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This site is dedicated to scientific community working on ALS. Our aim is to optimize researchers time and efforts by providing updated, well organized information on novel findings, available resources and research support.
AriSLA - The Foundation for research on ALS - has been set up to make ALS research investments more effective and efficient, to speed up the clinical research impact e and to provide patients with better care, improved conditions and life expectancy. Its aim is to boost Italian excellencies in basic, clinical and technological research. The Foundation founders are Fondazione Cariplo, Fondazione Telethon, Fondazione Vialli and Mauro and AISLA.

 

 

New article published on Scientific Reports by AriSLA Funded Projects ALSsiMO "Morpholino antisense oligomer against SOD1 for the development of ALS therapy"

Antisense oligonucleotides Morpholino therapy rescues ALS pathological effects in human and murine ALS models

 

 

 

Molecular therapy with Antisense Oligonucleotides Morpholino can improve ALS pathology in two experimental models. This important result has been published in the scientific international journal Scientific Reports.

It is a study performed by Monica Nizzardo e Stefania Corti from Centro Dino Ferrari, University of Milan, IRCCS Fondazione Ca' Granda, Ospedale Maggiore Policlinico.

 

Milan, 22 February 2016

In a study published online in Scientific Reports, researchers at Centro Dino Ferrari, University of Milan, IRCCS Fondazione Ca' Granda, Ospedale Maggiore Policlinico reported the therapeutic efficacy of antisense oligonucleotides morpholino in two experimental models of Amyotrophic Lateral Sclerosis (ALS).

ALS is a fatal neurological disease characterized by the degeneration and loss of upper and lower motor neurons, which lead to paralysis and death within 3-5 years of diagnosis. The majority of cases of ALS have no clear genetic linkage and are referred to as sporadic, while 10% of cases are familial. Mutations in the gene encoding for Cu/Zn superoxide dismutase 1 (SOD1) are relatively frequent, accounting for 20% of sporadic ALS and causing a toxic accumulation of the SOD1 protein. Currently, there is no effective treatment for this disease.

The principal investigator of the research is Prof. Corti, MD, PhD, associate professor of neurology, chief of the Neural Stem Cell Laboratory in the Neuroscience Section, Department of Pathophysiology and Transplantation, University of Milan.

Dr. Monica Nizzardo, the first of author of the paper, and colleagues demonstrated the feasibility and the therapeutic potential of silencing SOD1 protein expression in human ALS cells and in a ALS murine model. The researchers used an analogue of DNA, morpholino oligonucleotide, that inhibits SOD1 protein production and has a particularly excellent safety, distribution and efficacy profile. This strategy has been tested in a human model in culture, specifically in motor neurons (the cell-type affected in ALS) derived from induced pluripotent stem cells, obtained from skin fibroblasts of SOD1 familial and sporadic ALS patients. Morpholino treatment improved pathological phenotype increasing cell survival. Moreover, Morpholino has been injected in a murine model of ALS obtaining promising results in terms of disease progression, neuromuscular function and survival.

This study provides a relevant background for the development of morpholino-mediated therapy for ALS and for other neurodegenerative diseases.

The research was conducted with the support of AriSLA (Fondazione Italiana di ricerca per la SLA), Pilot Grant ALSsiMO to Dr. Nizzardo.

For the full text please follow this link.

For further information:http://www.centrodinoferrari.com/la-terapia-con-oligonucleotidi-antisenso-morfolino-corregge-gli-effetti-della-sla-in-un-modello-murino/?lang=en 

 

Dr. Monica Nizzardo

Centro Dino Ferrari, Università degli Studi di Milano, IRCCS Fondazione Ca' Granda Ospedale Maggiore Policlinico

Tel. +3902 55033833+3902 55033833

monica.nizzardo1@gmail.com

 

 

Prof. Stefania Corti

Centro Dino Ferrari, Università degli Studi di Milano, IRCCS Fondazione Ca' Granda Ospedale Maggiore Policlinico

Tel. +3902 55033817+3902 55033817

stefania.corti@unimi.it

 

 

 




     
     
     
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