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Molecular Therapy. Methods & Clinical Development
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August 14, 2018
Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice
Don Clarke, Yewande Pearse, Shih-Hsin Kan, et al.
Neurology. Genetics
|
December 16, 2022
Whole-Genome and Long-Read Sequencing Identify a Novel Mechanism in <i>RFC1</i> Resulting in CANVAS Syndrome
Katherine Abell King, Daniel J Wegner, Robert C Bucelli, et al.
Scientific Reports
|
December 3, 2016
Elevated cerebral spinal fluid biomarkers in children with mucopolysaccharidosis I-H
Gerald V Raymond, Marzia Pasquali, Lynda E Polgreen, et al.
The Neuroradiology Journal
|
July 14, 2017
Quantitative diffusion tensor imaging analysis does not distinguish pediatric canines with mucopolysaccharidosis I from control canines
Dana M Middleton, Jonathan Y Li, Steven D Chen, et al.
Laboratory Investigation; a Journal of Technical Methods and Pathology
|
March 9, 2011
Arterial pathology in canine mucopolysaccharidosis-I and response to therapy
Jeremiah A Lyons, Patricia I Dickson, Jonathan S Wall, et al.
Molecular Genetics and Metabolism
|
July 20, 2015
Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann-Pick C1
Timothy J Maarup, Agnes H Chen, Forbes D Porter, et al.
Plos One
|
March 18, 2016
Immune-Mediated Inflammation May Contribute to the Pathogenesis of Cardiovascular Disease in Mucopolysaccharidosis Type I
Omar Khalid, Moin U Vera, Philip L Gordts, et al.
The Neuroradiology Journal
|
June 21, 2017
Diffusion tensor imaging tensor shape analysis for assessment of regional white matter differences
Dana M Middleton, Jonathan Y Li, Hui J Lee, et al.
Clinica Chimica Acta; International Journal of Clinical Chemistry
|
May 23, 2020
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
Haoyue Zhang, Patricia I Dickson, Ashlee R Stiles, et al.
Molecular Genetics and Metabolism
|
March 10, 2012
Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I
Patricia I Dickson, N Matthew Ellinwood, Jillian R Brown, et al.
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of 10
Search research articles
Search
Showing results (41-50 of 93) with videos related to
Sort By:
Page
of 10
Molecular Therapy. Methods & Clinical Development
|
August 14, 2018
Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice
Don Clarke, Yewande Pearse, Shih-Hsin Kan, et al.
Neurology. Genetics
|
December 16, 2022
Whole-Genome and Long-Read Sequencing Identify a Novel Mechanism in <i>RFC1</i> Resulting in CANVAS Syndrome
Katherine Abell King, Daniel J Wegner, Robert C Bucelli, et al.
Scientific Reports
|
December 3, 2016
Elevated cerebral spinal fluid biomarkers in children with mucopolysaccharidosis I-H
Gerald V Raymond, Marzia Pasquali, Lynda E Polgreen, et al.
The Neuroradiology Journal
|
July 14, 2017
Quantitative diffusion tensor imaging analysis does not distinguish pediatric canines with mucopolysaccharidosis I from control canines
Dana M Middleton, Jonathan Y Li, Steven D Chen, et al.
Laboratory Investigation; a Journal of Technical Methods and Pathology
|
March 9, 2011
Arterial pathology in canine mucopolysaccharidosis-I and response to therapy
Jeremiah A Lyons, Patricia I Dickson, Jonathan S Wall, et al.
Molecular Genetics and Metabolism
|
July 20, 2015
Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann-Pick C1
Timothy J Maarup, Agnes H Chen, Forbes D Porter, et al.
Plos One
|
March 18, 2016
Immune-Mediated Inflammation May Contribute to the Pathogenesis of Cardiovascular Disease in Mucopolysaccharidosis Type I
Omar Khalid, Moin U Vera, Philip L Gordts, et al.
The Neuroradiology Journal
|
June 21, 2017
Diffusion tensor imaging tensor shape analysis for assessment of regional white matter differences
Dana M Middleton, Jonathan Y Li, Hui J Lee, et al.
Clinica Chimica Acta; International Journal of Clinical Chemistry
|
May 23, 2020
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
Haoyue Zhang, Patricia I Dickson, Ashlee R Stiles, et al.
Molecular Genetics and Metabolism
|
March 10, 2012
Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I
Patricia I Dickson, N Matthew Ellinwood, Jillian R Brown, et al.
Page
of 10