Detalhe da pesquisa
1.
Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic.
Cell;
184(18): 4651-4668.e25, 2021 09 02.
Artigo
em Inglês
| MEDLINE
| ID: mdl-34450028
2.
Rescue of a lysosomal storage disorder caused by Grn loss of function with a brain penetrant progranulin biologic.
Cell;
187(6): 1565-1566, 2024 Mar 14.
Artigo
em Inglês
| MEDLINE
| ID: mdl-38490183
3.
Characterization of Fluid Biomarkers Reveals Lysosome Dysfunction and Neurodegeneration in Neuronopathic MPS II Patients.
Int J Mol Sci;
21(15)2020 Jul 22.
Artigo
em Inglês
| MEDLINE
| ID: mdl-32707880
4.
High-Throughput Liquid Chromatography-Tandem Mass Spectrometry Quantification of Glycosaminoglycans as Biomarkers of Mucopolysaccharidosis II.
Int J Mol Sci;
21(15)2020 Jul 30.
Artigo
em Inglês
| MEDLINE
| ID: mdl-32751752
5.
Alterations in Lysosomal, Glial and Neurodegenerative Biomarkers in Patients with Sporadic and Genetic Forms of Frontotemporal Dementia.
bioRxiv;
2024 Feb 12.
Artigo
em Inglês
| MEDLINE
| ID: mdl-38405775
6.
Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome.
JCI Insight;
6(19)2021 10 08.
Artigo
em Inglês
| MEDLINE
| ID: mdl-34622797
7.
Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice.
Sci Transl Med;
12(545)2020 05 27.
Artigo
em Inglês
| MEDLINE
| ID: mdl-32461331