Detalhe da pesquisa
1.
Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice.
Mol Ther
; 32(4): 935-951, 2024 Apr 03.
Artigo
Inglês
| MEDLINE | ID: mdl-38327047
2.
Neuronal and epithelial cell rescue resolves chronic systemic inflammation in the lipid storage disorder Niemann-Pick C.
Hum Mol Genet
; 21(13): 2946-60, 2012 Jul 01.
Artigo
Inglês
| MEDLINE | ID: mdl-22493001
3.
Anatomically defined neuron-based rescue of neurodegenerative Niemann-Pick type C disorder.
J Neurosci
; 31(12): 4367-78, 2011 Mar 23.
Artigo
Inglês
| MEDLINE | ID: mdl-21430138
4.
Complement is dispensable for neurodegeneration in Niemann-Pick disease type C.
J Neuroinflammation
; 9: 216, 2012 Sep 17.
Artigo
Inglês
| MEDLINE | ID: mdl-22985423
5.
Diastereoselective preparation of (R)- and (S)-2-methoxy-2-phenylpent-3-ynoic acids and their use as reliable chiral derivatizing agents.
J Org Chem
; 77(4): 1640-52, 2012 Feb 17.
Artigo
Inglês
| MEDLINE | ID: mdl-22217094
6.
Combined in situ Hybridization/Immunohistochemistry (ISH/IH) on Free-floating Vibratome Tissue Sections.
Bio Protoc
; 4(18)2014 Sep 20.
Artigo
Inglês
| MEDLINE | ID: mdl-27547787
7.
Genetic dissection of a cell-autonomous neurodegenerative disorder: lessons learned from mouse models of Niemann-Pick disease type C.
Dis Model Mech
; 6(5): 1089-100, 2013 Sep.
Artigo
Inglês
| MEDLINE | ID: mdl-23907005
8.
A Quick, No Frills Approach to Mouse Genotyping.
Bio Protoc
; 2(15)2012 Aug 05.
Artigo
Inglês
| MEDLINE | ID: mdl-27547782