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Histone chaperone hira inhibitor for treating hepatitis b virus

The present invention concerns an inhibitor for use for preventing and/or treating an infection with hepatitis B virus (HBV) and an in vitro screening […]

Methods for the diagnosis and treatment of gastrointestinal stromal

The present invention relates to the diagnosis of gastrointestinal stromal tumors (GISTs). The present invention also relates to methods and […]

Exon skipping therapy of erythropoietic protoporphyria

The present invention relates to methods and pharmaceutical compositions for the treatment of Erythropoietic Protoporphyria. In particular, the […]

Hydrophobically modified antisense oligonucleotides comprising a cetal

The present invention concerns an oligonucleotide modified by substitution at the 3’ or the 5’ end by at least triple alkyl chain or at least one […]

Ctip fusion to cas9 enhances transgene integration by

The present invention relates to nuclease protein fusions for enhancing genome editing by homology-directed transgene integration (HDI). The […]

Ex vivo gene therapy based vaccine with nucleic acid molecule encoding

The present invention relates to a method of treating a B-cell malignancy in a subject using a vaccine composition comprising a population of […]

A microrna switch regulates the rise in hypothalamic gnrh production

A method for diagnosing a reproduction-related disorder in an individual, comprising: a) measuring the level of a miR200 nucleic acid and/or miR155 […]

New antisense oligonucleotide targeting all cystic fibrosis patients

The present invention relates to methods and pharmaceutical compositions for the treatment of cystic fibrosis (CF). In particular, the present […]

Lipid antisens oligonucleotide targeting fxyd2 as a therapeutic agent

The present invention relates to an inhibitor of FXYD2 gene expression for use in a method for treating neuropathic pain in a patient in need thereof. […]