Friday, February 26, 2016

Targeted RNA or BDNF gene transfer protects against frataxin deficiency

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Ian Fyfe, Research Highlight: Nature Reviews Neurology (2016), doi:10.1038/nrneurol.2016.19, Published online 26 February 2016 In two rece...
Thursday, February 25, 2016

Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy?

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Randy J Chandler, Matthew C LaFave, Gaurav K Varshney, Shawn M Burgess and Charles P Venditti; (Nature) Molecular Therapy 24, 198-201 (Febru...

MicroRNAs Form Triplexes with Double Stranded DNA at Sequence-Specific Binding Sites; a Eukaryotic Mechanism via which microRNAs Could Directly Alter Gene Expression

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Paugh SW, Coss DR, Bao J, Laudermilk LT, Grace CR, Ferreira AM, M. Brett Waddell, Granger Ridout, Deanna Naeve, Michael Leuze, Philip F. LoC...
Wednesday, February 24, 2016

Friedreich ataxia is not only a GAA repeats expansion disorder: implications for molecular testing and counselling

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Dorota Hoffman-Zacharska , Tomasz Mazurczak, Tomasz Zajkowski, Renata Tataj, Paulina Górka-Skoczylas, Katarzyna Połatyńska, Łukasz Kępczyńsk...

Combined Cerebellar Proton MR Spectroscopy and DWI Study of Patients with Friedreich’s Ataxia

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Laura Ludovica Gramegna, Caterina Tonon , David Neil Manners, Antonella Pini, Rita Rinaldi, Stefano Zanigni, Claudio Bianchini, Stefania Eva...
Tuesday, February 23, 2016

CNS Drug Delivery: Beyond the Spinal Cord

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Recently the intrathecal administration has been proposed as part of a hopeful therapy for FA ( Intrathecal delivery of frataxin mRNA encaps...

Retrotope Advances RT001 in Clinical Trials to Treat Friedreich's ataxia

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(Ref: Marketwired) February 22nd, 2016 LOS ALTOS, CA--(Marketwired) - Retrotope, a privately held clinical stage pharmaceutical company, t...
Monday, February 22, 2016

Long-term effect of epoetin alfa on clinical and biochemical markers in friedreich ataxia

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Saccà, F., Puorro, G., Marsili, A., Antenora, A., Pane, C., Casali, C., Marcotulli, C., Defazio, G., Liuzzi, D., Tatillo, C., Cambriglia, D....
Sunday, February 21, 2016

Reversal of epigenetic promoter silencing in Friedreich ataxia by a class I histone deacetylase inhibitor

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Yogesh K. Chutake, Christina C. Lam, Whitney N. Costello, Michael P. Anderson and Sanjay I. Bidichandani; Nucl. Acids Res. (2016) doi: 10.10...
Saturday, February 20, 2016

UNC gene therapy spinout Bamboo Therapeutics raises $49.5M Series A

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Startups, Biotech, By Meghana Keshavan http://medcitynews.com. The startup’s developing gene therapies for rare neurologic diseases, which...