Provided are a composition for ribonucleoprotein delivery, comprising a guide RNA free of 5′-terminal phosphates, and a method for ribonucleoprotein delivery, using the same.

The disclosure relates to methods and compositions that serve to reduce the potential for targeted nuclease activity for example, but not limited to, CRISPR enzymes such as Cas9 and/or Cas12a. The disclosure also relates to methods and compositions be used as an HDR substrate to prevent Cas9 re-cleavage, and as a result increase the frequency of perfect HDR.

The present invention relates to a composition for genome editing using a CRISPR/Cpf1 system and a use thereof and, more particularly, to a composition for genome editing comprising: a CRISPR RNA (crRNA) including a guide sequence capable of hybridizing with a target nucleotide sequence, and a uridine repeat sequence connected to the 3′-end of the guide sequence, or a DNA encoding the same; and a Cpf1 protein or a DNA encoding the same, a method for genome editing using the same, a method for construction of a genetically modified organism, and a genetically modified organism. The present invention can increase an indel efficiency and decrease off-target activity in genome editing of eukaryotic cells using the CRIPSPR/Cpf1 system and thus can easily construct a genetically modified cell or genetically modified animal or plant having a desired gene inserted thereinto (knock-in) or deleted therefrom (knock-out).

The instant invention provides RNA nanocubes, DNA nanocubes and R/DNA chimeric nanocubes comprising one or more functionalities. The multifunctional RNA nanocubes are suitable for therapeutic or diagnostic use in a number of diseases or disorders.

This invention relates to reagents and methods for increasing specificity and efficiency of genome editing by CRISPR associated (Cas) protein systems, more particularly by Cas:guide RNA complexes, by blocking off-target nucleic acids from cleavage by Cas:guide RNA complexes.

This invention provides compounds, compositions and methods for modulating the expression of target genes using RNA interference. RNAi structures and molecules of this invention can be used for modulating or silencing the expression of genes, with high levels of RNAi activity and reduced off target actions. Advantageous structures include siRNAs targeted to any gene having one or more 2′-deoxy nucleotides located in the seed region. The RNA interference molecules can be used in methods for preventing or treating diseases.

The present invention provides oligomers that binds RNA that consists of a lower affinity region and a higher affinity region, wherein the monomers in the higher affinity region increases the melting temperature (i.e. affinity) of the oligomer base paired to RNA more than the monomers used in the lower affinity region, wherein said increase in melting temperature is relatively to the alternative use of DNA monomers of the same (base) sequence. The oligomers of the invention are useful for binding to target RNA such as mRNA and non-coding RNA and have the advantage that they will be less prone to off-target binding via the lower affinity region than via the higher affinity region.

The present invention relates to RNA interference-inducing nucleic acid that inhibits noncanonical target genes of micro RNA, in which part of the sequence of a specific micro RNA has been modified, and by using the RNA interference-inducing nucleic acid of the present invention, the biological function micro RNA exhibits by inhibiting noncanonical target genes is effectively increased or there is the benefit of selectively exhibiting only one of the biological functions of conventional micro RNA, i.e., the function of inhibiting noncanonical target genes, and the interference-inducing nucleic acid of the present invention enables cell cycling, differentiation, dedifferentiation, formation, movement, splitting, proliferation or death adjustment, and it is expected that the invention can be used in various fields such as drugs and cosmetics.

The present disclosure provides DNA-guided CRISPR systems; polynucleotides comprising DNA, RNA and mixtures thereof for use with CRISPR systems; and methods of use involving such polynucleotides and DNA-guided CRISPR systems.

Novel oligonucleotide pairs which can form a duplex comprising one or more DNA-like nucleotides (e.g., 2′-substituted arabinonucleotides (ANA)); in combination with one or more RNA-like nucleotides (e.g., 2′-substituted ribonucleotides (RNA) and/or locked nucleic acid nucleotides (LNA)), are disclosed. The use of such oligonucleotide duplexes, such as for silencing the expression of a nucleic acid or gene of interest using small interfering RNA (siRNA) technologies, is also disclosed.