14 patents
Utility
Regulation of Gene Expression by Aptamer-modulated Rnase P Cleavage
4 Jan 24
The present disclosure provides polynucleotide constructs for the modulation of target gene expression by aptamer-mediated ribonuclease cleavage of the target gene RNA and methods of using the constructs to modulate gene expression in response to the presence or absence of a ligand that binds the aptamer.
Xuecui Guo
Filed: 19 Jul 23
Utility
Regulation of Gene Expression Via Aptamer-mediated Control of Self-cleaving Ribozymes
13 Jul 23
The invention provides polynucleotide constructs for the regulation of gene expression by aptamer-based modulation of self-cleaving ribozymes and methods of using the constructs to regulate gene expression in response to the presence or absence of a ligand that binds the aptamer.
Michael J. Volles, Olivier F. Danos, Alex R. Boyne, Veronique Zennou, Xuecui Guo
Filed: 29 Nov 22
Utility
Regulation of gene expression through aptamer-modulated polyadenylation
11 Jul 23
The invention provides polynucleotide constructs for the regulation of gene expression by aptamer-based modulation of U1 small nuclear ribonucleoprotein (snRNP)-mediated suppression of polyadenylation and methods of using the constructs to regulate gene expression in response to the presence or absence of a ligand that binds the aptamer.
Michael J. Volles, Olivier F. Danos, Xuecui Guo
Filed: 2 Feb 17
Utility
High Throughput Cell-Based Screening for Aptamers
2 Mar 23
The invention provides eukaryotic cell-based screening methods to identify an aptamer that specifically binds a ligand, or a ligand that specifically binds an aptamer, using a polynucleotide cassette for the regulation of the expression of a reporter gene where the polynucleotide cassette contains a riboswitch in the context of a 5′ intron-alternative exon-3′ intron.
Xuecui Guo, Alexandria Forbes, Lei Feng
Filed: 27 Jun 22
Utility
Methods for Treating Parkinson's Disease
16 Feb 23
Disclosed are methods for treating neurological disorders such as Parkinson's disease (PD) using glutamic acid decarboxylase (GAD) and identifying PD patients that will be most receptive to the method of treating PD.
Alexandria Forbes, Matthew During
Filed: 14 Dec 20
Utility
Stabilization of Retromer for the Treatment of Alzheimer's Disease and Other Neurodegenerative Disorders
26 Jan 23
The present disclosure relates to methods and compositions for elevating and stabilizing retromer for treating and/or preventing Alzheimer's disease and other neurodegenerative disorders.
Scott A. SMALL, Gregory PETSKO, Rebecca COX, Samuel D. WAKSAL, Alexandria FORBES, Yasir H. QURESHI
Filed: 7 Dec 20
Utility
Injection Systems and Methods of Their Use
1 Dec 22
An injection system comprising a syringe barrel; a first sealing element and a second sealing element moveably disposed in the syringe barrel; an injection chamber between them; a puncture element extending from the first sealing element to deliver an injection agent from the injection chamber into a biological space, wherein one or more of the syringe barrel, the first or the second sealing element are configured to prevent proximal movement of the first sealing element past a pre-selected location, while allowing the second sealing element to come in contact with the first sealing element, the system is configured such that, when a force is applied on the second sealing element in a distal direction, in response to a first opposing force, the puncture element advances and in response to a second opposing force, the puncture element remains stationary and the injection agent is conveyed through the puncture element.
Girish Chitnis, Jeff Karp, Edward Ahn, Bryan Laulicht
Filed: 20 Sep 20
Utility
Regulation of gene expression via aptamer-mediated control of self-cleaving ribozymes
29 Nov 22
The invention provides polynucleotide constructs for the regulation of gene expression by aptamer-based modulation of self-cleaving ribozymes and methods of using the constructs to regulate gene expression in response to the presence or absence of a ligand that binds the aptamer.
Michael J. Volles, Olivier F. Danos, Alex R. Boyne, Veronique Zennou, Xuecui Guo
Filed: 2 Feb 17
Utility
Regulation of Gene Expression by Aptamer-mediated Modulation of Alternative Splicing
8 Sep 22
The invention provides a platform and methods of using the platform for the regulation of the expression of a target gene using exposure to an aptamer ligand (for example, a small molecule).
Alex R. Boyne, Olivier F. Danos, Michael J. Volles, Xuecui Guo
Filed: 14 Feb 22
Utility
Modified AAV Capsid Proteins for Treatment of Arthritic Disease
25 Aug 22
The invention relates to recombinant adeno-associated virus (rAAV) virions for gene therapy, wherein the rAAV virions comprise a novel capsid protein.
Sabine Maria Gertrude Van Der Sanden, Susanne Anna Snoek, Niels Broekstra, Jonathan Douglas Finn, Dirk Grimm, Kathleen Börner
Filed: 15 Jul 20
Utility
Regulation of gene expression by aptamer-mediated modulation of alternative splicing
15 Feb 22
The invention provides a platform and methods of using the platform for the regulation of the expression of a target gene using exposure to an aptamer ligand (for example, a small molecule).
Alex R. Boyne, Olivier F. Danos, Michael J. Volles, Xuecui Guo
Filed: 22 Nov 19
Utility
Modified Raav Capsid Protein for Gene Therapy
11 Nov 20
The invention relates to recombinant adeno-associated virus (rAAV) virions for gene therapy, wherein the rAAV virions comprise a novel capsid protein.
Jonathan Douglas Finn, Dirk Grimm, Kathleen Börner, Niels Broekstra, Susanne Anna Snoek, Sabine Maria Gertrude Van Der Sanden
Filed: 16 Jan 19
Utility
High Throughput Cell-Based Screening For Aptamers
29 Jul 20
The invention provides eukaryotic cell-based screening methods to identify an aptamer that specifically binds a ligand, or a ligand that specifically binds an aptamer, using a polynucleotide cassette for the regulation of the expression of a reporter gene where the polynucleotide cassette contains a riboswitch in the context of a 5′ intron-alternative exon-3′ intron.
Xuecui Guo, Alexandria Forbes, Lei Feng
Filed: 2 Aug 17
Utility
Regulation of Gene Expression by Aptamer-mediated Modulation of Alternative Splicing
18 Mar 20
The invention provides a platform and methods of using the platform for the regulation of the expression of a target gene using exposure to an aptamer ligand (for example, a small molecule).
Alex R. Boyne, Olivier F. Danos, Michael J. Volles, Xuecui Guo
Filed: 21 Nov 19
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