The symposium will take place on November 13 and 14.
Further information can be obtained from the Continuing Medical Education Department at the Children’s Hospital of Philadelphia at (215) 590 5263.
Wednesday, September 9, 2009
Elucidation of the mechanism of mitochondrial iron loading in Friedreich's ataxia by analysis of a mouse mutant
Proceedings of the National Academy of Science USA
Edited by Solomon H. Snyder, Johns Hopkins University School of Medicine, Baltimore, MD, and approved July 30, 2009 (received for review June 17, 2009)
Michael Li-Hsuan Huanga, Erika M. Beckera, Megan Whitnalla, Yohan Suryo Rahmantoa, Prem Ponkab,1 and Des R. Richardson.
Keywords: muscle creatine kinase (MCK), frataxin knockout mouse, frataxin deficiency, iron metabolism, neurologic and cardiologic degeneration, iron-sulfur cluster (ISC) synthesis, cysteine desulferase Nfs1, mitochondrial ferritin, 5-aminolevulinate dehydratase, coproporphyrinogen oxidase, hydroxymethylbilane synthase, uroporphyrinogen III synthase, ferrochelatase, Sec15l1, mitoferrin-2 (Mfrn2).
Edited by Solomon H. Snyder, Johns Hopkins University School of Medicine, Baltimore, MD, and approved July 30, 2009 (received for review June 17, 2009)
Michael Li-Hsuan Huanga, Erika M. Beckera, Megan Whitnalla, Yohan Suryo Rahmantoa, Prem Ponkab,1 and Des R. Richardson.
Keywords: muscle creatine kinase (MCK), frataxin knockout mouse, frataxin deficiency, iron metabolism, neurologic and cardiologic degeneration, iron-sulfur cluster (ISC) synthesis, cysteine desulferase Nfs1, mitochondrial ferritin, 5-aminolevulinate dehydratase, coproporphyrinogen oxidase, hydroxymethylbilane synthase, uroporphyrinogen III synthase, ferrochelatase, Sec15l1, mitoferrin-2 (Mfrn2).
Saturday, September 5, 2009
The roles of SbcCD and RNaseE in the transcription of GAA·TTC repeats in Escherichia coli
Friday, September 4, 2009
The dorsal root ganglion in Friedreich's ataxia.
Acta Neuropathol. 2009 Aug 30. [Epub ahead of print]
Koeppen AH, Morral JA, Davis AN, Qian J, Petrocine SV, Knutson MD, Gibson WM, Cusack MJ, Li D.
Research Service (151), Veterans Affairs Medical Center, 113 Holland Ave, Albany, NY, 12208, USA, arnulf.koeppen@med.va.gov.
Keywords: Atrophy of dorsal root ganglia (DRG), thinning of dorsal roots (DR), Friedreich's ataxia (FRDA), selective vulnerability of larger neurons in DRG, selective vulnerability of thicker myelinated DR axons, ventral roots (VR), Schwann cells, laminin, ferritin, mitochondrial ferritin, ferroportin.
Koeppen AH, Morral JA, Davis AN, Qian J, Petrocine SV, Knutson MD, Gibson WM, Cusack MJ, Li D.
Research Service (151), Veterans Affairs Medical Center, 113 Holland Ave, Albany, NY, 12208, USA, arnulf.koeppen@med.va.gov.
Keywords: Atrophy of dorsal root ganglia (DRG), thinning of dorsal roots (DR), Friedreich's ataxia (FRDA), selective vulnerability of larger neurons in DRG, selective vulnerability of thicker myelinated DR axons, ventral roots (VR), Schwann cells, laminin, ferritin, mitochondrial ferritin, ferroportin.
Thursday, September 3, 2009
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.
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.....
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 ....
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 ....
Wednesday, September 2, 2009
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
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
Tuesday, September 1, 2009
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
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"
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"
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