10 results about "Antisense therapy" patented technology

Disclosed are methods and compositions relating to antisense therapy for treating epilepsy, such as a focal epilepsy and temporal lobe epilepsy, in a subject in need thereof by targeting GRIK2 mRNA. In particular, the disclosure provides methods for treating symptoms (e.g., seizures) of epilepsy in a subject by administering a particular dose in a defined volume and in a specific route of administration of an inhibitory ribopolynucleotide or adeno-associated viral vector encoding the same, which is capable of inhibiting expression of GRIK2.

The present disclosure relates to compounds useful for modulation of target nucleic acids, including DNA and RNA. The present disclosure further relates to methods for treatment of repeat expansion disorders, which can include administration of compounds provided herein that can bind pathogenic nucleotide repeats in DNA or RNA.

An isolated or purified antisense oligomer targeted to a nucleic acid molecule encoding a PTPN1 pre-mRNA, wherein the antisense oligomer inhibits expression of PTP1B.

Disclosed are methods and compositions relating to antisense therapy for treating epilepsy, such as a focal epilepsy and temporal lobe epilepsy, in a subject in need thereof by targeting GRIK2 mRNA. In particular, the disclosure provides methods for treating symptoms (e.g., seizures) of epilepsy in a subject by administering a particular dose in a defined volume and in a specific route of administration of an inhibitory ribopolynucleotide or adeno-associated viral vector encoding the same, which is capable of inhibiting expression of GRIK2.

The present disclosure relates generally to DG9 peptide-conjugated splice modulating therapies for the treatment of Duchenne muscular dystrophy and spinal muscular atrophy.

Disclosed are a carbocyclic nucleoside, an oligonucleotide and preparation methods therefor and medical uses thereof, and the carbocyclic nucleoside is a compound or salt shown in the following Formula (I). The oligonucleotide or pharmaceutically acceptable salt thereof provided in the present disclosure can be used for preparing a drug for gene therapy, gene vaccination, antisense therapy, short interfering RNA, or nucleic acid transfer. The oligonucleotide in the present disclosure has high binding affinity to single-stranded RNA, increase stability to nuclease degradation and plasma stability.

The invention discloses carbocyclic nucleoside, oligonucleotide and a preparation method and medical application of the carbocyclic nucleoside and oligonucleotide. The carbocyclic nucleoside is a compound or salt as shown in a formula (I). The carbocyclic nucleoside or oligonucleotide of the present invention has the use in the preparation of drugs for gene therapy, gene vaccination, antisense therapy, interfering RNA or nucleic acid transfer. The oligonucleotide provided by the invention has relatively high binding affinity, nuclease resistance and plasma stability to single-stranded RNA. # imgabs0 #

Disclosed in the present invention are a carbocyclic nucleoside, an oligonucleotide, a preparation method therefor, and medical uses thereof. The carbocyclic nucleoside is a compound or salt represented by formula (I). The carbocyclic nucleoside or oligonucleotide of the present invention is used in the preparation of a drug used for gene therapy, genetic vaccination, antisense therapy, interfering RNA, or nucleic acid transfer. The oligonucleotide of the present invention has high binding affinity to single-stranded RNA, and has nuclease resistance and plasma stability.

The present disclosure provides methods, compounds (molecules) and / or compositions for the treatment and / or prevention of diseases and / or disorders caused or contributed by missplicing of a downstream target gene of TAR DNA binding protein 43 (TDP-43) dependent TDP-43.

The invention provides compositions useful to knock down overexpression of UBE3A and treat conditions associated with Dup15q syndrome. The compositions include antisense oligonucleotides, preferably short oligonucleotides that are complementary to, and hybridize to, UBE3A transcripts in vivo. The ASOs prevent or inhibit successful translation of UBE3A mRNA into protein. Specifically, preferred embodiments include anti-UBE3A gapmers—oligos that include a central DNA portion flanked by RNA wings. When the gapmer hybridizes to UBE3A pre-mRNA or mRNA, the duplex hybrid recruits RNaseH, which cleaves, or digests, the UBE3A pre-mRNA or mRNA, preventing expression of the UBE3A protein. Because the ASOs prevent expression of the UBE3A protein, treatment with a composition including ASOs of the disclosure may be effective to knock down overexpression of UBE3A.