Detalhe da pesquisa
1.
Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease.
Nucleic Acids Res
; 48(1): 36-54, 2020 01 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-31745548
2.
AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models.
Mol Ther Methods Clin Dev
; 13: 334-343, 2019 Jun 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-30984798
3.
AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes.
Mol Ther Methods Clin Dev
; 15: 275-284, 2019 Dec 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-31737741