Author details

Full Name

Steven Q Le

Affiliation

ORCID

Career Start Year

2004

Papers

H Index

Expertise

CM4AI Collaborator

PMID	Paper Title	Journal Title	Published Year
36419468	Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease.	Mol Ther Methods Clin Dev	2022
36040802	Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep.	J Clin Invest	2022
34844863	Neuropathology of murine Sanfilippo D syndrome.	Molecular Genetics and Metabolism	2021
33839004	Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome.	Mol Genet Metab	2021
33320673	Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human Î±--Acetylglucosamine-6-Sulfatase in Neonatal Mice.	Molecular Pharmaceutics	2021
32537944	Myelin and Lipid Composition of the Corpus Callosum in Mucopolysaccharidosis Type I Mice.	Lipids	2020
31839529	Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study.	Mol Genet Metab	2020
31630958	Evaluation of non-reducing end pathologic glycosaminoglycan detection method for monitoring therapeutic response to enzyme replacement therapy in human mucopolysaccharidosis I.	Molecular Genetics and Metabolism	2020
32442432	Comparison of dermatan sulfate and heparan sulfate concentrations in serum, cerebrospinal fluid and urine in patients with mucopolysaccharidosis type I receiving intravenous and intrathecal enzyme replacement therapy.	Clinica Chimica Acta	2020
31019279	Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes.	Genet Med	2019
30101150	Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice.	Mol Ther Methods Clin Dev	2018
29159202	A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I.	Mol Ther Methods Clin Dev	2017
27340089	Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice.	Behavioural Brain Research	2016
26260077	Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I.	Molecular Genetics and Metabolism	2015
26484358	Data from subjects receiving intrathecal laronidase for cervical spinal stenosis due to mucopolysaccharidosis type I.	Data in Brief	2015
26222335	Diffusion tensor imaging and myelin composition analysis reveal abnormal myelination in corpus callosum of canine mucopolysaccharidosis I.	Exp Neurol	2015
26052536	A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I.	Mol Ther Methods Clin Dev	2015
24951454	Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I.	Molecular Genetics and Metabolism	2014
25267636	Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB.	Proceedings of the National Academy of Sciences of the United States of America	2014
24266751	Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of Î±-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts.	Biochemical Journal	2014
23582423	Features of brain MRI in dogs with treated and untreated mucopolysaccharidosis type I.	Comp Med	2013
24002329	Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients.	Pediatric Research	2013
23430522	Mannose 6-phosphate conjugation is not sufficient to allow induction of immune tolerance to phenylalanine ammonia-lyase in dogs.	JIMD Reports	2013
22402327	Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I.	Molecular Genetics and Metabolism	2012
21749451	Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidase.	APMIS	2011
22172101	Biochemical characterization of fluorescent-labeled recombinant human alpha-L-iduronidase in vitro.	Biotechnology and Applied Biochemistry	2011
20655780	Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I.	Molecular Genetics and Metabolism	2010
21123810	Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I.	Science Translational Medicine	2010
19562502	Continuous infusion of enzyme replacement therapy is inferior to weekly infusions in MPS I dogs.	Journal of Inherited Metabolic Disease	2009
18654665	Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.	Journal of Clinical Investigation	2008
17321776	Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid.	Molecular Genetics and Metabolism	2007
16052623	Cardiovascular-related proteins identified in human plasma by the HUPO Plasma Proteome Project pilot phase.	Proteomics	2005
16093497	The murine cardiac 26S proteasome: an organelle awaiting exploration.	Ann N Y Acad Sci	2005
15464431	Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I.	Mol Genet Metab	2004