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1.
Serum global metabolomics profiling reveals profound metabolic impairments in patients with MPS IIIA and MPS IIIB.
Metab Brain Dis
; 32(5): 1403-1415, 2017 10.
Artículo
en Inglés
| MEDLINE | ID: mdl-28382573
2.
Threshold for Pre-existing Antibody Levels Limiting Transduction Efficiency of Systemic rAAV9 Gene Delivery: Relevance for Translation.
Mol Ther Methods Clin Dev
; 13: 453-462, 2019 Jun 14.
Artículo
en Inglés
| MEDLINE | ID: mdl-31193101
3.
Targeting Root Cause by Systemic scAAV9-hIDS Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice.
Mol Ther Methods Clin Dev
; 10: 327-340, 2018 Sep 21.
Artículo
en Inglés
| MEDLINE | ID: mdl-30191159
4.
Effective Depletion of Pre-existing Anti-AAV Antibodies Requires Broad Immune Targeting.
Mol Ther Methods Clin Dev
; 4: 159-168, 2017 Mar 17.
Artículo
en Inglés
| MEDLINE | ID: mdl-28345001
5.
Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy.
Hum Gene Ther Clin Dev
; 28(4): 187-196, 2017 12.
Artículo
en Inglés
| MEDLINE | ID: mdl-29064732
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