Recent advances in gene therapy for lysosomal storage disorders
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چکیده
منابع مشابه
Recent advances in gene therapy for lysosomal storage disorders
Lysosomal storage disorders (LSDs) are a group of genetic diseases that result in metabolic derangements of the lysosome. Most LSDs are due to the genetic absence of a single catabolic enzyme, causing accumulation of the enzyme's substrate within the lysosome. Over time, tissue-specific substrate accumulations result in a spectrum of symptoms and disabilities that vary by LSD. LSDs are promisin...
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Significant advances in therapy for lysosomal storage disorders have occurred with an accelerating pace over the past decade. Although enzyme replacement therapy has improved the outcome of lysosomal storage disorders, antibody responses have occurred and sometimes prevented efficacy, especially in cross-reacting immune material negative patients with Pompe disease. Preclinical gene therapy exp...
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ژورنال
عنوان ژورنال: The Application of Clinical Genetics
سال: 2015
ISSN: 1178-704X
DOI: 10.2147/tacg.s57682