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Spotlight on Gene Therapy: finding a Cure

Mueller Lab at inKNOWvation event

Tuesday, May 10, 2016
By:  Florie Borel

The Mueller Lab for Gene Therapy will present its work on May 11th, 2016 at the inKNOWvation event in Cambridge, MA.

We will discuss two major projects at this event:

  • Our first poster "Treating neurodegenerative disorders with gene therapy" will give an overview of our silencing platform for neuro applications, and we will report progress made on our two lead programs, SOD1-linked ALS and Huntington's disease. Preclinical data will be presented, for SOD1-linked ALS we have proof-of-concept in ALS mice as well as in non-human primates, and for HD we have proof-of-concept in HD mice as well as in the HD sheep model.
    In addition to Dr Mueller, our collaborator Dr Robert Brown, a leader in the field of ALS, will also attend the event and contribute his expertise to the panel discussion.
  • Our second poster "Treating Alpha-1 Antitrypsin Lung and Liver Disease:
    Gene Therapy Approaches" will present our two programs for alpha-one antitrypsin deficiency. One is the gene augmentation therapy in collaboration with Dr Terry Flotte, a pioneer in AAV gene therapy who will be present at the event. The other is the dual function gene therapy, a proprietary vector which simultaneously augments normal AAT and silences mutant AAT and would therefore provide the first cure for this disease.

Learn more about this event here and register for free here!