-based gene therapies have been reported in humans, including restoration of vision in patients with Leber’s congenital amaurosis by retinal gene transfer and cures for severe immune deficiencies by gene transfer to hematopoietic stem cells. However, the mammalian immune system has evolved in parallel to fend off invading pathogens such as viruses. Innate and antigen-specific adaptive immune responses against viral vectors and therapeutic transgene products pose serious hurdles...
