S. Le

发表

Mannose 6-phosphate conjugation is not sufficient to allow induction of immune tolerance to phenylalanine ammonia-lyase in dogs.

J. Lemontt, P. Dickson, M. Vera, 2013, JIMD reports.

Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes

E. I. Pierpont, J. Tolar, J. Eisengart, 2019, Genetics in Medicine.

Neuropathology of murine Sanfilippo D syndrome.

P. Dickson, S. Kan, S. Le, 2021, Molecular genetics and metabolism.

Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts.

P. Dickson, S. Kan, K. Haitz, 2014, The Biochemical journal.

Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease

M. Iacovino, J. Cooper, P. Dickson, 2022, bioRxiv.

Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidase

M. Mcentee, Agnes H. Chen, P. Dickson, 2011, APMIS : acta pathologica, microbiologica, et immunologica Scandinavica.

Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB

P. Fitzpatrick, S. Bunting, A. Bagri, 2014, Proceedings of the National Academy of Sciences.

Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I.

W. Mobley, M. Mcentee, P. Dickson, 2004, Molecular genetics and metabolism.

Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I.

I. Kaitila, D. Naylor, Agnes H. Chen, 2015, Molecular genetics and metabolism.

Replacing the Enzyme α-l-Iduronidase at Birth Ameliorates Symptoms in the Brain and Periphery of Dogs with Mucopolysaccharidosis Type I

R. D. Whitley, C. Vite, E. Riedesel, 2010, Science Translational Medicine.

Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study.

J. Eisengart, Agnes H. Chen, P. Dickson, 2014, Molecular genetics and metabolism.

Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-N-Acetylglucosamine-6-Sulfatase in Neonatal Mice.

S. Ekins, T. Chou, P. Dickson, 2020, Molecular pharmaceutics.

A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I

M. Iacovino, J. Cooper, M. Sands, 2017, Molecular therapy. Methods & clinical development.

Cardiovascular‐related proteins identified in human plasma by the HUPO Plasma Proteome Project Pilot Phase

Peipei Ping, Chenggong Zong, P. Ping, 2005, Proteomics.

Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I.

C. Vite, M. Mcentee, M. Haskins, 2010, Molecular genetics and metabolism.

Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.

M. Mcentee, P. Dickson, E. Kakkis, 2008, The Journal of clinical investigation.

Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients

I. Kaitila, D. Naylor, Agnes H. Chen, 2013, Pediatric Research.

Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I.

P. Dickson, M. Vera, J. Brown, 2012, Molecular genetics and metabolism.

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.

D. Millington, Agnes H. Chen, P. Dickson, 2020, Clinica chimica acta; international journal of clinical chemistry.

Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep

S. Ekins, C. Whitelaw, S. Lillico, 2022, The Journal of clinical investigation.

Behavioral deficits and cholinergic pathway abnormalities in male Sanfilippo B mice

Braeden C. Benedict, M. Sands, P. Dickson, 2016, Behavioural Brain Research.

A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I

J. Jester, J. Cooper, E. Monuki, 2015, Molecular therapy. Methods & clinical development.

Recombinant NAGLU-IGF2 prevents physical and neurological disease and improves survival in Sanfilippo B syndrome

Jodi D. Smith, T. Chou, J. Cooper, 2021, bioRxiv.

Myelin and Lipid Composition of the Corpus Callosum in Mucopolysaccharidosis Type I Mice.

D. Elashoff, J. Tolar, J. Cooper, 2020, Lipids.

Diffusion tensor imaging and myelin composition analysis reveal abnormal myelination in corpus callosum of canine mucopolysaccharidosis I

James M. Provenzale, Igor Nestrasil, David Elashoff, 2015, Experimental Neurology.

Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid.

M. Mcentee, P. Dickson, C. Vogler, 2007, Molecular genetics and metabolism.

Features of brain MRI in dogs with treated and untreated mucopolysaccharidosis type I.

Igor Nestrasil, James M Provenzale, C. Vite, 2013, Comparative medicine.

Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome.

J. Cooper, M. Sands, P. Dickson, 2021, Molecular genetics and metabolism.

Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice

M. Iacovino, J. Cooper, P. Dickson, 2018, Molecular therapy. Methods & clinical development.

Evaluation of non-reducing end pathologic glycosaminoglycan detection method for monitoring therapeutic response to enzyme replacement therapy in human mucopolysaccharidosis I.

Agnes H. Chen, P. Dickson, M. Vera, 2020, Molecular genetics and metabolism.

Continuous infusion of enzyme replacement therapy is inferior to weekly infusions in MPS I dogs

P. Dickson, E. Kakkis, T. Lester, 2009, Journal of Inherited Metabolic Disease.

Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I.

J. Esko, M. Mcentee, P. Dickson, 2014, Molecular genetics and metabolism.

The Murine Cardiac 26S Proteasome: An Organelle Awaiting Exploration

P. Ping, T. Vondriska, A. Gomes, 2005, Annals of the New York Academy of Sciences.

Biochemical characterization of fluorescent‐labeled recombinant human alpha‐l‐iduronidase in vitro

P. Dickson, S. Kan, B. Tippin, 2011, Biotechnology and applied biochemistry.