The grant awarded by the Simone and Cino Del Duca Foundation is also intended to support the research project proposed by the laureate and his team. This project aims to improve the remarkable gene transfer method used, and apply it to two further cerebral pathologies: Alzheimer’s disease and Friedreich’s cerebellar ataxia.
- In Alzheimer’s patients, the research focuses on the correction of cholesterol anomalies, as conventional cholesterol-lowering agents do not work on the brain. The aim is to use a suitable vector to introduce into the brain the coding gene for "cholesterol-24-hydroxylase", an enzyme which participates in the elimination of cholesterol from this organ.
- In patients suffering from Friedreich’s ataxia, the study aims to introduce the functional gene of frataxine, a protein essential to the function of the cerebellum, found in precise cellular organelles: mitochondria. A lack of frataxine in these organelles leads to disturbance of a structure which is essential to their healthy functioning, and later an accumulation of iron. Patrick Aubourg’s team are testing two new viral vectors for gene therapy to determine which, after, intra-venous injection, is most conducive to frataxine expression not only in cerebellum cells but also in the heart and in spinal marrow.

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The legacy of Marie Schlau: literature to help cure Friedreich's Ataxia

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!



Research projects currently being financed by BabelFAmily

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.



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