Cell reprogramming for the treatment of multiple sclerosis

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Minimum 25.000 euros
 
 
 
2.665 out of 100.000 eurosRemaining 61 days

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Personalised thank-you letter

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Personalised thank-you letter + UCAM t-shirt (only for shipments within the Iberian Peninsula)

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Personalised thank-you letter + UCAM t-shirt + guided tour of the Los Jerónimos Monastery, UCAM’s seat, with distribution of a sports bag and a UCAM sweatshirt.

In the world, more than 2.5 million people suffer from Multiple Sclerosis. In Spain, more than 50,000 people suffer from it.

The project
Who is behind

What is our goal?

Multiple Sclerosis (MS) is a chronic inflammatory and autoimmune neurodegenerative disease characterised by the emergence of demyelinating lesions and of axonal damage in the central nervous system. It is the most frequent cause of non-traumatic neurological disability among young adults, mostly women.  70% of cases are diagnosed between 20 and 40 years old, therefore the impact of this disease in the personal, work and family life of these patients is very high.

Its natural evolution entails serious neurological deficits: loss of the movement, coordination, sensitivity, balance, vision, bowel control... which implies a gradual increase in the disability level and a progressive deterioration of the life quality. Although its cause is unknown, all the clues indicate that it occurs in people who are genetically predisposed on whom any environmental factor, that has not been identified yet, acts.

The main goal of this project is to obtain a treatment for the regeneration of the central nervous system in patients with Multiple Sclerosis. We will try to identify the factors related to age that act as genetic regulators in the repair of the myelin sheaths and in the neurodegeneration in the central nervous system. In this way, those who suffer from Multiple Sclerosis can have their damaged nervous cells repaired, prevent damages to the healthy cells and, therefore, improve their quality of life and maybe recover the lost functions.

To reach this goal we will develop a simple and reproducible experimental neuronal model with the different phases of the development of the disease, starting from induced pluripotent stem cells (iPSCs) (adult cells genetically reprogrammed to reach similar state to stem cells). We will obtain iPSCs from somatic cells of patients of different ages with Multiple Sclerosis, which will allow us to understand the disease evolution with aging and to test the effectiveness of experimental drugs and other advanced therapies avoiding the use of animals in the early stage of the experimentation.

Who will benefit from our project?

This project will benefit all patients diagnosed with Multiple Sclerosis who currently are more than 2.5 million in the world. In Spain, the prevalence of Multiple Sclerosis is medium-high (between 100 and 150 cases for each 100,000 inhabitants), having an approximate incidence of 1,800 new diagnosed cases per year, with a current total number of affected people of 55,000 (75% are women). Globally, the number of affected people doubled throughout the last two decades. This increase in the prevalence of the disease mainly occurs among women and it seems to be linked to environmental factors.

What will you achieve with your donation?

If we achieve the minimum goal (25,000 €) we will develop various cell lines compatible with different phases of the development of the disease. To do so, we will use iPSCs cells obtained from somatic cells from patients of different ages and in different phases of the disease affected by multiple sclerosis. In this way, we will be able to understand how this pathology progresses as the patient ages. We will establish a simple and replicable cell model to test drugs and other types of advanced therapies, thus avoiding the use of animals in the first phases of the experiment.

If we obtain 50,000 €, we will use these cell lines to determine, through the CRISPR/Cas9 technology (which received the Nobel Prize in Chemistry in 2020), together with the last RNA sequencing techniques, the role played by factors related to age in the myelinization of the neurons’ axons and, therefore, in the neurodegenerative processes in the advance of Multiple Sclerosis with ageing.

Lastly, in case of reaching the optimum goal (100,000 €), a global experimental approach will be carried out to reach a treatment that leads to the regeneration of the central nervous system in patients with Multiple Sclerosis, starting from the results obtained in the previous objectives.

Do you want more information?

The cell reprogramming and gene editing technologies (CRISPR), that will be used in this project to obtain a treatment for Multiple Sclerosis, have been widely used by the research team to study other diseases such as retinitis pigmentosa, progeria (an extremely rare genetic disease of childhood that causes sudden and premature ageing in children between their first and second year of life), muscular dystrophy or acute kidney disease, with very positive and encouraging results in animal models. The results of these projects have been published in the most prestigious scientific journals and have also been echoed by the most important generalist media in the world.

However, it is necessary to develop many additional studies to improve these techniques which, based on the results obtained so far, emerge as the most promising future for medicine to treat certain diseases, giving hope to many families.

Dr. Izpisúa’s experience in the field of regenerative medicine and the use of cell reprogramming and gene editing technologies attests to the success of this project. He is one of the most important scientists worldwide in the field of developmental biology. TIME magazine included him in the list of the 50 most influential people of 2018 in the field of health care for his scientific innovations to address the problem of the shortage of human organs for transplants. He is the only Spanish including in this distinguished group.

Abaout Precipita
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