AAV9-mediated erythropoietin gene delivery into the brain protects nigral dopaminergic neurons in a rat model of Parkinson's disease

Gene Therapy, advance online publication 3 September 2009; doi: 10.1038/gt.2009.113

Y-Q Xue1,7, B-F Ma1,7, L-R Zhao1,2,3, J B Tatom4, B Li2, L-X Jiang5, R L Klein3,4 and W-M Duan1,3,6
1Department of Cellular Biology and Anatomy, Louisiana State University Health Sciences Center, Shreveport, LA, USA
2Department of Neurology, Louisiana State University Health Sciences Center, Shreveport, LA, USA
3Gene Therapy Program, Louisiana State University Health Sciences Center, Shreveport, LA, USA
4Department of Pharmacology, Toxicology and Neuroscience, Louisiana State University Health Sciences Center, Shreveport, LA, USA
5Vector Gene Technology Company LTD, Beijing, PR China
6Department of Anatomy and Neurobiology, Capital Medical University, Beijing, PR China
Correspondence: Dr W-M Duan, Department of Cellular Biology & Anatomy, LSU Health Sciences Center in Shreveport, 1501 Kings Highway, Shreveport, LA 71130, USA. E-mail: wduan@lsuhsc.edu
7These authors contributed equally to this work.
Received 17 March 2009; Revised 3 August 2009; Accepted 5 August 2009; Published online 3 September 2009.

Promise Of Nanodiamonds For Safer Gene Therapy

Article Date: 03 Sep 2009 - 0:00 PDT
Medical News Today
Gene therapy holds promise in the treatment of a myriad of diseases, including cancer, heart disease and diabetes, among many others. However, developing a scalable system for delivering genes to cells both efficiently and safely has been challenging.
Read more.....

China cracks down on stem cell tourism

NewScientist

00:01 03 September 2009 by Andy Coghlan


Chinese and European researchers have today published ethical guidelines aimed at discouraging Chinese doctors from offering patients unproven or sham treatments based on stem cells.
The authors hope the move will reinforce legal curbs on stem cell treatments introduced on 1 May by China's ministry of health.



Read more ....

Neuroprotective effects of compounds with antioxidant and anti-inflammatory properties in a Drosophila model of Parkinson's disease

OPEN ACCESS

Katharina Faust , Stephan Gehrke , Yufeng Yang , Lichuan Yang , Flint Beal and Bingwei Lu
BMC Neuroscience 2009, 10:109doi:10.1186/1471-2202-10-109
Published: 1 September 2009

Abstract (provisional)

Background
Parkinson's disease (PD) is the most common movement disorder. Extrapyramidal motor symptoms stem from the degeneration of the dopaminergic pathways in patient brain. Current treatments for PD are symptomatic, alleviating disease symptoms without reversing or retarding disease progression. Although the cause of PD remains unknown, several pathogenic factors have been identified, which cause dopaminergic neuron (DN) death in the substantia nigra (SN). These include oxidative stress, mitochondrial dysfunction, inflammation and excitotoxicity. Manipulation of these factors may allow the development of disease-modifying treatment strategies to slow neuronal death. Inhibition of DJ-1A, the Drosophila homologue of the familial PD gene DJ-1, leads to oxidative stress, mitochondrial dysfunction, and DN loss, making fly DJ-1A model an excellent in vivo system to test for compounds with therapeutic potential.
Results
In the present study, a Drosophila DJ-1A model of PD was used to test potential neuroprotective drugs. The drugs applied are the Chinese herb celastrol, the antibiotic minocycline, the bioenergetic amine coenzyme Q10 (coQ10), and the glutamate antagonist 2,3-dihydroxy-6-nitro-7-sulphamoylbenzo[f]-quinoxaline (NBQX). All of these drugs target pathogenic processes implicated in PD, thus constitute mechanism-based treatment strategies. We show that celastrol and minocycline, both having antioxidant and anti-inflammatory properties, confer potent dopaminergic neuroprotection in Drosophila DJ-1A model, while coQ10 shows no protective effect. NBQX exerts differential effects on cell survival and brain dopamine content: it protects against DN loss but fails to restore brain dopamine level.
Conclusions
The present study further validates Drosophila as a valuable model for preclinical testing of drugs with therapeutic potential for neurodegenerative diseases. The lower cost and amenability to high throughput testing make Drosophila PD models effective in vivo tools for screening novel therapeutic compounds. If our findings can be further validated in mammalian PD models, they would implicate drugs combining antioxidant and anti-inflammatory properties as strong therapeutic candidates for mechanism-based PD treatment.


