Disclosed herein are improved PNA based monomers, nucleobases, oligomers and probes for use in a variety of different methods of analysing nucleic acids. Further, the disclosure provides methods of preparing the modified and improved PNA molecules as well as methods of using the same.

The. invention relates to a method for inducing or promoting skipping of exon 45 of DMD pre-mRNA in a Duchenne Muscular Dystrophy patient, preferably in an isolated (muscle) cell, the method comprising providing said cell with an antisense molecule that binds to a continuous stretch of at least 21 nucleotides within said exon. The invention further relates to such antisense molecule used in said method.

Biologically active nucleotide molecules are configured, with the nucleotide sequence thereof, to be able to trigger several, in particular a plurality of “off-target” effects to cause cell-killing stress by means of binding of same, by means of which off-target effects cells are so massively influenced that the cells die off or programmed cell death (apoptosis) is induced in the cells.

The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Glial cell derived neurotrophic factor (GDNF), in particular, by targeting natural antisense polynucleotides of Glial cell derived neurotrophic factor (GDNF). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of GDNF.

Provided herein are composition for sensitizing tumors to anti-tumor therapies. The compositions include antisense oligonucleotides against TGFβ2, wherein the compositions sensitize tumors to anti-tumor therapies. Also provided herein are methods for treating cancer using the compositions described herein.

The present invention discloses a peptide nucleic acid for porcine reproductive and respiratory syndrome virus (PRRSV) and use thereof. The peptide nucleic acid of the present invention is selected from any one or more from the peptide nucleic acids having a) a nucleic acid sequence of Sequence 1 as shown in the Sequencing List; b) a nucleic acid sequence of Sequence 2 as shown in the Sequencing List; c) a nucleic acid sequence of Sequence 3 as shown in the Sequencing List; and d) a nucleic acid sequence of Sequence 3 as shown in the Sequencing List. The peptide nucleic acid of the present invention has no toxic side effect and no resistance, is able to specifically directly inhibit the replication of PRRSV, has a good anti-viral effect, and suffers no food safety problems including drug residue and others.

The present disclosure relates to antisense oligomers and related compositions and methods for inducing exon inclusion as a treatment for glycogen storage disease type II (GSD-II) (also known as Pompe disease, glycogenosis II, acid maltase deficiency (AMD), acid alpha-glucosidase deficiency, and lysosomal alpha-glucosidase deficiency), and more specifically relates to inducing inclusion of exon 2 and thereby restoring levels of enzymatically active acid alpha-glucosidase (GAA) protein encoded by the GAA gene.

Materials and methods for regulating gene expression using nanoparticles functionalized with antisense oligonucleotides are provided.

Disclosed are biomolecule based bioprobes that exhibit improved water solubility and mono layer-forming properties with substantially little or no aggregation that can appreciably interfere with binding of the bioprobes to a target nucleotide. The bioprobes may be used in conjunction with a suitable reporter system to detect very small quantities of biological markers. The bio-probes comprise a nucleobase sequence capable of hybridizing to a target nucleotide; and at least one charged functional group attached to said nucleobase sequence. Also disclosed are biosensors, and sensing devices that comprise the bin-probe. Further disclosed are suitable electrochemical reporter systems for use with the bioprobes. Methods of use of these devices and probes, including for the detection of target biomarkers, including biomarkers for cancer cells or pathogens, are also included.

A method of reducing the level of a transcription product in a cell comprising contacting with the cell a composition comprising a double-stranded nucleic acid complex comprising a first nucleic acid strand annealed to a second nucleic acid strand, wherein: (i) the first nucleic acid strand hybridizes to the transcription product and comprises (a) a region consisting of at least 4 consecutive nucleotides that are recognized by RNase H when the strand is hybridized to the transcription product, (b) one or more nucleotide analogs located on 5′ terminal side of the region, (c) one or more nucleotide analogs located on 3′ terminal side of the region and (d) a total number of nucleotides and nucleotide analogs ranging from 8 to 35 nucleotides and (ii) the second nucleic acid strand comprises (a) nucleotides and optionally nucleotide analogs and (b) at least 4 consecutive RNA nucleotides.