Abstract:
The present invention relates to a method to cleave target nucleic acid sequence by the catalytic domain of a GIY-YIG homing endonucleases I-TevI. More precisely, the invention relates to the deciphering of new preferential I-TevI cleavage sites for efficient and specific cleavage activity. The invention concerns a method for the generation of TevI specific chimeric endonucleases to target nucleic acid sequence including such cleavage sites and methods of using same for gene editing.
Abstract:
The present invention relates to genetically engineered immune cells expressing new anti-MUC1 chimeric antigen receptors and their use in the treatment of solid tumors, particularly suited for allogeneic cell immunotherapy.
Abstract:
The invention relates to therapeutic compositions for allogeneic cellular therapy comprising TCR deficient T-cells, which are genetically engineered to express immune cell engagers, and methods related thereto.
Abstract:
The present invention relates to polypeptides and more particularly to Transcription Activator-Like Effector derived proteins that allow to efficiently target and/or process nucleic acids. Particularly, the present invention reports the characterization of TALE derived proteins that can efficiently target methylated DNA. The present invention more specifically relates to TALE derived proteins that allow activation of methylated promoters responsible for gene silencing.
Abstract:
The present invention relates to a method to cleave target nucleic acid sequence by the catalytic domain of a GIY-YIG homing endonucleases I-TevI. More precisely, the invention relates to the deciphering of new preferential I-TevI cleavage sites for efficient and specific cleavage activity. The invention concerns a method for the generation of TevI specific chimeric endonucleases to target nucleic acid sequence including such cleavage sites and methods of using same for gene editing.
Abstract:
The present invention relates to a method for the generation of compact Transcription Activator-Like Effector Nucleases (TALENS) that can efficiently target and process double-stranded DNA. More specifically, the present invention concerns a method for the creation of TALENs that consist of a single TALE DNA binding domain fused to at least one catalytic domain such that the active entity is composed of a singe polypeptide chain for simple and efficient vectorization and does not require dimerization to target a specific single double-stranded DNA tar et sequence of interest and process DNA nearby said DNA target sequence. The present invention also relates to compact TALENs, vectors, compositions and kits used to implement the method.
Abstract:
The present invention generally relates to the field of genome engineering (gene editing), and more specifically to gene therapy for the treatment of Hyper-lgE syndrome (HIES). In particular, the present invention provides means and methods for genetically modifying HSCs or T-cells involving gene editing reagents, such as TALE-nucleases, that specifically target an endogenous STATS gene comprising at least one mutation causing Hyper-lgE syndrome (HIES), thereby allowing the restoration of the normal cellular phenotype. The present invention also provides populations of engineered HSCs or T-cells which comprise cells comprising an exogenous polynucleotide sequence comprising at least a partial or complete sequence of a functional STATS gene, said exogenous polynucleotide sequence being integrated in an endogenous STATS gene comprising at least one mutation causing Hyper-lgE syndrome (HIES), resulting in the expression of a functional STATS polypeptide. The present invention further provides pharmaceutical compositions comprising the cell populations of the invention, and their use in gene therapy for the treatment of Hyper-lgE syndrome (HIES).
Abstract:
The present disclosure provides methods to genetically modify cells by insertion of an artificial exon (ArtEx) for delivery of therapeutic proteins in specific cell types and more particularly engineered cells for expression of a transgene into the brain of a patient.
Abstract:
The present invention concerns new engineered immune cells expressing two CARs directed against two different targets, polynucleotides for preparing said immune cells, pharmaceutical compositions comprising said immune cells, and the use of said immune cells in the treatment of cancers.
Abstract:
The present invention relates to polypeptides and more particularly to Transcription Activator-Like Effector derived proteins that allow to efficiently target and/or process nucleic acids. The present invention also concerns methods to use these proteins. The present invention also relates to vectors, compositions and kits in which RVD domains and Transcription Activator-Like Effector (TALE) proteins of the present invention are used.