ArticleAPMIS : acta pathologica, microbiologica, et immunologica Scandinavica2011
Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidase.
Article in APMIS : acta pathologica, microbiologica, et immunologica Scandinavica, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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Who cites it
11 citing papers in PubMed, 16 citations in OpenAlex.
- Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Review
- Quantitative brain MRI morphology in severe and attenuated forms of mucopolysaccharidosis type I.Molecular genetics and metabolism · 2022Article
- Transcranial pulsed ultrasound facilitates brain uptake of laronidase in enzyme replacement therapy for Mucopolysaccharidosis type I disease.Orphanet journal of rare diseases · 2017Article
- Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model.Molecular genetics and metabolism · 2016Article
- Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I.Molecular therapy : the journal of the American Society of Gene Therapy · 2014Article
- Aging in the canine and feline brain.The Veterinary clinics of North America. Small animal practice · 2014Review
- Isokinetic muscle strength differences in patients with mucopolysaccharidosis I, II, and VI.Journal of pediatric rehabilitation medicine · 2014Article
- Features of brain MRI in dogs with treated and untreated mucopolysaccharidosis type I.Comparative medicine · 2013Article
- Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl-protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis.Molecular genetics and metabolism · 2012Article
- Delivering drugs to the central nervous system: an overview.Drug delivery and translational research · 2012Article
- Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I.Molecular genetics and metabolismArticle
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Authors and funding
11 authors at 6 institutions in 1 country.
Funding
Abstract
Intrathecal (IT) recombinant human α-l-iduronidase (rhIDU) has been shown to reduce mean brain glycosaminoglycans (GAGs) to normal levels in mucopolysaccharidosis I (MPS I) dogs. In this study, we examined storage in neuroanatomical regions of the MPS I dog brain, including frontal lobe, cerebellum, basal ganglia, thalamus, hippocampal formation, and brainstem, to determine the response of these functional regions to treatment with IT rhIDU. GAG storage in untreated MPS I dogs was significantly different from normal dogs in all examined sections. GAG levels in normal dogs varied by region: frontal lobe (mean: 2.36 ± 0.54 μg/mg protein), cerebellum (2.67 ± 0.33), basal ganglia and thalamus (3.51 ± 0.60), hippocampus (3.30 ± 0.40), and brainstem (3.73 ± 1.10). Following IT treatment, there was a reduction in GAG storage in each region in all treatment groups, except for the brainstem. Percent reduction in GAG levels from untreated to treated MPS I dogs in the deeper regions of the brain was 30% for basal ganglia and thalamus and 30% for hippocampus, and storage reduction was greater in superficial regions, with 61% reduction in the frontal lobe and 54% in the cerebellum compared with untreated MPS I dogs. Secondary lipid storage in neurons was also reduced in frontal lobe, but not in the other brain regions examined. Response to therapy appeared to be greater in more superficial regions of the brain, particularly in the frontal lobe cortex.
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