In utero gene therapy for fetuses

Details In utero gene therapy for fetuses In utero gene therapy for fetuses

A - Human Necessities

61

K

A61K 48/00 (2006.01) C12N 15/86 (2006.01)

Patent

CA 2177998

A process for effecting gene therapy in utero in a fetus, which comprises transducing fetal cells in vivo with at least one nucleic acid sequence encoding a therapeutic agent. The fetal cells may be transduced with a viral vector (such as a retroviral vector) which includes the nucleic acid sequence encoding the therapeutic agent. The viral vector may be contained in a viral supernatant which is administered to the fetus, or may be generated by a producer cell line which is administered to the fetus.

Procédé de thérapie génique s'adressant à un foetus in utero, selon lequel on transduit des cellules foetales in vivo avec au moins une séquence nucléotidique codant un agent thérapeutique. Les cellules foetales peuvent être transduites avec un vecteur viral (tel qu'un vecteur rétroviral) qui comprend la séquence nucléotidique codant l'agent thérapeutique. Le vecteur viral peut être contenu dans un surnageant viral qui est administré au foetus ou qui peut être généré par une lignée cellulaire productrice administrée au foetus.

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