Detalhe da pesquisa
1.
Profound phenotypic and epigenetic heterogeneity of the HIV-1-infected CD4+ T cell reservoir.
Nat Immunol
; 24(2): 359-370, 2023 02.
Artigo
Inglês
| MEDLINE | ID: mdl-36536105
2.
Adeno-associated viral gene therapy corrects a mouse model of argininosuccinic aciduria.
Mol Genet Metab
; 125(3): 241-250, 2018 11.
Artigo
Inglês
| MEDLINE | ID: mdl-30253962
3.
Determining the Minimally Effective Dose of a Clinical Candidate Adeno-Associated Virus Vector in a Mouse Model of Hemophilia A.
Hum Gene Ther
; 33(7-8): 421-431, 2022 04.
Artigo
Inglês
| MEDLINE | ID: mdl-34652966
4.
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.
Nat Commun
; 12(1): 6393, 2021 11 04.
Artigo
Inglês
| MEDLINE | ID: mdl-34737297
5.
A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson Disease.
Hum Gene Ther Clin Dev
; 30(1): 29-39, 2019 03.
Artigo
Inglês
| MEDLINE | ID: mdl-30693797
6.
AAV8 Gene Therapy Rescues the Newborn Phenotype of a Mouse Model of Crigler-Najjar.
Hum Gene Ther
; 29(7): 763-770, 2018 07.
Artigo
Inglês
| MEDLINE | ID: mdl-29448836
7.
Determining the Minimally Effective Dose of a Clinical Candidate AAV Vector in a Mouse Model of Crigler-Najjar Syndrome.
Mol Ther Methods Clin Dev
; 10: 237-244, 2018 Sep 21.
Artigo
Inglês
| MEDLINE | ID: mdl-30112420
8.
Optimized Adeno-Associated Viral-Mediated Human Factor VIII Gene Therapy in Cynomolgus Macaques.
Hum Gene Ther
; 2018 Jul 23.
Artigo
Inglês
| MEDLINE | ID: mdl-29890905
9.
AAV8 Gene Therapy for Crigler-Najjar Syndrome in Macaques Elicited Transgene T Cell Responses That Are Resident to the Liver.
Mol Ther Methods Clin Dev
; 11: 191-201, 2018 Dec 14.
Artigo
Inglês
| MEDLINE | ID: mdl-30547050