|
|||||
|
|
Partial phenotypic correction and immune tolerance induction to enzyme replacement therapy after hematopoietic stem cell gene transfer of α‐glucosidase in Pompe disease |
|
| Authors: | Gaëlle Douillard‐Guilloux Emmanuel Richard Lionel Batista Catherine Caillaud |
| Affiliation: | 1. Institut Cochin, Université Paris Descartes, CNRS (UMR 8104), Paris, France;2. INSERM, U567, Paris, France;3. Both investigators contributed equally and should be considered as senior authors. |
| Abstract: | |
| Keywords: | acid maltase glycogen storage disease hematopoietic stem cell immune tolerance induction lentiviral vector Pompe disease |