Detalhe da pesquisa
1.
Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.
Mol Ther
; 24(2): 206-216, 2016 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-26447927
2.
Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I.
Proc Natl Acad Sci U S A
; 111(41): 14894-9, 2014 Oct 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-25267637
3.
Pharmacodynamics, pharmacokinetics and biodistribution of recombinant human N-acetylgalactosamine 4-sulfatase after 6months of therapy in cats using different IV infusion durations.
Mol Genet Metab
; 117(2): 157-63, 2016 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-26776148
4.
Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.
Mol Ther
; 23(8): 1298-1307, 2015 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-26022732
5.
Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I.
Mol Ther
; 22(12): 2018-2027, 2014 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-25027660
6.
Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease.
Sci Transl Med
; 7(276): 276ra26, 2015 Feb 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-25717099