Detalhe da pesquisa
1.
Generation and characterization of an immunodeficient mouse model of mucopolysaccharidosis type II.
Mol Genet Metab;
138(4): 107539, 2023 04.
Artigo
em Inglês
| MEDLINE
| ID: mdl-37023503
2.
Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice.
Mol Ther Methods Clin Dev;
32(1): 101201, 2024 Mar 14.
Artigo
em Inglês
| MEDLINE
| ID: mdl-38374962
3.
Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells.
Hum Gene Ther;
33(23-24): 1279-1292, 2022 12.
Artigo
em Inglês
| MEDLINE
| ID: mdl-36226412