Materials and methods relating to the transfer of nucleic...

Details Materials and methods relating to the transfer of nucleic... Materials and methods relating to the transfer of nucleic...

C - Chemistry, Metallurgy

12

N

C12N 5/10 (2006.01) A61K 31/70 (2006.01) A61K 35/12 (2006.01) A61K 48/00 (2006.01) C07K 14/475 (2006.01) C12N 15/85 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2231735

Materials and methods for transferring nucleic acid encoding a polypeptide for treating a disease or disorder into populations of quiescent cells such as haematopoietic stem cells (HSCs), using retroviral packaging cell lines and retroviral particles expressing and displaying a growth factor such as stem cell factor (SCF) on the cell surface or as a fusion with a viral envelope protein. The present invention also relates to compositions comprising the retroviral packaging cell lines and retroviral particles, and their use in methods of medical treatment, in vivo and ex vivo.

Matériels et procédés de transfert d'acide nucléique codant un polypeptide, dans des populations de cellules quiescentes, telles que des cellules souches hématopoïétiques, destinés au traitement d'une maladie ou d'un trouble. Ces procédés consistent à utiliser des lignées cellulaires d'encapsidation rétrovirale et des particules rétrovirales, lesquelles expriment et montrent, sur la surface cellulaire, un facteur de croissance, tel qu'un facteur de cellule souche, ou une fusion avec une protéine d'enveloppe virale. La présente invention concerne également des compositions comprenant les lignées cellulaires d'encapsidation rétrovirale et des particules rétrovirales, ainsi que l'utilisation de celles-ci dans des méthodes de traitement médical, in vivo et ex vivo.

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