Friedreich's ataxia: a study of frataxin deficiency in cardiac myocytes. Author: Èlia Obis Monné, Director: Jordi Tamarit Sumalla, Joaquim Ros Salvador; Thesis, date of defense:2014-12-10, Universitat de Lleida. Departament de Ciències Mèdiques Bàsiques. Tesis Doctorals en Xarxa (Universitat de Lleida)
Full text files in this thesis will be available from 2015-12-10
Frataxin deficiency in cardiac myocytes causes an alteration of the mitochondrial network and oxidative stress. Furthermore, cardiac myocytes undergo a change in the metabolic profile and accumulate large amounts of fatty acids in lipid droplets.
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Thursday, July 23, 2015
RNA-based drugs, very promising research (RaNA Therapeutics)
RNA-based drugs, very promising research (RaNA Therapeutics). EXOME News, Ben Fidler July 23rd, 2015 and The Boston Globe (Bussines) Globe Correspondent
RaNA Therapeutics, a Cambridge-based drug startup, announced today will help fund the preclinical work needed to get at least two programs into human trials in 2017. So far the company has touted potential therapies for spinal muscular atrophy and Friedreich’s Ataxia. These therapies are RNA-based drugs meant to switch back on genes that are silenced in certain diseases, and thus don’t produce critical proteins (like spinal motor neuron in the case of people with SMA, and frataxin for those with Friedreich’s). (EXOME)
The company said Thursday that it raised the funds from a group of new and existing investors led by MRL Ventures, an arm of the drug giant Merck, and the Baupost Group. RaNA plans to have one or two treatments for spinal muscular atrophy and Friedreich’s ataxia in clinical trials by 2017. (The Boston Globe)
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RaNA Therapeutics, a Cambridge-based drug startup, announced today will help fund the preclinical work needed to get at least two programs into human trials in 2017. So far the company has touted potential therapies for spinal muscular atrophy and Friedreich’s Ataxia. These therapies are RNA-based drugs meant to switch back on genes that are silenced in certain diseases, and thus don’t produce critical proteins (like spinal motor neuron in the case of people with SMA, and frataxin for those with Friedreich’s). (EXOME)
The company said Thursday that it raised the funds from a group of new and existing investors led by MRL Ventures, an arm of the drug giant Merck, and the Baupost Group. RaNA plans to have one or two treatments for spinal muscular atrophy and Friedreich’s ataxia in clinical trials by 2017. (The Boston Globe)
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