Treatment strategies for lysosomal storage disorders
نویسندگان
چکیده
منابع مشابه
Treatments for lysosomal storage disorders.
There are over 70 human diseases that are caused by defects in various aspects of lysosomal function. Until 20 years ago, the only specific therapy available for lysosomal storage disorders was allogeneic haemopoietic stem cell transplantation. Over the last two decades, there has been remarkable progress and there are now licensed treatments for seven of these diseases. In some cases, a choice...
متن کاملstrategies in enzyme replacement therapy of lysosomal storage disorders
lysosomal storage diseases (lsds) result from a genetic defect in synthesis and cellular transport of lysosomal enzyme to the lysosomes. lsds are progressive and may present at any age affecting multiple tissues and organ systems. they comprise a diverse group of over 40 clinically distinct inherited disorders. as a group they occur in approximately 1 in 5000 to 8000 births in the western socie...
متن کاملNew biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders.
This review discusses the multiple bio- and nanotechnological strategies developed in the last few decades for treatment of a group of fatal genetic diseases termed lysosomal storage disorders. Some basic foundation on the biomedical causes and social and clinical relevance of these diseases is provided. Several treatment modalities, from those currently available to novel therapeutic approache...
متن کاملTreatment options for lysosomal storage disorders: developing insights.
INTRODUCTION Lysosomal storage disorders (LSDs) are clinically heterogeneous disorders that result primarily from lysosomal accumulation of macromolecules in various tissues. LSDs are always progressive, and often lead to severe symptoms and premature death. The identification of the underlying genetic and enzymatic defects has prompted the development of various treatment options. AREAS COVE...
متن کاملNew strategies for the treatment of lysosomal storage diseases (review).
The lysosomal storage diseases (LSDs) are a group of inherited metabolic disorders caused by the deficiency of any of the lysosomal functions, in most cases of lysosomal hydrolases. LSDs are typically characterized by storage of a variety of substrates in multiple tissues and organs and by the variable association of unusual clinical manifestation...
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ژورنال
عنوان ژورنال: Developmental Medicine & Child Neurology
سال: 2017
ISSN: 0012-1622
DOI: 10.1111/dmcn.13600