Abstract: The present invention relates to a biodegradable multilayer nanocapsule for the delivery of at least one biologically active agent into at least one target cell consisting of at least two layers of at least two biodegradable polymers which are laid one onto the other and whereby the biologically active agent is layered onto a layer of a biodegradable polymer and covered with a further layer of a biodegradable polymer, whereby one biologically active agent is a nucleic acid.

Abstract: The present invention relates to modified Cas9 nuclease comprising a substantial part of a Cas9 nuclease and fused thereto at least one substantial part of a dominant negative effector on non-homologous end-joining selected from the group consisting of RNF168, 53BP1, Ku80 and DNA-PK which compete with NHEJ promoting factors and CtIP.

Abstract: The invention pertains to the field of cell therapy and HIV treatments. It provides with highly specific reagents for reducing or inactivating expression of CCR5 in primate and human primary cells, especially under the form of TALE-nucleases. These reagents allow the production of safer primary hematopoietic cells made resistant to HIV, stem cells or differentiated cells, for their infusion into HIV patients.

Abstract: The present invention relates to a novel chimeric antigen receptor (CAR) comprising an antigen-binding fragment which binds specifically to PSMA antigen, and a method of manufacturing high-quality CAR T cell products by transfection and/or transduction of T cells therewith, which allows to effectively treat tumors in vivo alone or in combination with pharmaceutical drugs, such chemotherapies, biopharmaceutical drugs, such as antibodies, or small-molecule drugs, such as protein kinase inhibitors.

Abstract: The present invention relates to a biodegradable multilayer nanocapsule for the delivery of at least one biologically active agent into at least one target cell consisting of at least two layers of at least two biodegradable polymers which are laid one onto the other and whereby the biologically active agent is layered onto a layer of a biodegradable polymer and covered with a further layer of a biodegradable polymer, whereby one biologically active agent is a nucleic acid.

Abstract: Disclosed is a method for high-throughput detection of genome-wide modifications in a nucleic acid genome obtained from a cell or tissue caused by the activity of a designer nuclease comprising the following steps: a) Extraction of the genomic DNA from cells that were exposed to a designer nuclease under conditions which allow the designer nuclease to introduce a DNA double-strand break (DSB) in the genomic DNA of the cell, b) fragmentation of the nucleic acid to obtain random fragments, c) performing an end repair in order to obtain blunt ends, d) ligation with a linker comprising a sequence complementary to a so called “linker primer”, e) performing a first nucleic acid amplification reaction with a “linker primer” and a so called “ON-target primer”, whereby one primer is located upstream and one primer is located downstream of the on-target site, wherein at least one decoy primer is present in the reaction mixture, f) performing a second nucleic acid amplification reaction whereby so called “nested prim

Abstract: The present invention relates to a biodegradable multilayer nanocapsule for the delivery of at least one biologically active agent into at least one target cell consisting of at least two layers of at least two biodegradable polymers which are laid one onto the other and whereby the biologically active agent is layered onto a layer of a biodegradable polymer and covered with a further layer of a biodegradable polymer, whereby one biologically active agent is a nucleic acid.

Abstract: The present invention relates to a novel chimeric antigen receptor (CAR) comprising an antigen-binding fragment which binds specifically to PSMA antigen, and a method of manufacturing high-quality CAR T cell products by transfection and/or transduction of T cells therewith, which allows to eradicate tumors in vivo alone or in combination with pharmaceutical drugs, such chemotherapies, biopharmaceutical drugs, such as antibodies, or small-molecule drugs, such as protein kinase inhibitors.

Abstract: Disclosed is a method for high-throughput detection of genome-wide modifications in a nucleic acid genome obtained from a cell or tissue caused by the activity of a designer nuclease comprising the following steps: a) Extraction of the genomic DNA from cells that were exposed to a designer nuclease under conditions which allow the designer nuclease to introduce a DNA double-strand break (DSB) in the genomic DNA of the cell, b) fragmentation of the nucleic acid to obtain random fragments, c) performing an end repair in order to obtain blunt ends, d) ligation with a linker comprising a sequence complementary to a so called “linker primer”, e) performing a first nucleic acid amplification reaction with a “linker primer” and a so called “ON-target primer”, whereby one primer is located upstream and one primer is located downstream of the on-target site, wherein at least one decoy primer is present in the reaction mixture, f) performing a second nucleic acid amplification reaction whereby so called “nested prim

Abstract: The present invention relates to the in vivo delivery system of the DNA modifying enzymes comprising the stem cells-derived extracellular vesicles collected in serum-free conditions, characterized in that the said extracellular vesicles are the population of extracellular vesicles defined by the expression of surface markers. Moreover, the present invention is dedicated for use in a treatment of a genetic disease and/or disorder.

Abstract: Herein described is a construct for epigenomic modification of genes composed of: (a) a Krüppel-associated box zinc finger protein or homologous, (b) a DNA region capable of binding to the target gene or homologous, (c) a human DNA methyltransferase DNMT3A or homologous and (d) a murine DNA methyltransferase Dnmt3L or homologous, wherein components a), b), c) and d) are linked to each other either directly or via at least one linker. The construct is a designer epigenome modifier that can be used to silence genes coding for a protein in leukocytes that avoids the internalization of HI viruses in immune cells.

Abstract: The invention pertains to the field of cell therapy and HIV treatments. It provides with highly specific reagents for reducing or inactivating expression of CCR5 in primate and human primary cells, especially under the form of TALE-nucleases. These reagents allow the production of safer primary hematopoietic cells made resistant to HIV, stem cells or differentiated cells, for their infusion into HIV patients.

Abstract: The present invention relates to a biodegradable multilayer nanocapsule for the delivery of at least one biologically active agent into at least one target cell consisting of at least two layers of at least two biodegradable polymers which are laid one onto the other and whereby the biologically active agent is layered onto a layer of a biodegradable polymer and covered with a further layer of a biodegradable polymer, whereby one biologically active agent is a nucleic acid.

Abstract: The present invention concerns a construct for epigenomic modification of genes that includes the following components: a) a Krüppel-associated box zinc finger protein or homologous, b) a DNA region capable of binding to the target gene or homologous, c) a human DNA methyltransferase DNMT3A or homologous and d) a murine DNA methyltransferase Dnmt3L or homologous whereby components a), b), c) and d) are linked to each other either directly or via at least one linker. The construct is a designer epigenome modifier which can be used to silence genes coding for a protein in leukocytes which avoids the internalization of HI viruses in immune cells.

Abstract: The present invention concerns a construct for epigenomic modification of genes comprising the following components: a) a Krüppel-associated box zinc finger protein or homologous, b) a DNA region capable of binding to the target gene or homologous, c) a human DNA methyltransferase DNMT3A or homologous and d) a murine DNA methyltransferase Dnmt3L or homologous whereby components a), b), c) and d) are linked to each other either directly or via at least one linker. The construct is a designer epigenome modifier which can be used to silence genes coding for a protein in leukocytes which avoids the internalization of HI viruses in immune cells.