The present invention provides ocular cells, genetically modified by a CRISPR system targeting the expression of B2M for ocular cell therapy. The invention further provides methods of generating an expanded population of genetically modified ocular cells, for example limbal stem cells (LSCs) or corneal endothelial cells (CECs), wherein the cells are expanded involving the use of a LATS inhibitor and the expression of B2M in the cells has been reduced or eliminated. The present invention also provides a cell populations, preparations, uses and methods of therapy comprising said cells.
[EN] 6-6 FUSED BICYCLIC HETEROARYL COMPOUNDS AND THEIR USE AS LATS INHIBITORS<br/>[FR] COMPOSÉS HÉTÉROARYLE BICYCLIQUES FUSIONNÉS EN 6-6 ET LEUR UTILISATION COMME INHIBITEURS DE LATS
申请人:NOVARTIS AG
公开号:WO2018198077A3
公开(公告)日:2019-02-14
[EN] METHODS AND COMPOSITIONS FOR OCULAR CELL THERAPY<br/>[FR] PROCÉDÉS ET COMPOSITIONS POUR UNE THÉRAPIE CELLULAIRE OCULAIRE
申请人:NOVARTIS AG
公开号:WO2021220132A1
公开(公告)日:2021-11-04
The present invention provides ocular cells, genetically modified by a CRISPR system targeting the expression of B2M for ocular cell therapy. The invention further provides methods of generating an expanded population of genetically modified ocular cells, for example limbal stem cells (LSCs) or corneal endothelial cells (CECs), wherein the cells are expanded involving the use of a LATS inhibitor and the expression of B2M in the cells has been reduced or eliminated. The present invention also provides cell populations, preparations, uses and methods of therapy comprising said cells.