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.

GAA triplet-repeats cause nucleosome depletion in the human genome

GAA triplet-repeats cause nucleosome depletion in the human genome. Hongyu Zhao, Yongqiang Xing, Guoqing Liu, Ping Chen, Xiujuan Zhao, Guohong Li, Lu Cai, Available online 20 June 2015, ISSN 0888-7543, http://dx.doi.org/10.1016/j.ygeno.2015.06.010.

Oxidative stress in inherited mitochondrial diseases

Oxidative stress in inherited mitochondrial diseases. Genki Hayashi, Gino Cortopassi, Free Radical Biology and Medicine, Available online 12 June 2015, ISSN 0891-5849, http://dx.doi.org/10.1016/j.freeradbiomed.2015.05.039.

Cardioprotective HIF-1α-frataxin signaling against ischemia-reperfusion injury.

Cardioprotective HIF-1α-frataxin signaling against ischemia-reperfusion injury. Gayani Nanayakkara , Abdullah Alasmari , Shravanti Mouli , Haitham Eldoumani , John C. Quindry , Graham McGinnis , Xiaoyu Fu , Avery Berlin , Bridget Peters , Juming Zhong , Rajesh H. Amin, American Journal of Physiology - Heart and Circulatory Physiology Published 12 June 2015 Vol. no. , DOI: 10.1152/ajpheart.00875.2014

we postulated that HIF-1α transcriptionally regulates frataxin expression in response to hypoxia

Molecular and clinical study of a cohort of 110 Algerian patients with autosomal recessive ataxia

Molecular and clinical study of a cohort of 110 Algerian patients with autosomal recessive ataxia. Wahiba Hamza, Lamia Ali Pacha, Tarik Hamadouche, Jean Muller, Nathalie Drouot, Farida Ferrat, Samira Makri, Malika Chaouch, Meriem Tazir, Michel Koenig and Traki Benhassine; BMC Medical Genetics 2015, 16:36 doi:10.1186/s12881-015-0180-3

OPEN ACCESS, Full text Pdf