Posts by Clotilde Lagier-Tourenne, MD, PhD
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Antisense Drugs Mimic TDP-43 Function, Might Treat ALS and Some Dementias
Clotilde Lagier-Tourenne, MD, PhD, and colleagues have demonstrated in vitro and in vivo efficacy of antisense oligonucleotides in preventing missplicing of stathmin-2 in a mouse model of amyotrophic lateral sclerosis attributable to deficiency of the protein TDP-43.
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Genomic Therapeutics in Action: Antisense Oligonucleotides Interventions Reverses Neurodegeneration
Physician-investigators at Massachusetts General Hospital recently published findings that provide a critical step forward in understanding the mechanisms underlying a host of neurodegenerative diseases.
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Researchers Identify a New Therapeutic Target for TDP-43-mediated Neurodegenerative Diseases
Loss of TDP-43 nuclear function is a major event in ALS pathogenesis and leads to widespread RNA processing alterations including reduced stathmin-2 levels. Stathmin-2 is shown to be crucial for axonal regeneration and to represent a novel therapeutic target in ALS.
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Identifying New Potential Therapeutic Targets for ALS FTD
Transcripts of repeat expansions in the C90RF72 gene undergo a repeat-associated non-AUG (RAN) translation pathway that generates multiple aberrant proteins. Researchers at Mass General Hospital identified new potential therapeutic targets for ALS FTD.
Biography
Clotilde trained as both a medical geneticist and a neuroscientist, earning her MD at the Medical School of Strasbourg, France and a PhD from the University Louis Pasteur, Strasbourg. She received further training in Medical Genetics at Columbia University, where her work focused on the identification of new genetic causes of neurological disorders. After a postdoctoral training with Dr. Don Cleveland at the Ludwig Institute for Cancer Research, she became an Assistant Professor at the University of California San Diego until 2015, when she moved to Massachusetts General Hospital. She received the Alphonse Laveran Prize, the Milton-Safenowitz Postdoctoral Fellowship from the Amyotrophic Lateral Sclerosis Association, the Muscular Dystrophy Association Career Development Award, and the Frick Foundation 2013 Award (conjointly with Dr. Luc Dupuis). In 2014, Clotilde was awarded the 6th International Medicine Paulo Gontijo Award, which recognizes the most promising scientist under the age of 40 working on ALS. Clotilde is an Associate Member of the Broad Institute of MIT and Harvard.
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