AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease)
نویسندگان
چکیده
منابع مشابه
globoid cell leukodystrophy (krabbe disease)
how to cite this article: tavasoli a. globoid cell leukodystrophy (krabbe disease). iran j child neurol. autumn 2014;8;4(suppl.1):14-15. pls see pdf.
متن کاملCentral Nervous System Pathology Progresses Independently of KC and CXCR2 in Globoid-Cell Leukodystrophy
Globoid-cell Leukodystrophy (GLD; Krabbe's disease) is a rapidly progressing inherited demyelinating disease caused by a deficiency of the lysosomal enzyme Galactosylceramidase (GALC). Deficiency of GALC leads to altered catabolism of galactosylceramide and the cytotoxic lipid, galactosylsphingosine (psychosine). This leads to a rapidly progressive fatal disease with spasticity, cognitive disab...
متن کاملElectrodiagnostic study of peripheral nervous system in Behcet’s disease
Introduction: Bechcet’s disease (BD) is a chronic disease with multiple organ involvement such as central nervous system (CNS) and peripheral nervous system (PNS). Involvement of PNS has been reported controversially and uncommon in textbooks and literatures. The aim of this study in to evaluate the auxiliary role of electrodiagnosis in early detection of PNS involvement to prevent neuropathy...
متن کامل[Globoid cell leukodystrophy].
A 29 year old male with onset of globoid cell leukodystrophy at age 14 is described. This is the first case of enzymatically confirmed globoid cell leukodystrophy with onset of symptoms after the age of ten. This patient is unique because of the late onset and slow progression and extends the clinical spectrum of globoid cell leukodystrophy. Globoid cell leukodystrophy is caused by the deficien...
متن کاملEvaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII
Mucopolysaccharidosis VII (MPS VII) is a lysosomal storage disease arising from mutations in β-d-glucuronidase (GUSB), which results in glycosaminoglycan (GAG) accumulation and a variety of clinical manifestations including neurological disease. Herein, MPS VII dogs were injected intravenously (i.v.) and/or intrathecally (i.t.) via the cisterna magna with AAV9 or AAVrh10 vectors carrying the ca...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
ژورنال
عنوان ژورنال: Human Gene Therapy
سال: 2018
ISSN: 1043-0342,1557-7422
DOI: 10.1089/hum.2017.151