Novel systems for nucleic acid regulation

Abstract: the present invention relates to a nucleic acid comprising two or more rna encoding sequences, wherein the each of the rna comprises a hairpin and a guide sequence substantially complementary to part of a gene of choice, and to associated aavs, compositions, pharmaceutical compositions and uses in treatments thereof.

Inventor(s): Irena Bockaj, Morgane Sarah Wartel, Sebastian Niklas Kieper, Astrid Valles Sánchez, Seyda Acar Broekmans, Melvin Maurice Evers

CPC Classification: C12N15/113 (Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; {Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing (when used in plants )})

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