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5 results about "Antisense therapy" patented technology
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Antisense therapy is a form of treatment for genetic disorders or infections. When the genetic sequence of a particular gene is known to cause a particular disease, it is possible to synthesize a strand of nucleic acid (DNA, RNA or a chemical analogue) that will bind to the messenger RNA (mRNA) produced by that gene and inactivate it, effectively turning that gene "off". This is because mRNA has to be single stranded for it to be translated. Alternatively, the strand might be targeted to bind a splicing site on pre-mRNA and modify the exon content of an mRNA.
Methods and compositions for treating epilepsy
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.
Owner:UNIQURE FRANCE
Antisense therapeutics for treatment of repeat expansion disorders
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.
Owner:HELEXVA INC
Methods and compositions for treating epilepsy
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.
Owner:UNIQURE FRANCE
Pre-mrna slice modulating peptide-conjugated antisense therapeutics for the treatment of diseases
PendingEP4469576A4Polypeptide with localisation/targeting motifSplicing alterationDiseaseCell biology
The present disclosure relates generally to DG9 peptide-conjugated splice modulating therapies for the treatment of Duchenne muscular dystrophy and spinal muscular atrophy.
Owner:THE GOVERNORS OF THE UNIV OF ALBERTA +1
Antisense therapy
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.
Owner:THE UNIV COURT OF THE UNIV OF EDINBURGH