Detalhe da pesquisa
1.
Lentiviral gene therapy with IGF2-tagged GAA normalizes the skeletal muscle proteome in murine Pompe disease.
J Proteomics
; 291: 105037, 2024 01 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-38288553
2.
Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage.
Hum Gene Ther
; 35(7-8): 256-268, 2024 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-38085235
3.
Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II.
Mol Ther Methods Clin Dev
; 31: 101149, 2023 Dec 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38033460
4.
Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease.
Mol Ther Methods Clin Dev
; 25: 520-532, 2022 Jun 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-35662813
5.
IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy.
Mol Ther Methods Clin Dev
; 27: 109-130, 2022 Dec 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36284764