Abstract: The present invention is directed to antisense oligomeric compounds that may be used in the treatment Pompe disease as well as method for modulating the splicing of the GAA gene and method to treat Pompe disease. Also pharmaceutical compositions comprising the antisense oligomeric compounds are part of the invention.

Abstract: A method of manufacturing a microstructure comprises printing a positive mold structure, filling the positive mold structure with a second material to form an elastically deformable negative mold structure, filling the negative mold structure with a third material to form the microstructure, and releasing the microstructure from the negative mold structure. Advantageously, the negative mold structure can be stretched to facilitate the release of the microstructure. For example, the microstructure comprises a chamber with capped micropillars for the generation and/or analysis of muscle tissue.

Abstract: The invention is in the field of cell culturing. More specifically, it is in the field of generating and expanding myogenic cells from induced pluripotent stem (iPS) cells. The invention relates inter alia to cells generated and expanded via such a method, a growth medium specifically suited for the purpose of expanding isolated myogenic cells, and methods for screening compounds on cell structures such as myotubes and myofibers.

Abstract: The invention is in the field of cell culturing. More specifically, it is in the field of generating and expanding myogenic cells from induced pluripotent stem (i PS) cells. The invention relates inter alia to cells generated and expanded via such a method, a growth medium specifically suited for the purpose of expanding isolated myogenic cells, and methods for screening compounds on cell structures such as myotubes and myofibers.

Abstract: The invention relates to method for repairing aberrant; splicing, wherein such aberrant: splicing is caused by the presence of a natural pseudo exon, comprising blocking of either the natural cryptic 3? splice site or the natural cryptic 5? splice site of said natural pseudo exon with an antisense oligomeric compound (AON). Further, the invention comprises an antisense oligomeric compound targeting SEQ ID NO: 1 or SEQ ID NO: 171, preferably selected from the sequences of SEQ ID NO: 267-2040, sequences that are complementary to said sequences or sequences that have an identity of 80% with said sequences or the complementary sequences. The invention further envisages the use of two antisense oligomeric compounds, a first AON targeting SEQ ID NO: 1 and a second targeting AON or SEQ ID NO: 171. These AONs are specifically for use in the treatment of Pompe disease. It is an aspect of the invention that antisense therapy using the above AONs or combinations thereof is used in combination with ERT.

Abstract: The present invention is direct to the treatment of Pompe disease by administration of an enzyme or nucleic acid encoding for said enzyme suitable for Enzyme Replacement Therapy for Pompe disease in combination with the administration of an antisense oligomeric compound that modulates the splicing of acid alpha-glucosidase (GAA) gene.

Abstract: The present invention is directed to antisense oligomeric compounds that may be used in the treatment Pompe disease as well as method for modulating the splicing of the GAA gene and method to treat Pompe disease. Also pharmaceutical compositions comprising the antisense oligomeric compounds are part of the invention.

Abstract: The present invention is direct to the treatment of Pompe disease by administration of an enzyme or nucleic acid encoding for said enzyme suitable for Enzyme Replacement Therapy for Pompe disease in combination with the administration of an antisense oligomeric compound that modulates the splicing of acid alpha-glucosidase (GAA) gene.

Abstract: The present invention is directed to antisense oligomeric compounds that may be used in the treatment Pompe disease as well as method for modulating the splicing of the GAA gene and method to treat Pompe disease. Also pharmaceutical compositions comprising the antisense oligomeric compounds are part of the invention.

Abstract: The invention relates to methods for gene therapy in a subject suffering from Pompe disease, comprising gene-editing of a glucosidase, acid, alpha gene (GAA) in said subject. The invention further relates to a cell culture of genetically changed, differentiated myogenic progenitor cells derived from a donor subject suffering from Pompe disease, to a vector for use in a method for gene-editing a eukaryotic cell, and to a myotube prepared from myogenic progenitor cells that have been genetically changed by gene-editing.

Abstract: The disclosure provides method and kits for characterizing spliced m RNA isoforms. The disclosure also provides methods of screening for mutations and oligonucleotides that modulate splicing.

Abstract: The invention relates to method for repairing aberrant; splicing, wherein such aberrant: splicing is caused by the presence of a natural pseudo exon, comprising blocking of either the natural cryptic 3? splice site or the natural cryptic 5? splice site of said natural pseudo exon with an antisense oligomeric compound (AON). Further, the invention comprises an antisense oligomeric compound targeting SEQ ID NO: 1 or SEQ ID NO: 171, preferably selected from the sequences of SEQ ID NO: 267-2040, sequences that are complementary to said sequences or sequences that have an identity of 80% with said sequences or the complementary sequences. The invention further envisages the use of two antisense oligomeric compounds, a first AON targeting SEQ ID NO: 1 and a second targeting AON or SEQ ID NO: 171. These AONs are specifically for use in the treatment of Pompe disease. It is an aspect of the invention that antisense therapy using the above AONs or combinations thereof is used in combination with ERT.

Abstract: The present invention is directed to antisense oligomeric compounds that may be used in the treatment Pompe disease as well as method for modulating the splicing of the GAA gene and method to treat Pompe disease. Also pharmaceutical compositions comprising the antisense oligomeric compounds are part of the invention.

Abstract: The present invention is directed to antisense oligomeric compounds that may be used in the treatment Pompe disease as well as method for modulating the splicing of the GAA gene and method to treat Pompe disease. Also pharmaceutical compositions comprising the antisense oligomeric compounds are part of the invention.

Abstract: The invention is in the field of cell culturing. More specifically, it is in the field of generating and expanding myogenic cells from induced pluripotent stem (i PS) cells. The invention relates inter alia to cells generated and expanded via such a method, a growth medium specifically suited for the purpose of expanding isolated myogenic cells, and methods for screening compounds on cell structures such as myotubes and myofibers.

Abstract: The present invention is direct to the treatment of Pompe disease by administration of an enzyme or nucleic acid encoding for said enzyme suitable for Enzyme Replacement Therapy for Pompe disease in combination with the administration of an antisense oligomeric compound that modulates the splicing of acid alpha-glucosidase (GAA) gene.

Abstract: The disclosure provides method and kits for characterizing spliced m RNA isoforms. The disclosure also provides methods of screening for mutations and oligonucleotides that modulate splicing.

Abstract: The present invention is directed to antisense oligomeric compounds that may be used in the treatment Pompe disease as well as method for modulating the splicing of the GAA gene and method to treat Pompe disease. Also pharmaceutical compositions comprising the antisense oligomeric compounds are part of the invention.