Detalhe da pesquisa
1.
A Stable Micellar Formulation of RAD001 for Intracerebroventricular Delivery and the Treatment of Alzheimer's Disease and Other Neurological Disorders.
Int J Mol Sci
; 24(24)2023 Dec 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38139306
2.
TFEB activation restores migration ability to Tsc1-deficient adult neural stem/progenitor cells.
Hum Mol Genet
; 26(17): 3303-3312, 2017 09 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-28637240
3.
Use of Polylactide-Co-Glycolide-Nanoparticles for Lysosomal Delivery of a Therapeutic Enzyme in Glycogenosis Type II Fibroblasts.
J Nanosci Nanotechnol
; 15(4): 2657-66, 2015 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-26353478
4.
HIV-1 TAT-mediated protein transduction of human HPRT into deficient cells.
Biochem Biophys Res Commun
; 441(1): 114-9, 2013 Nov 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-24129187
5.
Analysis of the HPRT1 gene in 35 Italian Lesch-Nyhan families: 45 patients and 77 potential female carriers.
Mutat Res
; 692(1-2): 1-5, 2010 Oct 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-20638392
6.
Description of the Lesch-Nyhan neurobehavioral disorder and its management through participant observation of three young individuals.
JIMD Rep
; 52(1): 63-71, 2020 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-32154061
7.
Age-related hearing loss in four Italian genetic isolates: an epidemiological study.
Int J Audiol
; 48(7): 465-72, 2009.
Artigo
em Inglês
| MEDLINE | ID: mdl-19925333
8.
In vitro treatment of congenital disorder of glycosylation type Ia using PLGA nanoparticles loaded with GDPMan.
Int J Mol Med
; 44(1): 262-272, 2019 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-31115488
9.
Early intrathecal infusion of everolimus restores cognitive function and mood in a murine model of Alzheimer's disease.
Exp Neurol
; 311: 88-105, 2019 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-30243986
10.
Metabolic correction in oligodendrocytes derived from metachromatic leukodystrophy mouse model by using encapsulated recombinant myoblasts.
J Neurol Sci
; 255(1-2): 7-16, 2007 Apr 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-17341424
11.
Evaluation of a LC-MS method for everolimus preclinical determination in brain by using [(13)C2D4]RAD001 internal standard.
J Chromatogr B Analyt Technol Biomed Life Sci
; 985: 155-63, 2015 Mar 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-25682337
12.
The local mammalian target of rapamycin (mTOR) modulation: a promising strategy to counteract neurodegeneration.
Neural Regen Res
; 14(10): 1711-1712, 2019 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-31169184
13.
An Italian cohort study identifies four new pathologic mutations in the ARSA gene.
J Mol Neurosci
; 50(2): 284-90, 2013 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-23559313
14.
Quantitative evaluation of the clinical effects of S-adenosylmethionine on mood and behavior in Lesch-Nyhan patients.
Nucleosides Nucleotides Nucleic Acids
; 32(4): 174-88, 2013.
Artigo
em Inglês
| MEDLINE | ID: mdl-24001191
15.
Specific treatment of Prader-Willi syndrome through cyclical rehabilitation programmes.
Disabil Rehabil
; 33(19-20): 1837-47, 2011.
Artigo
em Inglês
| MEDLINE | ID: mdl-21269146
16.
The therapeutic potential of neural stem/progenitor cells in murine globoid cell leukodystrophy is conditioned by macrophage/microglia activation.
Neurobiol Dis
; 21(2): 314-23, 2006 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-16199167
17.
Robust in vivo gene transfer into adult mammalian neural stem cells by lentiviral vectors.
Proc Natl Acad Sci U S A
; 101(41): 14835-40, 2004 Oct 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-15466696