20 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
OPA1 Antisense Oligomers for Treatment of Conditions and Diseases
14 Sep 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: 30 Apr 21
Utility
OPA1 Antisense Oligomers for Treatment of Conditions and Diseases
10 Aug 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 for Treatment of Conditions and Diseases
13 Apr 23
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, Anne Christiansen, Meena Meena, Baruch Ticho, Gene Liau
Filed: 3 Jun 22
Utility
Compositions and Methods for Modulating Splicing and Protein Expression
15 Sep 22
Alternative splicing events 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 diseased patients and/or inhibit aberrant protein expression.
Isabel Aznarez
Filed: 16 Feb 22
Utility
Methods and Compositions for Modulating Splicing of Alternative Introns
26 May 22
Provided herein are methods and compositions for modulating expression of a target protein or a target RNA by modulating splicing pre-mRNA and for treating diseases or conditions associated with expression level of the target protein or the target RNA.
Isabel Aznarez
Filed: 3 Nov 21
Utility
Antisense Oligomers for Treatment of Conditions and Diseases
26 May 22
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
Filed: 26 Aug 21
Utility
Methods and Compositions for Modulating Splicing and Translation
28 Apr 22
Alternative splicing events in genes can lead to non-productive or less productive mRNA transcripts, 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, Juergen Scharner
Filed: 3 Nov 21
Utility
Antisense Oligomers and Uses Thereof
21 Apr 22
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: 19 Jul 21
Utility
Antisense Oligomers for Treatment of Autosomal Dominant Mental RETARDATION-5 and Dravet Syndrome
24 Mar 22
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: 23 Jun 21
Utility
Methods and Compositions for Treatment of Cholesteryl Ester Storage Disease
2 Dec 21
Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient LAL protein expression or a subject having Cholesteryl Ester Storage Disease.
Isabel AZNAREZ
Filed: 3 May 19
Utility
Antisense Oligomers for Treatment of Conditions and Diseases
7 Oct 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: 18 Dec 20
Utility
Antisense Oligomers for Treatment of Non-sense Mediated Rna Decay Based Conditions and Diseases
2 Sep 21
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, Enxuan JING, Jacob KACH, Aditya VENKATESH, Juergen SCHARNER, Baruch TICHO, Gene LIAU
Filed: 23 Oct 18
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
Utility
Antisense Oligomers for Treatment of Conditions and Diseases
22 Jan 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
Utility
Antisense Oligomers for Treatment of Conditions and Diseases
22 Jan 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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