Patents by Inventor Charleen Hunt
Charleen Hunt has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).
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Publication number: 20240093316Abstract: Methods and compositions are provided herein for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo and ex vivo. The methods and compositions employ cells and non-human animals comprising a Cas expression cassette such as a genomically integrated Cas expression cassette so that the Cas protein can be constitutively available or available in a tissue-specific or temporal-specific manner. Methods and compositions are also provided for making and using these non-human animals, including use of these non-human animals to assess CRISPR/Cas activity in vivo via adeno-associated virus (AAV)-mediated delivery of guide RNAs to the non-human animals.Type: ApplicationFiled: November 27, 2023Publication date: March 21, 2024Applicant: Regeneron Pharmaceuticals, Inc.Inventors: Guochun Gong, Charleen Hunt, Daisuke Kajimura, Suzanne Hartford, Brian Zambrowicz
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Patent number: 11866794Abstract: Methods and compositions are provided herein for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo and ex vivo. The methods and compositions employ cells and non-human animals comprising a Cas expression cassette such as a genomically integrated Cas expression cassette so that the Cas protein can be constitutively available or available in a tissue-specific or temporal-specific manner. Methods and compositions are also provided for making and using these non-human animals, including use of these non-human animals to assess CRISPR/Cas activity in vivo via adeno-associated virus (AAV)-mediated delivery of guide RNAs to the non-human animals.Type: GrantFiled: August 24, 2021Date of Patent: January 9, 2024Assignee: Regeneron Pharmaceuticals, Inc.Inventors: Guochun Gong, Charleen Hunt, Daisuke Kajimura, Suzanne Hartford, Brian Zambrowicz
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Patent number: 11730150Abstract: Provided are non-human animals comprising a mutation in the Fbn1 gene to model neonatal progeroid syndrome with congenital lipodystrophy (NPSCL). Also provided are methods of making such non-human animal models. The non-human animal models can be used for screening compounds for activity in inhibiting or reducing NPSCL or ameliorating NPSCL-like symptoms or screening compounds for activity potentially harmful in promoting or exacerbating NPSCL as well as to provide insights in to the mechanism of NPSCL and potentially new therapeutic and diagnostic targets.Type: GrantFiled: December 17, 2019Date of Patent: August 22, 2023Assignee: Regeneron Pharmaceuticals, Inc.Inventors: Charleen Hunt, Jason Mastaitis, Guochun Gong, Ka-Man Venus Lai, Jesper Gromada, Aris N. Economides
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Publication number: 20230123296Abstract: Non-human animal cells and non-human animals comprising CRISPR/Cas synergistic activation mediator system components and methods of making and using such non-human animal cells and non-human animals are provided. Methods are provided for using such non-human animals to increase expression of target genes in vivo and to assess CRISPR/Cas synergistic activation mediator systems for the ability to increase expression of target genes in vivo.Type: ApplicationFiled: November 1, 2022Publication date: April 20, 2023Applicant: Regeneron Pharmaceuticals, Inc.Inventors: Charleen Hunt, Suzanne Hartford, Guochun Gong, Brian Zambrowicz
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Publication number: 20230123787Abstract: This disclosure relates to an animal model of human disease. More specifically, this disclosure relates to a rodent model of mood disorders such as unipolar depression and an anxiety disorder. Disclosed herein are genetically modified rodent animals that carry a humanized G protein-coupled receptor 156 (GPR156) gene that encodes a mutant human GPR156 protein comprising Asp at an amino acid position corresponding to position 533 in a full length wild type human GPR156 protein.Type: ApplicationFiled: August 10, 2022Publication date: April 20, 2023Applicant: REGENERON PHARMACEUTICALS, INC.Inventors: Meghan Drummond Samuelson, Brian Zambrowicz, Ka-Man Venus Lai, Charleen Hunt, Susannah Brydges, Andrew J. Murphy, Claudia Gonzaga-Jauregui, Jose Rojas, Nicole Alessandri-Haber, Robert Breese, Susan D. Croll
