Cell‐Based Therapy for Canavan Disease Using Human iPSC‐Derived NPCs and OPCs
نویسندگان
چکیده
منابع مشابه
Long-term follow-up after gene therapy for canavan disease.
Canavan disease is a hereditary leukodystrophy caused by mutations in the aspartoacylase gene (ASPA), leading to loss of enzyme activity and increased concentrations of the substrate N-acetyl-aspartate (NAA) in the brain. Accumulation of NAA results in spongiform degeneration of white matter and severe impairment of psychomotor development. The goal of this prospective cohort study was to asses...
متن کاملLithium citrate for Canavan disease.
Current evidence suggests that the effects of lithium on metabolic and signaling pathways in the brain may vary depending on the specific clinical condition or disease model. For example, lithium increases levels of cerebral N-acetyl aspartate in patients with bipolar disorder but does not appear to affect N-acetyl aspartate levels in normal human subjects. Conversely, lithium significantly dec...
متن کاملalexander and canavan disease
how to cite this article: shalbafan b. alexander and canavan disease. iran j child neurol. autumn 2014;8;4(suppl.1):20-21.
متن کاملNon-genetic therapeutic approaches to Canavan disease.
Canavan disease (CD) is a rare leukodystrophy characterized by diffuse spongiform white matter degeneration, dysmyelination and intramyelinic oedema with consequent impairment of psychomotor development and early death. The molecular cause of CD has been identified as being mutations of the gene encoding the enzyme aspartoacylase (ASPA) leading to its functional deficiency. The physiological ro...
متن کاملCanavan disease: studies on the knockout mouse.
Canavan disease (CD) is an autosomal recessive disorder, characterized by spongy degeneration of the brain. Patients with CD have aspartoacylase (ASPA) deficiency, which results accumulation of N-acetylaspartic acid (NAA) in the brain and elevated excretion of urinary NAA. Clinically, patients with CD have macrocephaly, mental retardation and hypotonia. A knockout mouse for CD which was enginee...
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ژورنال
عنوان ژورنال: Advanced Science
سال: 2020
ISSN: 2198-3844,2198-3844
DOI: 10.1002/advs.202002155