TR3-05471: iPSC-derived Friedreich's ataxia

TR3-05471: iPSC-derived Friedreich's ataxia. CIRN, California Institute for Regenerative Medecine.
Recommendation: Not Recommended for Funding Scientific Score: --Total Funds Requested $6,521,212 Award Type : Development Candidate Award.

Not all are good news

Ultrasound-guided nerve blocks in the Charcot–Marie–Tooth disease and Friedreich's ataxia

Ultrasound-guided nerve blocks in the Charcot–Marie–Tooth disease and Friedreich's ataxia. J. B. Barbary, F. Remérand, J. Brilhault, M. Laffon J. Fusciardi. Br. J. Anaesth. (2012) 108 (6): 1042-1043. doi: 10.1093/bja/aes160

Keywords: Peripheral nerve blocks (PNBs), neurological disease, electrical stimulation, ultrasound-guided PNBs, neurofibromatosis, hypertrophic peripheral neuropathy [Charcot–Marie–Tooth disease type 1 (CMTD1)], Friedreich's ataxia (FRA).

Resveratrol Protects Cortical Neurons against Microglia-mediated Neuroinflammation

Resveratrol Protects Cortical Neurons against Microglia-mediated Neuroinflammation, Zhang, F., Wang, H., Wu, Q., Lu, Y., Nie, J., Xie, X. and Shi, J. (2012), Phytother. Res.. doi: 10.1002/ptr.4734

 Keywords: resveratrol, neuroprotection, neuroinflammation, microglial activation, neurological disorders.

  I would like to stress that currently in Australia is running a clinical trial in FA patients, we are looking forward to its findings

TALE proteins induced the expression of the frataxin gene.

TALE proteins induced the expression of the frataxin gene. Prof. Jacques P Tremblay, Mr. Pierre Chapdelaine, Zoé Coulombe, and Joël Rousseau. Human Gene Therapy. -Not available-, ahead of print. doi:10.1089/hum.2012.034.

 The TALEFrat increased by 2 to 3 folds the frataxin gene expression and potentially could alleviate the symptoms of Friedreich ataxia.

Keywords: Tal effector proteins(TALE), transcription activator, expression of a gene, Friedreich ataxia (FRDA), frataxin, TALE genes (TALEFrat), VP16 peptides, mCherry reporter gene

Worldwide status of clinical experimentation with stem cells in neurologic diseases

Worldwide status of clinical experimentation with stem cells in neurologic diseases. Dimitrios Karussis, MD, PhD. Neurology April 24, 2012 78:1334-1336.

KEYWORDS: stem cells, CNS repair, Embryonic stem cells (ESC), pluripotent, potential carcinomatous transformation, somatic stem cells, neuronal (NSC), mesenchymal stem cells (MSC), Fetal NSC, ethical, legislative, immunomodulatory effects, neurotrophic factors, bone marrow, blood–brain barrier, animal models of neurologic disorders.

Friedreich's Ataxia Causes Redistribution of Iron, Copper, and Zinc in the Dentate Nucleus.

Friedreich's Ataxia Causes Redistribution of Iron, Copper, and Zinc in the Dentate Nucleus. Koeppen AH, Ramirez RL, Yu D, Collins SE, Qian J, Parsons PJ, Yang KX, Chen Z, Mazurkiewicz JE, Feustel PJ.. Cerebellum. 2012 May 5. [Epub ahead of print].

 Keywords:Friedreich's ataxia (FRDA), large neurons, dentate nucleus (DN), iron (Fe), copper (Cu), zinc (Zn), quantitative X-ray fluorescence (XRF), hilar white matter, DN gray matter, oligodendroglia.

Spinal Cord Atrophy Correlates with Disability in Friedreich's Ataxia.

Spinal Cord Atrophy Correlates with Disability in Friedreich's Ataxia. Chevis CF, da Silva CB, D'Abreu A, Lopes-Cendes I, Cendes F, Bergo FP, França MC. Cerebellum 2012 May 5.

Keywords: Friedreich's ataxia, spinal cord atrophy, spinal cord area, eccentricity at C2/C3 level, Friedreich ataxia rating scale (FARS).

What the Orphan Drug Act Has Done Lately for Children With Rare Diseases: A 10-Year Analysis

What the Orphan Drug Act Has Done Lately for Children With Rare Diseases: A 10-Year Analysis. Chandana Thorat, BTech, Kui Xu, MD, PhD, Scott N. Freeman, PhD, Renan A. Bonnel, PharmD, MPH, Francesca Joseph, MD, M. Ian Phillips, PhD, DSc, Menfo A. Imoisili, MD, MPH. Pediatrics Vol. 129 No. 3 March 1, 2012. pp. 516 -521 (doi: 10.1542/peds.2011-1798)

KEYWORDS: orphan drugs, rare diseases, Orphan Drug Act, pediatrics

Induced neural stem cells: a new tool for studying neural development and neurological disorders

Induced neural stem cells: a new tool for studying neural development and neurological disorders. Cell Research advance online publication 1 May 2012; doi: 10.1038/cr.2012.73, Guang-Hui Liu, Fei Yi, Keiichiro Suzuki, Jing Qu, Juan Carlos Izpisua Belmonte.
National Laboratory of Biomacromolecules, Institute of Biophysics, Chinese Academy of Sciences, Beijing China. Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, CA 92037, USA. Center for Regenerative Medicine in Barcelona, 08003 Barcelona, Spain.

Recent advances in the generation of multipotent and expandable induced neural stem cells are exciting. They not only hold great promises for potential clinical applications but may also open up a new era for neural stem cell research in the near future.

alpha-Tocotrienol quinone modulates oxidative stress response and the biochemistry of aging. Bioorganic & Medicinal Chemistry Letters 21 (2011) 3693–3698, William D. Shrader, Akiko Amagata, Adam Barnes, Gregory M. Enns, Andrew Hinman, Orion Jankowski , Viktoria Kheifets, Ryo Komatsuzaki, Edgar Lee, Paul Mollard, Katsuyuki Murase, Alfredo A. Sadun, Martin Thoolen, Kieron Wesson, Guy Miller.

Keywords: Redox, Antioxidants, Quinone, Aging, Digital Biology, frataxin, Friedreich's Ataxia.