Correction: Enzyme replacement and enhancement therapies: lessons from lysosomal disorders
نویسندگان
چکیده
منابع مشابه
strategies 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 کاملEnzyme replacement therapy for lysosomal diseases: lessons from 20 years of experience and remaining challenges.
In 1964, Christian de Duve first suggested that enzyme replacement might prove therapeutic for lysosomal storage diseases (LSDs). Early efforts identified the major obstacles, including the inability to produce large quantities of the normal enzymes, the lack of animal models for proof-of-concept studies, and the potentially harmful immune responses to the "foreign" normal enzymes. Subsequently...
متن کاملEnzyme replacement therapy for lysosomal storage diseases.
Enzyme replacement therapy (ERT) has been approved for 6 lysosomal storage diseases (LSDs) worldwide including Japan. These diseases include Gaucher disease (GD), Fabry disease, mucopolysaccharidosis (MPS) types I, II, and VI, and Pompe disease (PD). The efficacy and safety of ERT for LSDs has been confirmed by extensive clinical trials. However, there are still obstacles to successful ERT, suc...
متن کاملRTB Lectin: a novel receptor-independent delivery system for lysosomal enzyme replacement therapies
Enzyme replacement therapies have revolutionized patient treatment for multiple rare lysosomal storage diseases but show limited effectiveness for addressing pathologies in "hard-to-treat" organs and tissues including brain and bone. Here we investigate the plant lectin RTB as a novel carrier for human lysosomal enzymes. RTB enters mammalian cells by multiple mechanisms including both adsorptiv...
متن کاملSpecific Treatment for Lysosomal Storage Disorders: Enzyme Replacement Therapy, Bone Marrow Transplant and Others
Received December 15, 2003 Abstract In this article, we review specific therapies that tackle the basic biochemical defects of lysosomal storage diseases. These include bone marrow transplantation, substrate deprivation therapy, enzyme replacement therapy and enzyme enhancement therapy. We particularly update the progress of development of enzyme replacement therapy, which plays a major role in...
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ژورنال
عنوان ژورنال: Nature Reviews Genetics
سال: 2003
ISSN: 1471-0056,1471-0064
DOI: 10.1038/nrg1016