Gene therapy for the neurological manifestations in lysosomal storage disorders
نویسندگان
چکیده
منابع مشابه
Immunomodulatory gene therapy in lysosomal storage disorders.
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...
متن کاملRecent 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...
متن کاملCritical Review Therapy for Lysosomal Storage Disorders
In the last years, much progress has been achieved in the field of lysosomal storage disorders. In the past, no specific treatment was available for the affected patients; management mainly consisted of supportive care and treatment of complications. As orphan drug regulations, however, encouraged development of drugs for these disorders by granting marketing exclusivity for 10 years and other ...
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 کاملStem Cell Therapy in Pediatric Neurological Disorders
Pediatric neurological disorders including muscular dystrophy, cerebral palsy, and spinal cord injury are defined as a heterogenous group of diseases, of which some are known to be genetic. The two significant features represented for stem cells, leading to distinguish them from other cell types are addressed as below: they can renew themselves besides the ability to differentiate into cells wi...
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ژورنال
عنوان ژورنال: Journal of Lipid Research
سال: 2014
ISSN: 0022-2275
DOI: 10.1194/jlr.r047175