Detalhe da pesquisa
1.
An anti-neuroinflammatory that targets dysregulated glia enhances the efficacy of CNS-directed gene therapy in murine infantile neuronal ceroid lipofuscinosis.
J Neurosci
; 34(39): 13077-82, 2014 Sep 24.
Artigo
em Inglês
| MEDLINE | ID: mdl-25253854
2.
Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease.
Mol Ther Methods Clin Dev
; 27: 452-463, 2022 Dec 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-36419468
3.
A Cure for Sanfilippo Syndrome? A Summary of Current Therapeutic Approaches and their Promise.
Med Res Arch
; 8(2)2020 Feb 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32733997
4.
Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice.
Mol Ther Methods Clin Dev
; 10: 113-127, 2018 Sep 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-30101150
5.
Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene.
Hum Gene Ther
; 25(3): 223-39, 2014 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-24372003
6.
Human neural progenitor cell engraftment increases neurogenesis and microglial recruitment in the brain of rats with stroke.
PLoS One
; 7(11): e50444, 2012.
Artigo
em Inglês
| MEDLINE | ID: mdl-23185625