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1.
Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction.
J Biol Chem
; 298(12): 102625, 2022 12.
Article
in English
| MEDLINE | ID: mdl-36306823
2.
A lacZ reporter gene expression atlas for 313 adult KOMP mutant mouse lines.
Genome Res
; 25(4): 598-607, 2015 Apr.
Article
in English
| MEDLINE | ID: mdl-25591789
3.
Prophylactic Prednisolone Promotes AAV5 Hepatocyte Transduction Through the Novel Mechanism of AAV5 Coreceptor Platelet-Derived Growth Factor Receptor Alpha Upregulation and Innate Immune Suppression.
Hum Gene Ther
; 35(1-2): 36-47, 2024 Jan.
Article
in English
| MEDLINE | ID: mdl-38126359
4.
Sphingosine-1-phosphate lyase expression in embryonic and adult murine tissues.
J Lipid Res
; 53(9): 1920-31, 2012 Sep.
Article
in English
| MEDLINE | ID: mdl-22781001
5.
Mutant mice derived by ICSI of evaporatively dried spermatozoa exhibit expected phenotype.
Reproduction
; 143(4): 449-53, 2012 Apr.
Article
in English
| MEDLINE | ID: mdl-22274886
6.
Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B.
Drug Deliv Transl Res
; 10(2): 425-439, 2020 04.
Article
in English
| MEDLINE | ID: mdl-31942701
7.
Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice.
Mol Ther Methods Clin Dev
; 6: 43-53, 2017 Sep 15.
Article
in English
| MEDLINE | ID: mdl-28664165
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