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Gene therapy offers many conceptual advantages to treat muscle
diseases, especially various forms of muscular dystrophies;
however, it faces a number of unique challenges, including the need
to deliver a therapeutic vector to all muscles throughout the body.
In Muscle Gene Therapy: Methods and Protocols, expert researchers
in the field present a collection of techniques aimed at bridging
the translational gap in muscle gene therapy between the prevalent
rodent models and vitally important larger animal models. Divided
into three sections, this volume examines basic protocols for
optimizing the muscle gene expression cassette and for evaluating
the therapeutic outcomes, new developments in muscle gene therapy
technology such as adeno-associated viral vector (AAV),
oligonucleotide-mediated exon-skipping, and novel RNA-based
strategies, and step-by-step guidance on muscle gene delivery in
swine, ovine, canine, and non-human primates. Written in the highly
successful Methods in Molecular Biology (TM) series format,
chapters include introductions to their respective topics, lists of
the necessary materials and reagents, detailed, readily
reproducible laboratory protocols, and tips on troubleshooting and
avoiding known pitfalls. Authoritative and cutting-edge, Muscle
Gene Therapy: Methods and Protocols serves as an invaluable
resource for graduate students, post-doctoral fellows, and
principle investigators pursuing the crucial advancement of muscle
disease gene therapy in the hope of someday curing these
debilitating disorders.
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.
Gene therapy offers many conceptual advantages to treat muscle
diseases, especially various forms of muscular dystrophies;
however, it faces a number of unique challenges, including the need
to deliver a therapeutic vector to all muscles throughout the body.
In Muscle Gene Therapy: Methods and Protocols, expert researchers
in the field present a collection of techniques aimed at bridging
the translational gap in muscle gene therapy between the prevalent
rodent models and vitally important larger animal models. Divided
into three sections, this volume examines basic protocols for
optimizing the muscle gene expression cassette and for evaluating
the therapeutic outcomes, new developments in muscle gene therapy
technology such as adeno-associated viral vector (AAV),
oligonucleotide-mediated exon-skipping, and novel RNA-based
strategies, and step-by-step guidance on muscle gene delivery in
swine, ovine, canine, and non-human primates. Written in the highly
successful Methods in Molecular Biology(TM) series format, chapters
include introductions to their respective topics, lists of the
necessary materials and reagents, detailed, readily reproducible
laboratory protocols, and tips on troubleshooting and avoiding
known pitfalls. Authoritative and cutting-edge, Muscle Gene
Therapy: Methods and Protocols serves as an invaluable resource for
graduate students, post-doctoral fellows, and principle
investigators pursuing the crucial advancement of muscle disease
gene therapy in the hope of someday curing these debilitating
disorders.
About 7 million people worldwide are suffering from various
inherited neuromuscular diseases. Gene therapy brings the hope of
treating these diseases at their genetic roots. Muscle Gene Therapy
is the only book dedicated to this topic. The first edition was
published in 2010 when the field was just about to enter its prime
time. The progress made since then has been unprecedented. The
number of diseases that have been targeted by gene therapy has
increased tremendously. The gene therapy toolbox is expanded
greatly with many creative novel strategies (such as genome editing
and therapy with disease-modifying genes). Most importantly,
clinical benefits have begun to emerge in human patients. To
reflect rapid advances in the field, we have compiled the second
edition of Muscle Gene Therapy with contributions from experts that
have conducted gene therapy studies either in animal models and/or
in human patients. The new edition offers a much needed, up-to-date
overview and perspective on the foundation and current status of
neuromuscular disease gene therapy. It provides a framework to the
development and regulatory approval of muscle gene therapy drugs in
the upcoming years. This book is a must-have for anyone who is
interested in neuromuscular disease gene therapy including those in
the research arena (established investigators and trainees in the
fields of clinical practice, veterinary medicine and basic
biomedical sciences), funding and regulatory agencies, and patient
community.
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