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Author
Chillón Bosch, Guillem
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Abstract
Amyotrophic lateral sclerosis (ALS) is one of the most devastating disorders affecting the function of nerves and muscles, with an estimated 5,000 new cases diagnosed each year. ALS is the most common adult motor neuron (MN) disease leading to paralysis and death in 2-5 years after disease onset. Despite the wide variety of research involved in it, there is no cure for ALS.
Gene therapy, currently represent one of the most important areas in untreatable diseases such as in ALS. This therapy uses genetic material to act as a therapeutic agent for delivering therapeutic genes or silencing specific genes involved in MN degradation. Understanding the mode of action of the therapy could give us hints of its efficacy, efficiency, and safety.
Here I have focused on the histopathological study of an ALS animal model to analyze how the treatment acts to cells of interest. The challenge of this project focuses on optimizing the immunostaining of different biological markers of ALS both in brain and spinal cord tissues and acquire preliminary data of the potency of our treatment.
For this, I have studied several variables for each individual immunohistochemistry study: 1) the localization of histologic sections; 2) the dilution range of the antibodies used; and 3) the type or composition of the dilution buffers. Specifically, tissue from an ALS-like symptomatic mouse strain has been collected, cryosectioned, and tested with seven different specific an-tibodies.
With the present work, the treatment’s efficiency has been analyzed in a micro-level for the first time, protocols have been optimized for future work and preliminary data showing a re-duction of ALS proteinopathy have been obtained in our mouse model.
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