Detalhe da pesquisa
1.
A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency.
Brain
; 141(7): 2014-2031, 2018 07 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29788236
2.
Systemic immune challenge exacerbates neurodegeneration in a model of neurological lysosomal disease.
EMBO Mol Med
; 2024 Jun 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-38890537
3.
Trial and error: how the unclonable human mitochondrial genome was cloned in yeast.
Pharm Res
; 28(11): 2863-70, 2011 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-21739320
4.
Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes.
Sci Rep
; 9(1): 14105, 2019 Oct 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31575939
5.
Brain-targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms.
EMBO Mol Med
; 10(7)2018 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-29884617