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Plos One
|
July 25, 2025
Recombinant human alpha-N-acetylglucosamine-6-sulfatase delivered to Sanfilippo D mice with repeated intracerebroventricular injections corrects CNS pathology
Grant L Austin, Feng Wang, Steven Q Le, et al.
Data in Brief
|
October 21, 2015
Data from subjects receiving intrathecal laronidase for cervical spinal stenosis due to mucopolysaccharidosis type I
P I Dickson, I Kaitila, P Harmatz, et al.
Lipids
|
June 16, 2020
Myelin and Lipid Composition of the Corpus Callosum in Mucopolysaccharidosis Type I Mice
Steven Q Le, Igor Nestrasil, Shih-Hsin Kan, et al.
Molecular Genetics and Metabolism
|
August 12, 2015
Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I
Patricia I Dickson, Ilkka Kaitila, Paul Harmatz, et al.
Molecular Genetics and Metabolism
|
October 22, 2019
Evaluation of non-reducing end pathologic glycosaminoglycan detection method for monitoring therapeutic response to enzyme replacement therapy in human mucopolysaccharidosis I
Moin U Vera, Steven Q Le, Alla Victoroff, et al.
Molecular Genetics and Metabolism
|
April 11, 2021
Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome
Shih-Hsin Kan, Ibrahim Elsharkawi, Steven Q Le, et al.
The Biochemical Journal
|
November 26, 2013
Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts
Shih-hsin Kan, Larisa A Troitskaya, Carolyn S Sinow, et al.
American Journal of Medical Genetics. Part A
|
September 17, 2008
Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression
Maria-Veronica Munoz-Rojas, Taiane Vieira, Ronaldo Costa, et al.
Molecular Therapy. Methods & Clinical Development
|
November 24, 2022
Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease
Yewande Pearse, Don Clarke, Shih-Hsin Kan, et al.
Advances in Cell and Gene Therapy
|
August 29, 2025
Intra-Articular AAV9 <i>α</i>-l-Iduronidase Gene Replacement in the Canine Model of Mucopolysaccharidosis Type I
Raymond Yu-Jeang Wang, Shih-Hsin Kan, Haoyue Zhang, et al.
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Search research articles
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Showing results (51-60 of 93) with videos related to
Sort By:
Page
of 10
Plos One
|
July 25, 2025
Recombinant human alpha-N-acetylglucosamine-6-sulfatase delivered to Sanfilippo D mice with repeated intracerebroventricular injections corrects CNS pathology
Grant L Austin, Feng Wang, Steven Q Le, et al.
Data in Brief
|
October 21, 2015
Data from subjects receiving intrathecal laronidase for cervical spinal stenosis due to mucopolysaccharidosis type I
P I Dickson, I Kaitila, P Harmatz, et al.
Lipids
|
June 16, 2020
Myelin and Lipid Composition of the Corpus Callosum in Mucopolysaccharidosis Type I Mice
Steven Q Le, Igor Nestrasil, Shih-Hsin Kan, et al.
Molecular Genetics and Metabolism
|
August 12, 2015
Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I
Patricia I Dickson, Ilkka Kaitila, Paul Harmatz, et al.
Molecular Genetics and Metabolism
|
October 22, 2019
Evaluation of non-reducing end pathologic glycosaminoglycan detection method for monitoring therapeutic response to enzyme replacement therapy in human mucopolysaccharidosis I
Moin U Vera, Steven Q Le, Alla Victoroff, et al.
Molecular Genetics and Metabolism
|
April 11, 2021
Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome
Shih-Hsin Kan, Ibrahim Elsharkawi, Steven Q Le, et al.
The Biochemical Journal
|
November 26, 2013
Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts
Shih-hsin Kan, Larisa A Troitskaya, Carolyn S Sinow, et al.
American Journal of Medical Genetics. Part A
|
September 17, 2008
Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression
Maria-Veronica Munoz-Rojas, Taiane Vieira, Ronaldo Costa, et al.
Molecular Therapy. Methods & Clinical Development
|
November 24, 2022
Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease
Yewande Pearse, Don Clarke, Shih-Hsin Kan, et al.
Advances in Cell and Gene Therapy
|
August 29, 2025
Intra-Articular AAV9 <i>α</i>-l-Iduronidase Gene Replacement in the Canine Model of Mucopolysaccharidosis Type I
Raymond Yu-Jeang Wang, Shih-Hsin Kan, Haoyue Zhang, et al.
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