Detalhe da pesquisa
1.
Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology.
Brain Sci
; 11(2)2021 Jan 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-33498212
2.
Secreted therapeutics: monitoring durability of microRNA-based gene therapies in the central nervous system.
Brain Commun
; 3(2): fcab054, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34704020
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
4.
In-Depth Characterization of a Mifepristone-Regulated Expression System for AAV5-Mediated Gene Therapy in the Liver.
Mol Ther Methods Clin Dev
; 13: 512-525, 2019 Jun 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-31194088
5.
Development of an AAV-Based MicroRNA Gene Therapy to Treat Machado-Joseph Disease.
Mol Ther Methods Clin Dev
; 15: 343-358, 2019 Dec 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-31828177