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Publication number: 20230102342Abstract: Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized TTR locus comprising a V30M mutation and methods of making and using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized TTR locus express a human TTR protein or a chimeric TTR protein, fragments of which are from human TTR. Methods are provided for using such non-human animals comprising a humanized TTR locus to assess in vivo efficacy of human-TTR-targeting reagents such as nuclease agents designed to target human TTR.Type: ApplicationFiled: March 23, 2021Publication date: March 30, 2023Applicant: Regeneron Pharmaceuticals, Inc.Inventors: Meghan Drummond Samuelson, Jeffery Haines, Charleen Hunt, Guochun Gong, Brian Zambrowicz
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Publication number: 20230001019Abstract: Nucleic acid constructs and compositions that allow insertion and/or expression of a retinoschisin coding sequence are provided. Nuclease agents targeting RS1 loci are provided. Compositions and methods of using such constructs for integration into a target genomic locus and/or expression in a cell are also provided. Methods of treating X-linked juvenile retinoschisis using the nucleic acid constructs and compositions are also provided.Type: ApplicationFiled: November 7, 2020Publication date: January 5, 2023Applicant: Regeneron Pharmaceuticals, Inc.Inventors: Charleen Hunt, Yang Liu, Guochun Gong, Carmelo Romano, Brian Zambrowicz
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Patent number: 11519004Abstract: Non-human animal cells and non-human animals comprising CRISPR/Cas synergistic activation mediator system components and methods of making and using such non-human animal cells and non-human animals are provided. Methods are provided for using such non-human animals to increase expression of target genes in vivo and to assess CRISPR/Cas synergistic activation mediator systems for the ability to increase expression of target genes in vivo.Type: GrantFiled: March 19, 2019Date of Patent: December 6, 2022Assignee: Regeneran Pharmaceuticals, Inc.Inventors: Charleen Hunt, Suzanne Hartford, Guochun Gong, Brian Zambrowicz
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Patent number: 11470828Abstract: This disclosure relates to an animal model of human disease. More specifically, this disclosure relates to a rodent model of mood disorders such as unipolar depression and an anxiety disorder. Disclosed herein are genetically modified rodent animals that carry a humanized G protein-coupled receptor 156 (GPR156) gene that encodes a mutant human GPR156 protein comprising Asp at an amino acid position corresponding to position 533 in a full length wild type human GPR156 protein.Type: GrantFiled: January 16, 2020Date of Patent: October 18, 2022Assignee: Regeneran Pharmaceuticals, Inc.Inventors: Meghan Drummond Samuelson, Brian Zambrowicz, Ka-Man Venus Lai, Charleen Hunt, Susannah Brydges, Andrew J. Murphy, Claudia Gonzaga-Jauregui, Jose Rojas, Nicole Alessandri-Haber, Robert Breese, Susan D. Croll
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Publication number: 20210392862Abstract: Methods and compositions are provided herein for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo and ex vivo. The methods and compositions employ cells and non-human animals comprising a Cas expression cassette such as a genomically integrated Cas expression cassette so that the Cas protein can be constitutively available or available in a tissue-specific or temporal-specific manner. Methods and compositions are also provided for making and using these non-human animals, including use of these non-human animals to assess CRISPR/Cas activity in vivo via adeno-associated virus (AAV)-mediated delivery of guide RNAs to the non-human animals.Type: ApplicationFiled: August 24, 2021Publication date: December 23, 2021Applicant: Regeneron Pharmaceuticals, Inc.Inventors: Guochun Gong, Charleen Hunt, Daisuke Kajimura, Suzanne Hartford, Brian Zambrowicz
