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1.
Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional Benefit in MPS IIIB Mice after IV rAAV9-hNAGLU Gene Delivery.
Mol Ther
; 25(3): 792-802, 2017 03 01.
Artículo
en Inglés
| MEDLINE | ID: mdl-28143737
2.
Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery.
Mol Ther Methods Clin Dev
; 3: 16036, 2016.
Artículo
en Inglés
| MEDLINE | ID: mdl-27331076
3.
Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates.
Hum Gene Ther Clin Dev
; 25(2): 72-84, 2014 Jun.
Artículo
en Inglés
| MEDLINE | ID: mdl-24720466
4.
Amyloidosis, synucleinopathy, and prion encephalopathy in a neuropathic lysosomal storage disease: the CNS-biomarker potential of peripheral blood.
PLoS One
; 8(11): e80142, 2013.
Artículo
en Inglés
| MEDLINE | ID: mdl-24278249
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