Lysosomal Disorders of the Brain: Recent Advances in Molecular and Cellular Pathogenesis and Treatmemt

Book Info
Univ. of Oxford, UK. Details recent advances in the molecular and cellular pathologies of a variety of lysosomal storage diseases. Also discusses the development of effective therapies. Provides an overview of each storage disease and describes the molecular mechanisms of storage. For clinicians.


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Lysosomal storage diseases are inherited metabolic disorders characterized by severe pathology, typically involving the brain. Although individually rare, they collectively represent a significant group of diseases that primarily present in early infancy or childhood. In recent years considerable progress has been made in understanding the molecular mechanisms that lead to storage. Unravelling the basis for these diseases is providing unique insight into the normal biology of cells and pointing the way to the development of therapeutic strategies for their treatment. Lysosomal disorders of the brain details recent advances in the molecular and cellular pathologies of these diseases and in the development of effective therapies. After an overview of these diseases, the book describes the molecular mechanisms of storage, model systems and pathophysiological mechanisms, and finally, advice on treatment. With each chapter written by leading experts in their field, this book will be valuable for clinicians and scientists in helping them understand the mechanisms underlying these disorders, how they can be diagnosed, and what treatment options are currently available.


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