Provided herein are compositions and methods for modifying a predetermined nucleic acid sequence. A programmable nucleoprotein molecular complex containing a polypeptide moiety and a specificity conferring nucleic acid (SCNA) which assembles in-vivo, in a target cell, and is capable of interacting with the predetermined target nucleic acid sequence is provided. The programmable nucleoprotein molecular complex is capable of specifically modifying and/or editing a target site within the target nucleic acid sequence and/or modifying the function of the target nucleic acid sequence.

Provided herein are compositions and methods for modifying a predetermined nucleic acid sequence. A programmable nucleoprotein molecular complex containing a polypeptide moiety and a specificity conferring nucleic acid (SCNA) which assembles in-vivo, in a target cell, and is capable of interacting with the predetermined target nucleic acid sequence is provided. The programmable nucleoprotein molecular complex is capable of specifically modifying and/or editing a target site within the target nucleic acid sequence and/or modifying the function of the target nucleic acid sequence.

The present invention relates to antisense LNA oligonucleotides (oligomers) complementary to ATXN3 pre-mRNA sequences, which are capable of inhibiting the expression of ATXN3 protein. Inhibition of ATXN3 expression is beneficial for the treatment of spinocerebellar ataxia.

An aptamer, capable of inhibiting DNA methyltransferase 1 (DNMT1) for use in therapy of diseases characterised by aberrant DNA methylation, e.g. cancer. Method for identifying inhibitors of DNA methyltransferase. An aptamer, capable of inhibiting DNA methyltransferase 1 (DNMT1) for use in therapy of diseases characterised by aberrant DNA methylation, e.g. cancer. SELEX method for identifying aptamers of DNA methyltransferase optionally using 2-fluoro-pyrimindine nucleotide derivatives.

The present invention relates to PIKFYVE antisense oligonucleotides (ASOs), pharmaceutical compositions containing them, and methods for treating, inhibiting, suppressing, and preventing neurological diseases with them.

The invention relates to compositions and methods for the preparation, manufacture, and therapeutic use of compositions comprising mRNA and a lipid nanoparticle comprising a compound of the invention and an ionizable lipid.

The invention relates to compositions and methods for the preparation, manufacture, and therapeutic use of compositions comprising mRNA and a lipid nanoparticle comprising a compound of the invention and an ionizable lipid.

An agent for inhibiting the action of a target RNA based on RNA interference and ASO with a passenger strand and a guide strand for a target RNA, wherein both ends of double-stranded RNA formed pairing of the passenger strand and the guide strand are blunt ends, and one or two or more selected from the group consisting of the units represented by formula (1) and formula (2) below at the following positions (a) and (b): (a) 5′ end side and 3′ end side of the passenger strand, (b) 3′ end side of the guide strand

##STR00001##

(in which X represents an oxygen atom or sulfur atom),

##STR00002##

(in which X represents an oxygen atom or sulfur atom).

An agent for inhibiting the action of a target RNA based on RNA interference and ASO with a passenger strand and a guide strand for a target RNA, wherein both ends of double-stranded RNA formed pairing of the passenger strand and the guide strand are blunt ends, and one or two or more selected from the group consisting of the units represented by formula (1) and formula (2) below at the following positions (a) and (b): (a) 5′ end side and 3′ end side of the passenger strand, (b) 3′ end side of the guide strand

##STR00001##

(in which X represents an oxygen atom or sulfur atom),

##STR00002##

(in which X represents an oxygen atom or sulfur atom).

Aspects of the disclosure relate to methods for producing an antigen-specific immune response to human cytomegalovirus (hCMV) in a subject by administering mRNA vaccines.