New Gene Therapy Study in Friedreich’s Ataxia (FA) Sponsored by LEXEO Therapeutics, Inc

The study is being conducted with multiple sites in the United States and Canada seeking to recruit approximately 9 participants aged 18-40 with a diagnosis of FA-associated cardiomyopathy. The study will be conducted by Dr. Antoine Duquette at the CHUM in Montreal. The choice of Montreal as a site reflects...

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Research grant 2020

Since its inception, the Ataxia Canada Foundation has had the mission of supporting research to contribute to the development of therapies targeting both the causes and the effects of different forms of ataxia. The pursuit of this objective is still very much alive within the administration and the members of the Foundation. Research funded by...

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Gene editing of human hematopoietic stem and progenitor cells for Friedreich’s ataxia using CRISPR/Cas9 technology

  Gene editing of human hematopoietic stem and progenitor cells for Friedreich’s ataxia using CRISPR/Cas9 technology Dr. Celine Rocca 1, Ms. Shi Yanmeng 1, Mr. Jay Sharma 1, Dr. Prashant Mali 1, Dr. Stephanie Cherqui 1 University of California, San Diego Our goal is now to develop an autologous HSPC...

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