Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease.

@article{Corti2017SafetyOI,
  title={Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease.},
  author={Manuela Corti and Cristina Liberati and Barbara Kellerman Smith and Lee Ann Lawson and Ibrahim S. Tuna and Thomas J. Conlon and Kirsten E Coleman and Saleem Islam and Roland W. Herzog and D D Fuller and Shelley Wells Collins and Barry Byrne},
  journal={Human gene therapy. Clinical development},
  year={2017},
  volume={28 4},
  pages={
          208-218
        }
}
A first-in-human trial of diaphragmatic gene therapy (AAV1-CMV-GAA) to treat respiratory and neural dysfunction in early-onset Pompe disease was conducted. The primary objective of this study was to assess the safety of rAAV1-CMV-hGAA vector delivered to the diaphragm muscle of Pompe disease subjects with ventilatory insufficiency. Safety was assessed by measurement of change in serum chemistries and hematology, urinalysis, and immune response to GAA and AAV, as well as change in level of… CONTINUE READING
Recent Discussions
This paper has been referenced on Twitter 6 times over the past 90 days. VIEW TWEETS