6 patents
Utility
Antisense oligomers for treatment of conditions and diseases
16 Jan 24
Alternative splicing events in SCN1A gene can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in SCN1A gene can modulate the expression level of functional proteins in Dravet Syndrome patients and/or inhibit aberrant protein expression.
Isabel Aznarez, Zhou Han
Filed: 18 Dec 20
Utility
OPA1 antisense oligomers for treatment of conditions and diseases
14 Nov 23
Alternative splicing events in genes can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in genes can modulate the expression level of functional proteins in patients and/or inhibit aberrant protein expression.
Isabel Aznarez, Aditya Venkatesh, Gene Liau
Filed: 21 Apr 23
Utility
Antisense oligomers and uses thereof
24 Aug 21
Provided herein are methods and compositions for increasing the expression of a protein, and for treating a subject in need thereof, e.g., a subject with deficient protein expression or a subject having a disease described herein.
Isabel Aznarez, Huw M. Nash, Adrian Krainer
Filed: 13 Jun 18
Utility
Antisense oligomers for treatment of autosomal dominant mental retardation-5 and Dravet Syndrome
10 Aug 21
Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.
Isabel Aznarez, Huw M. Nash, Adrian Krainer
Filed: 14 Dec 16
Utility
Antisense oligomers for treatment of conditions and diseases
9 Feb 21
Alternative splicing events in SCN1A gene can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in SCN1A gene can modulate the expression level of functional proteins in Dravet Syndrome patients and/or inhibit aberrant protein expression.
Isabel Aznarez, Zhou Han
Filed: 5 Sep 19
Utility
Antisense oligomers for treatment of conditions and diseases
15 Jun 20
Alternative splicing events in SCN1A gene can lead to non-productive mRNA transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in SCN1A gene can modulate the expression level of functional proteins in Dravet Syndrome patients and/or inhibit aberrant protein expression.
Isabel Aznarez, Zhou Han
Filed: 4 Sep 19
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