Detalhe da pesquisa
1.
Progression of vertebral bone disease in mucopolysaccharidosis VII dogs from birth to skeletal maturity.
Mol Genet Metab
; 133(4): 378-385, 2021 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-34154922
2.
Global CNS correction in a large brain model of human alpha-mannosidosis by intravascular gene therapy.
Brain
; 143(7): 2058-2072, 2020 07 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32671406
3.
Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I.
Mol Ther
; 28(6): 1455-1463, 2020 06 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-32330426
4.
Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1.
Mol Ther
; 24(1): 26-33, 2016 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-26354342
5.
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
6.
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
7.
Clinical, electrophysiological, and biochemical markers of peripheral and central nervous system disease in canine globoid cell leukodystrophy (Krabbe's disease).
J Neurosci Res
; 94(11): 1007-17, 2016 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-27638585
8.
Cerebrospinal Fluid Calbindin D Concentration as a Biomarker of Cerebellar Disease Progression in Niemann-Pick Type C1 Disease.
J Pharmacol Exp Ther
; 358(2): 254-61, 2016 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-27307499
9.
Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model.
Mol Genet Metab
; 119(1-2): 124-30, 2016 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-27386755
10.
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
11.
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
12.
The effect of neonatal gene therapy with a gamma retroviral vector on cardiac valve disease in mucopolysaccharidosis VII dogs after a decade.
Mol Genet Metab
; 110(3): 311-8, 2013 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-23860311
13.
The effect of neonatal gene therapy on skeletal manifestations in mucopolysaccharidosis VII dogs after a decade.
Mol Genet Metab
; 109(2): 183-93, 2013 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-23628461
14.
Pathogenesis of mitral valve disease in mucopolysaccharidosis VII dogs.
Mol Genet Metab
; 110(3): 319-28, 2013 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-23856419
15.
Neonatal gene therapy with a gamma retroviral vector in mucopolysaccharidosis VI cats.
Mol Ther
; 20(5): 898-907, 2012 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-22395531
16.
Effect of neonatal gene therapy on lumbar spine disease in mucopolysaccharidosis VII dogs.
Mol Genet Metab
; 107(1-2): 145-52, 2012 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-22510705
17.
Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer.
Mol Ther
; 19(3): 461-9, 2011 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-21119624
18.
Transduction characteristics of alternative adeno-associated virus serotypes in the cat brain by intracisternal delivery.
Mol Ther Methods Clin Dev
; 26: 384-393, 2022 Sep 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36034772
19.
How do patients and physicians communicate about hereditary angioedema in the United States?
PLoS One
; 16(12): e0260805, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34855883
20.
Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors.
Mol Ther Methods Clin Dev
; 20: 247-257, 2021 Mar 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-33473358