RNA - Genetherapys

SPOKE 3 - RNA-based Therapies for Neurodegeneration

Neurodegeneration is a chronic condition that damages and destroys the nervous system over time, which has become an extremely relevant health and socio-economic problem. Incurable diseases such as Alzheimer’s, Parkinson’s, and ALS (amyotrophic lateral sclerosis) pose an extremely important challenge to those in the world of pharmacological research. Other nervous system afflictions include genetic diseases such as Huntington’s disease, neurodevelopmental disorders, and some types of epilepsies, as well as acute stroke.

There are multiple biological causes for these diseases. For those with Alzheimer’s and Parkinson’s, specific groups of neurons accumulate proteins that form insoluble aggregates and stop functioning correctly. In the case of Huntington’s disease and neurodevelopmental disorders, the causes are genetic and linked to anomalies or rare mutations of specific genes.

Developing targeted therapies for neurodegeneration is additionally complicated as many patients experience long asymptomatic preclinical phases.  This silent biological process can last for years before demonstrating progressive neuronal damage, and once the disease becomes clinically evident, treatment may be too late. Additionally, drug administration is highly limited due to the Blood-Brain Barrier, which filters the blood that arrives in the brain and serves to protect the brain from factors that could damage it.

Led by the Italian Institute of Technology, Spoke 3 believes that RNA-based therapies represent a new virtuous strategy for the development of personalized drugs for these diseases. By exploiting the molecular versatility of RNAs, Spoke 3 knows that synthesizing them could offer an economically sustainable way to help a limited number of patients with a common genomic profile.

By identifying various mutations affecting diseases of the nervous system, Spoke 3 aims to identify causative target molecules of the disease in a homogeneous group of patients, which will lead to the development of therapeutic RNA molecules capable of reaching the altered areas of the brain.

Spoke 3 affiliates include the National Research Council of Italy, University of di Catanzaro, University of Bari Aldo Moro, University of Bologna, University of Florence, University of Milan, University of Naples Federico II, University of Padua, University of Pisa, Sapienza University of Rome, and University of Turin.

 

Written by

Laura De Cantis, Ellen Corcoran and Martina Corte De Checco

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