Full text: http://www.biomedcentral.com/content/pdf/1471-2202-10-109.pdf

Finding The ZIP-code For Gene Therapy: Scientists Imitate Viruses To Deliver Therapeutic Genes

ScienceDaily (Aug. 31, 2009) — A research report featured on the cover of the September 2009 print issue of The FASEB Journal describes how Australian scientists developed a new gene therapy vector that uses the same machinery that viruses use to transport their cargo into our cells. As a result of this achievement, therapeutic DNA can be transferred to a cell's nucleus far more efficiently than in the past, raising hopes for more effective treatment of genetic disorders and some types of cancers. Read more ....

Original scientific source: Multifunctional protein nanocarriers for targeted nuclear gene delivery in nondividing cells. Dominic J. Glover, Su May Ng, Adam Mechler, Lisandra L. Martin, and David A. Jans

Nicotinic receptors: allosteric transitions and therapeutic targets in the nervous system

Nature Reviews Drug Discovery 8, 733-750 (September 2009) doi:10.1038/nrd2927
Antoine Taly1, Pierre-Jean Corringer2, Denis Guedin3, Pierre Lestage3 & Jean-Pierre Changeux


Laboratoire de Chimie Biophysique, Institut de Science et d'Ingénierie Supramoléculaires, UMR 7006 (CNRS-Université de Strasbourg), 8 Allée Gaspard Monge, 67000 Strasbourg, France. Email: a.taly@isis.u-strasbg.fr
Channel Receptors, CNRS URA 2182, Institut Pasteur, 25 rue du Dr Roux, 75015 Paris, France. Email: pjcorrin@pasteur.fr
Institut de Recherches Servier, 125 Chemin de Ronde, 78290 Croissy-sur-Seine. Email: denis.guedin@fr.netgrs.com; Email: pierre.lestage@fr.netgrs.com
CNRS URA 2182, Institut Pasteur, 25 rue du Dr Roux, 75015 Paris, France. Email: changeux@pasteur.fr

Highlight: "Varenicline (Chantix or Champix) is a nicotinic receptor partial agonist"

Excess iron and Friedreich’s ataxia

LifeScientist

Graeme O'Neill 28/08/2008 13:40:00


While Friedreich’s ataxia is a rare disorder, tracking down the role of the protein implicated in the disease has opened up new therapeutic potentials.

Keywords: free radicals, Friedreich's ataxia, Martin Delatycki, Murdoch Childrens Research Institute, iron chelators, Australia, antioxidants, clinical trials, Friedreich’s Ataxia Research Association (Australasia).

What Is Ataxia? What Causes Ataxia?

Article Date: 31 Aug 2009 - 0:00 PDT
Medical News Today .

Journalistic article written by Christian Nordqvist, make a simple revision of the ataxias, but fine.

MRI methods can show bone marrow stem cells’ viability as brain-repairing therapy

Stem Cell Research Blog, 20 Aug 2009

WASHINGTON - Researchers at Tel Aviv University have offered new hope for people with incurable neurodegenerative diseases like Huntington’s, Alzheimer’s, and Parkinson’s by showing that the viability of stem cells created from a patient’s own bone marrow can be determined using MRI tracking methods.

The role of iron in mitochondrial function.

Biochim Biophys Acta. 2009 Jul;1790(7):629-36. Epub 2008 Oct 7.

Levi S, Rovida E.Vita-Salute San Raffaele University, Via Olgettina 58, 20132, Milano, Italy. levi.sonia@hsr.it