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May 24, 2022 News by Vanda Pinto, PhD

Avrobio‘s AVR-RD-03 Gene Therapy Effective in Pompe Mouse Model

AVR-RD-03, Avrobio‘s experimental blood stem cell gene therapy for classic infantile-onset Pompe disease, was found to be safe and effective in a mouse model of the disease. The findings, presented as a poster at the 2022 annual meeting of the American Society of Gene & Cell Therapy (ASGCT)…

July 8, 2020 News by Inês Martins, PhD

Stem Cell Gene Therapy Restores Glycogen Levels, Aids Movement in Mouse Study

Transplanting blood stem cells modified to carry the GAA gene raised glycogen levels almost to normal across a range of organs, including the brain, and improved locomotion in a mouse model of Pompe disease, a study reported. These findings suggest that blood stem cell gene therapy, in which a patient’s…

Recent Posts

  • Orthodontic care benefits young girl with infantile Pompe disease
  • Myozyme may help LOPD patients walk, breathe over long term
  • Genetic test flags Pompe disease in expectant father with no symptoms


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