Breaking bad: R-loops and genome integrity

Breaking bad: R-loops and genome integrity. Julie Sollier, Karlene A. Cimprich, Trends in Cell Biology, Available online 1 June 2015, ISSN 0962-8924, http://dx.doi.org/10.1016/j.tcb.2015.05.003.

How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration

How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration. Pat Furlong, John F. P. Bridges, Lawrence Charnas, Justin R. Fallon, Ryan Fischer, Kevin M. Flanigan, Timothy R. Franson, Neera Gulati, Craig McDonald, Holly Peay and H. Lee Sweeney; Orphanet Journal of Rare Diseases 2015, 10:82 doi:10.1186/s13023-015-0281-2 Published: 24 June 2015

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Transcription blockage by stable H-DNA analogs in vitro

Transcription blockage by stable H-DNA analogs in vitro. Shristi Pandey, Anna M. Ogloblina, Boris P. Belotserkovskii, Nina G. Dolinnaya, Marianna G. Yakubovskaya, Sergei M. Mirkin and Philip C. Hanawalt, Nucl. Acids Res. (2015) doi: 10.1093/nar/gkv622 First published online: June 22, 2015

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Formation of H-DNA has been implicated in transcription blockage by expanded (GAA)n repeats responsible for the human genetic disease Friedreich's ataxia

TAT-MTS-FXN Protein as Replacement Therapy for Friedreich’s Ataxia

TAT-MTS-FXN Protein as Replacement Therapy for Friedreich’s Ataxia, Dalia Megiddo, MD, Chief Development Officer, Hagar Greif, PhD, Senior Director of Pre Clinical Studies.

Cumulative data highly support TAT-MTS(cs)-Frataxin as Protein Replacement Therapeutic Candidatefor Friedreich’s Ataxia

Patent: FRATAXIN ENHANCER

Patent: FRATAXIN ENHANCER Original document: WO2015064447 (A1) ― 2015-05-07 Chibazakura, Taku; Tanaka, Tohru; Abe, Fuminori; Nakajima, Motowo; Kamiya, Atsuko; Haga, Naomi; Takahashi Kiwamu. Tokyo University Of Agriculture; Sbi Pharmaceuticals Co. Ltd - 2015-05-07 (full text in Japanese)

The purpose of the invention is to provide a drug effective in the treatment and prevention of diseases and the like caused by a decrease in frataxin production. [Solution] The invention provides a frataxin enhancer containing 5-aminolevulinic acid (ALA) or a derivative thereof or a salt of these, and a therapeutic agent and/or prophylactic agent for diseases caused by a decrease in frataxin production.