The knowledge of lysosomal biology and the consequences of its
dysfunction have increased dramatically in the past 60 years.
Research of these disorders has moved from diseases with unknown
etiology to disorders with clear and defined pathophysiology and
some of them have benefited from the development of disease
specific therapeutics. Lysosomal Biology and Storage Disorders
describes the nature of the diseases, the historical evolution of
the field and future perspectives for the treatment of these
clinical entities. Organized as a textbook, Lysosomal Biology and
Storage Disorders describes the nature of lysosomal dysfunction,
the synthesis and targeting of lysosomal enzymes and the
implications of the targeting mechanisms for the development of new
therapies. Disease specific chapters provide thorough reviews of
the clinical features of lysosomal storage disorders, their
molecular basis and the commercial or experimental therapeutic
approaches sought in this area.
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