Suggestions

Du même auteur

Long-term neurologic and cardiac correction in the Pompe disease mice by intrathecal gene therapy

Archive ouverte | Hordeaux, Juliette | CCSD

National audience. Pompe disease (glycogen storage disease type II) is a lysosomal storage disorder caused by acidalpha- glucosidase (GAA) deficiency leading to progressive accumulation of glycogen in the heart, mus...

Long-term neurologic and cardiac correction by intrathecal gene therapy in Pompe disease

Archive ouverte | Hordeaux, Juliette | CCSD

International audience. Pompe disease is a lysosomal storage disorder caused by acid-a-glucosidase (GAA) deficiency, leading to glycogen storage. The disease manifests as a fatal cardiomyopathy in infantile form. En...

Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats

Archive ouverte | Hordeaux, Juliette | CCSD

Intracerebral administration of recombinant adeno-associated vector (AAV) has been performed in several clinical trials. However, delivery into the brain requires multiple injections and is not efficient to target the spinal cord,...

Chargement des enrichissements...