“High tech” involvement in mitochondrial medicine. Outcomes of the 10th Italian Meeting on Mitochondrial Diseases
The 10th Italian Meeting on Mitochondrial Diseases, which was held virtually on 9 and 10 October, has been particularly rich. The meeting involved the international scientific community, with more than 300 participants among experts, patients and family members from over 30 countries.
Mitochondrial diseases are very complex disorders that until a few years ago were little known by the scientific community itself. In recent years, great strides have been made in the study of the basic mechanisms involved in these diseases, confirmed by the shift of the main focus of research from the study of the possibilities of making diagnoses, to the identification and development of therapeutic strategies and clinical trials.
Main themes of the 10th Italian Meeting on Mitochondrial Diseases have been the updates on gene therapy and the acceleration of the involvement of “high tech” in daily practice, together with the progress made by science in mitochondrial medicine. A very important part was dedicated to the research of therapies, with a specific focus on ongoing clinical trials.
“This tenth edition of our annual meeting on mitochondrial diseases has been dedicated to one of the fathers of mitochondrial medicine, Prof. Salvatore Di Mauro, for decades leading one of the first and main research groups on mitochondria at Columbia University of NY (USA), which has attracted and raised entire generations of mitochondriologists in Italy and around the world.
For us, patients and family members of patients, what happened in the world of mitochondrial diseases over the past decade seems like a dream. It is a dream to know that therapies are starting to be available (although very few, for the moment), but, at this point, ideas and trials are on the agenda ”, Piero Santantonio, President of Mitocon.
To watch the videos of the presentations on Mitocon’s YouTube channel click here.
Below direct links to each video:
Special lecture: the mitochondrial journey. Celebrating the 80th years of Billi Di Mauro, M. Zeviani
Telemedicine during Covid-19 , C. Lamperti (online soon)
Epigenetics of pregnancy: when DNA is not the boss, D. Zuccarello
Overview of biological biomarkers, A. Suomalainen
PTC743 Mitochondrial Epilepsy Trial Update, M. Klein
Abliva’s innovative drug development programs for primary mitochondrial diseases, M. Hansson
MAT: Minovia’s journey with a Personalized Therapy, A. Gill
Lumevoq Gene Therapy in Leber Hereditary Optic Neuropathy (LHON), M. Taiel
Highlights on the state of the art of Idebenone therapy in LHON, X. Llòria
Overview of REN001 Clinical Development Program, A. Dorenbaum
Khondrion’s Sonlicromanol: progress update, J. Smeitink
Changing mtDNA heteroplasmy with DNA editing enzymes, C. T. Moraes
Experimental models for mtDNA maintenance defects, C. Viscomi
Treatment of mitochondrial diseases with AAV: the proposal of gene therapy for MNGIE, R. Martí
A bacterial cytidine deaminase toxin enables CRISPR-free mitochondrial base editing, B. Y. Mok
Outcome measures in primary mitochondrial myopathies. M. Mancuso
Clinical course of MELAS. Can we predict it?, R. McFarland
Inflammation and mitochondrial diseases, D. Martinelli (online soon)
iPS models for OPA1 and POL Gassociated with parkinsonism, V. Broccoli
Inhibition of autophagy corrects the visual defects in a model of ADOA, L. Scorrano