|
Parkinson Disease
|
0.100 |
Biomarker
|
disease |
BEFREE |
Conversion of human umbilical cord mesenchymal stem cells in Wharton's jelly to dopamine neurons mediated by the Lmx1a and neurturin in vitro: potential therapeutic application for Parkinson's disease in a rhesus monkey model.
|
23724014 |
2013 |
|
Parkinson Disease
|
0.100 |
Biomarker
|
disease |
BEFREE |
As alternative therapeutic strategies, the following four approaches are currently under evaluation for clinical gene therapy trials in PD; 1) recombinant adeno-associated virus 2 system encoding aromatic L-amino acid decarboxylase (AADC), 2) glutamic acid decarboxylase (GAD) and 3) Neurturin, and 4) equine infectious anemia virus-based lentiviral system encoding AADC, tyrosine hydroxylase (TH) and GTP cyclohydrolase I (GCH) in a single transcriptional unit.
|
22834832 |
2012 |
|
Parkinson Disease
|
0.100 |
Biomarker
|
disease |
BEFREE |
Current gene therapy approaches for Parkinson's disease (PD) deliver neurotrophic factors like glial cell line-derived neurotrophic factor (GDNF) or neurturin via neuronal transgene expression.
|
22086235 |
2012 |
|
Parkinson Disease
|
0.100 |
AlteredExpression
|
disease |
BEFREE |
NRTN-expression was associated with occasional, sparse TH-induction in the striatum of PD, but nothing apparent in the SNc.
|
21322017 |
2011 |
|
Parkinson Disease
|
0.100 |
Biomarker
|
disease |
BEFREE |
Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial.
|
20970382 |
2010 |
|
Parkinson Disease
|
0.100 |
Biomarker
|
disease |
BEFREE |
Neurturin (NTN) and glial cell line-derived neurotrophic factor have potent neuroprotective and neurorestorative effects on dopaminergic neurons, but their use in treating Parkinson's disease has been limited by significant delivery obstacles.
|
19349823 |
2009 |
|
Parkinson Disease
|
0.100 |
Biomarker
|
disease |
BEFREE |
In this phase I, open-label clinical trial, 12 patients aged 35-75 years with a diagnosis of PD for at least 5 years in accordance with the UK Brain Bank Criteria received bilateral, stereotactic, intraputaminal injections of adeno-associated virus serotype 2-neurturin (CERE-120).
|
18387850 |
2008 |
|
Parkinson Disease
|
0.100 |
Biomarker
|
disease |
BEFREE |
The mechanisms by which transgenic therapy treats PD might involve functional enhancement of residual dopaminergic neurons by NTN, which significantly reduces the number of rotations in animals, but not increase the numbers of existing dopaminergic neurons.
|
17336273 |
2007 |
|
Parkinson Disease
|
0.100 |
Biomarker
|
disease |
BEFREE |
Issues regarding gene therapy products for Parkinson's disease: the development of CERE-120 (AAV-NTN) as one reference point.
|
18267286 |
2007 |
|
Parkinson Disease
|
0.100 |
Biomarker
|
disease |
BEFREE |
CERE-120, an adeno-associated virus serotype 2 (AAV2)-based gene delivery vector encoding human NTN, is being developed as a potential therapeutic for PD.
|
17532642 |
2007 |
|
Parkinson Disease
|
0.100 |
Biomarker
|
disease |
BEFREE |
CERE-120 is an adeno-associated viral vector encoding NTN, developed for the treatment of PD.
|
17164776 |
2007 |
|
Abdominal Pain
|
0.100 |
Biomarker
|
phenotype |
HPO |
|
|
|
|
Diarrhea
|
0.100 |
Biomarker
|
phenotype |
HPO |
|
|
|
|
Dwarfism
|
0.100 |
Biomarker
|
disease |
HPO |
|
|
|
|
Sensorineural Hearing Loss (disorder)
|
0.100 |
Biomarker
|
disease |
HPO |
|
|
|
|
Intestinal Obstruction
|
0.100 |
Biomarker
|
disease |
HPO |
|
|
|
|
Nausea and vomiting
|
0.100 |
Biomarker
|
phenotype |
HPO |
|
|
|
|
Thyroid Neoplasm
|
0.100 |
Biomarker
|
disease |
HPO |
|
|
|
|
Sepsis
|
0.100 |
Biomarker
|
disease |
HPO |
|
|
|
|
Short stature
|
0.100 |
Biomarker
|
phenotype |
HPO |
|
|
|
|
Intestinal Polyposis
|
0.100 |
Biomarker
|
disease |
HPO |
|
|
|
|
Weight decreased
|
0.100 |
Biomarker
|
phenotype |
HPO |
|
|
|
|
Failure to thrive in infancy
|
0.100 |
Biomarker
|
phenotype |
HPO |
|
|
|
|
Short Stature, CTCAE
|
0.100 |
Biomarker
|
phenotype |
HPO |
|
|
|
|
Adducted thumb
|
0.100 |
Biomarker
|
phenotype |
HPO |
|
|
|