Detalhe da pesquisa
1.
Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B.
Mol Ther Methods Clin Dev
; 28: 396-411, 2023 Mar 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-36910588
2.
Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients.
Front Immunol
; 13: 857276, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-35464422
3.
Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI.
NEJM Evid
; 1(7): EVIDoa2200052, 2022 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-38319253
4.
Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA.
Mol Ther Methods Clin Dev
; 15: 333-342, 2019 Dec 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-31788497