Abstract: 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.

Abstract: The present invention is related to 6-6 Fused Bicyclic Heteroaryl Compounds of the Formula A2 or A1 and their Use as LATS Inhibitors, or a salt, stereoisomer or pharmaceutical composition thereof; wherein the variables are as defined herein. The present invention further relates to a method of LATS inhibition in a cell population using a compound of Formula A1, or a salt, stereoisomer or pharmaceutical composition thereof. The present invention further provides a method for manufacturing compounds of the invention, and its therapeutic uses. The invention further provides methods to their preparation, to their medical use, their use in the treatment and management of diseases or disorders.

Abstract: The present invention is related to 6-6 Fused Bicyclic Heteroaryl Compounds of the Formula A2 or A1 and their Use as LATS Inhibitors, or a salt, stereoisomer or pharmaceutical composition thereof; wherein the variables are as defined herein. The present invention further relates to a method of LATS inhibition in a cell population using a compound of Formula A1, or a salt, stereoisomer or pharmaceutical composition thereof. The present invention further provides a method for manufacturing compounds of the invention, and its therapeutic uses. The invention further provides methods to their preparation, to their medical use, their use in the treatment and management of diseases or disorders.

Abstract: 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.

Abstract: The present invention is related to 6-6 Fused Bicyclic Heteroaryl Compounds of the Formula A2 or A1 and their Use as LATS Inhibitors, or a salt, stereoisomer or pharmaceutical composition thereof; wherein the variables are as defined herein. The present invention further relates to a method of LATS inhibition in a cell population using a compound of Formula A1, or a salt, stereoisomer or pharmaceutical composition thereof. The present invention further provides a method for manufacturing compounds of the invention, and its therapeutic uses. The invention further provides methods to their preparation, to their medical use, their use in the treatment and management of diseases or disorders.

Abstract: PiggyBac transposons and transposases with enhanced transposition activity in cells are provided. Also provided are associated methods and kits for both introducing exogenous DNA inserts into the genomes of host cells as well as for the removal of the inserts from the host cell genomes. Cells obtained by use of the compositions, methods and kits are also provided.

Abstract: The present invention provides a new fluorescent protein, engineered to facilitate release from self-cleaving peptides.

Abstract: The present invention provides a new fluorescent protein, engineered to facilitate release from self-cleaving peptides.

Abstract: PiggyBac transposons and transposases with enhanced transposition activity in cells are provided. Also provided are associated methods and kits for both introducing exogenous DNA inserts into the genomes of host cells as well as for the removal of the inserts from the host cell genomes. Cells obtained by use of the compositions, methods and kits are also provided.

Abstract: The present invention provides methods and materials that can be used to detect, examine and exploit protease mediated protein expression. An illustrative embodiment of the invention is a polynucleotide encoding a polypeptide having a plurality of operatively linked elements comprising a polypeptide degradation signal that functions as a proteosome substrate; a polypeptide of interest having an observable activity; and a linker disposed between the polypeptide degradation signal and the polypeptide of interest. The linker comprises a protease recognition motif that is capable of being specifically recognized and cleaved by a protease. The activity of the polypeptide of interest is observed when the motif is specifically recognized and cleaved by the protease; and the activity of the polypeptide of interest is decreased or not observed when the motif is not specifically recognized and cleaved by the protease.

Abstract: The present invention provides methods and materials that can be used to detect, examine and exploit protease mediated protein expression. An illustrative embodiment of the invention is a polynucleotide encoding a polypeptide having a plurality of operatively linked elements comprising a polypeptide degradation signal that functions as a proteosome substrate; a polypeptide of interest having an observable activity; and a linker disposed between the polypeptide degradation signal and the polypeptide of interest. The linker comprises a protease recognition motif that is capable of being specifically recognized and cleaved by a protease. The activity of the polypeptide of interest is observed when the motif is specifically recognized and cleaved by the protease; and the activity of the polypeptide of interest is decreased or not observed when the motif is not specifically recognized and cleaved by the protease.