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Latest news on Friedreich's Ataxia research
Updated results from clinical trials and research initiatives
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Results: We have engineered 12 TALE genes (TALEFrat) coding for TALEFrat proteins each specifically targeting different 14 bp DNA sequences within the proximal region of the human frataxin promoter. When the genes of these TALEFrat were fused with a transcription activator, i.e., four VP16 peptides (i.e., VP64), the resulting TALEFrat-VP64 proteins induced the expression of a mCherry reporter gene fused to a mini-CMV promoter able to be activated by the insertion of the frataxin proximal promoter upstream to the mini promoter. These TALEFrat-VP64 also increased by 2 to 3 folds the frataxin gene expression (detected by qRT-PCR) in the cells. Conclusion: TALEFrat proteins targeting the frataxin promoter are thus a method to increase the expression of frataxin mRNA and potentially could alleviate the symptoms of Friedreich ataxia. This new methodology of TALE effector opens a new field, which could be used to develop TALE proteins to treat other diseases by inducing the expression of specific genes.
If you feel like reading an unputdownable novel while collaborating with a just and solidary cause, "The Legacy of Marie Schlau" is your book! 100% of all funds raised will be dedicated to medical research to find a cure for Friedreich's Ataxia, a neurodegenerative disease that affects mostly young people, shortening their life expectancy and confining them to a wheelchair.
The life of Marie Schlau, a German Jewish girl born in 1833 hides great unsolved mysteries: accidents, disappearances, enigmas, unknown diagnoses, disturbing murders, love, tenderness, greed, lies, death ... alternatively a different story unfolds every time and takes us closer to the present. Thus, there are two parallel stories unravelling, each in a different age and place, which surprisingly converge in a revelatory chapter.
Paperback and Kindle versions for "The legacy of Marie Schlau" available for sale at Amazon now!
Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:
1) Gene Therapy for Friedreich's Ataxia research project:
The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.
2) Frataxin delivery research project:
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.
The study is headed by Ernest Giralt, head of the Peptides and Proteins Lab, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles—molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients’ associations in 2013*, it has been developing another two projects into Friedrich’s Ataxia.