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.