hematopoietic stem cell gene therapy

Stem Cell Gene Therapy Blocks Anti-GAA Immune Response in Mice

Administrating a stem cell gene therapy before beginning enzyme-replacement therapy (ERT) prevented the development of antibodies against the acid alpha-glucosidase (GAA) enzyme, improved survival, and enhanced therapeutic benefits in a mouse model of Pompe disease, a recent study found. “Together, this study indicates that [stem cell] gene therapy induces…

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)…

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…