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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New Collaborative Clinical Research Site on Friedreich’s Ataxia in Montreal

Friedreich Ataxia Collaborative Clinical Research Network and Natural History Research Project We want  to inform you that Dr. Antoine Duquette, a neurologist at the CHUM, and his research team will now be responsible for a site in Montreal that integrates the Collaborative Research Network in Friedreich Ataxia (CCRN in FA)...

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The Working Life of People with Degenerative Cerebellar Ataxia

The aim of the present study was to characterize and analyze the most important individual and organizational variables associated with job accommodation in subjects with degenerative cerebellar ataxia by administering a series of international and validated work activity-related scales. Twenty-four workers (W) and 58 non-workers (NW) were recruited: 34 with...

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Defining the genetic etiology of late onset episodic ataxias

We want to present the research of Martine Tétreault Assistant professor Department of neuroscience, University of Montreal The identification of the genetic causes and mechanisms associated with rare Mendelian diseases has always been an important challenge for the medical field. The identification of mutations and/or genes associated with a disease...

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New developments in pharmacotherapy for Friedreich ataxia article

Current and emerging therapies for Friedrich’s Ataxia (FA) focus on reversing the deleterious effects of such deficiency including mitochondrial augmentation and increasing frataxin levels, providing the possibility of treatment options for this physiologically complex, multisystem disorder. In this review article, the authors discuss the current and prior in vivo and...

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Gene therapy abroad

Neurocrine Biosciences and Voyager Therapeutic announced the formation of a strategic collaboration focused on the development and commercialization of Voyager’s gene therapy programs, VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia, as well as rights to two programs to be determined. This collaboration combines Neurocrine Biosciences’ expertise in neuroscience,...

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