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Methods for increasing fetal hemoglobin content in eukaryotic cells

The clinical severity of β-hemoglobinopathies is alleviated by the co-inheritance of genetic mutations causing a sustained fetal γ-globin chain […]

Gene therapy approach combined to chemotherapy for improving treatment

The present invention concerns products containing (i) at least one nucleic acid sequence coding for the human somatostatin 2 receptor protein (sst2), […]

Exon skipping therapy of erythropoietic protoporphyria

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

Pyk2-based gene therapy gene therapy attenuates cognitive deficits

In the present invention it is shown that the inactivation of the Pyk2 gene does not alter hippocampal development but prevents hippocampal-dependent […]

Pyk2-based gene therapy attenuates cognitive deficits associated to

In the present invention it is shows that the inactivation of the Pyk2 gene does not alter hippocampal development but prevents hippocampal-dependent […]

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 […]

Modulator of wip1 levels to treat wolfram syndrome

The present invention relates to novel therapeutic ways for treating Wolfram Syndrome (WS) by targeting the neuronal calcium sensor 1 (NCS1). The […]

Ex-vivo active immunotherapy against infectious diseases and tumoral

The present invention concerns lentiviral vectors enabling the expression of membrane-anchored and secreted antibodies by B cells. This invention […]

Improved vector for driving the targeted integration of a transgene

The present invention relates to polypeptide for engineering integrase chimeric proteins and their use in gene therapy. A key challenge for gene […]

Soluble appsα-based gene therapy in alzheimer’s disease

The present invention relates to a method of treating Alzheimer’s disease by administration to the subject of a therapeutically effective amount of […]