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Patent number: 11130999Abstract: Methods and compositions are provided herein for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo and ex vivo. The methods and compositions employ cells and non-human animals comprising a Cas expression cassette such as a genomically integrated Cas expression cassette so that the Cas protein can be constitutively available or available in a tissue-specific or temporal-specific manner. Methods and compositions are also provided for making and using these non-human animals, including use of these non-human animals to assess CRISPR/Cas activity in vivo via adeno-associated virus (AAV)-mediated delivery of guide RNAs to the non-human animals.Type: GrantFiled: July 31, 2018Date of Patent: September 28, 2021Assignee: Regeneron Pharmaceuticals, Inc.Inventors: Guochun Gong, Charleen Hunt, Daisuke Kajimura, Suzanne Hartford, Brian Zambrowicz
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Publication number: 20210261985Abstract: Methods and compositions are provided for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo or ex vivo. The methods and compositions employ non-human animals comprising a CRISPR reporter such as a genomically integrated CRISPR reporter for detecting and measuring targeted excision of a sequence between two CRISPR/Cas nuclease cleavage sites or disruption of a sequence near a CRISPR/Cas nuclease cleavage site and/or measuring CRISPR/Cas-induced recombination of the CRISPR reporter with an exogenous donor nucleic acid to convert the coding sequence for a first reporter protein to the coding sequence for a different second reporter protein. Methods and compositions are also provided for making and using these non-human animals.Type: ApplicationFiled: April 30, 2021Publication date: August 26, 2021Applicant: Regeneron Pharmaceuticals, Inc.Inventors: Guochun Gong, Charleen Hunt, Susannah Brydges, Suzanne Hartford, David Frendewey, Brian Zambrowicz, Andrew J. Murphy
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Patent number: 11021719Abstract: Methods and compositions are provided for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo or ex vivo. The methods and compositions employ non-human animals comprising a CRISPR reporter such as a genomically integrated CRISPR reporter for detecting and measuring targeted excision of a sequence between two CRISPR/Cas nuclease cleavage sites or disruption of a sequence near a CRISPR/Cas nuclease cleavage site and/or measuring CRISPR/Cas-induced recombination of the CRISPR reporter with an exogenous donor nucleic acid to convert the coding sequence for a first reporter protein to the coding sequence for a different second reporter protein. Methods and compositions are also provided for making and using these non-human animals.Type: GrantFiled: July 31, 2018Date of Patent: June 1, 2021Assignee: Regeneron Pharmaceuticals, Inc.Inventors: Guochun Gong, Charleen Hunt, Susannah Brydges, Suzanne Hartford, David Frendewey, Brian Zambrowicz, Andrew J. Murphy
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Publication number: 20210079394Abstract: Lipid nanoparticles comprising CRISPR/Cas synergistic activation mediator system components together in the same lipid nanoparticle and methods of using such lipid nanoparticles to increase expression of target genes in vivo and ex vivo and to assess CRISPR/Cas synergistic activation mediator systems for the ability to increase expression of target genes in vivo and ex vivo are provided.Type: ApplicationFiled: September 11, 2020Publication date: March 18, 2021Inventors: CHARLEEN HUNT, Suzanne Hartford, Guochun Gong, Brian Zambrowicz
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Publication number: 20200229409Abstract: This disclosure relates to an animal model of human disease. More specifically, this disclosure relates to a rodent model of mood disorders such as unipolar depression and an anxiety disorder. Disclosed herein are genetically modified rodent animals that carry a humanized G protein-coupled receptor 156 (GPR156) gene that encodes a mutant human GPR156 protein comprising Asp at an amino acid position corresponding to position 533 in a full length wild type human GPR156 protein.Type: ApplicationFiled: January 16, 2020Publication date: July 23, 2020Inventors: Meghan Drummond Samuelson, Brian Zambrowicz, Ka-Man Venus Lai, Charleen Hunt, Susannah Brydges, Andrew J. Murphy, Claudia Gonzaga-Jauregui, Jose Rojas, Nicole Alessandri-Haber, Robert Breese, Susan D. Croll
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Publication number: 20200107527Abstract: Provided are non-human animals comprising a mutation in the Fbn1 gene to model neonatal progeroid syndrome with congenital lipodystrophy (NPSCL). Also provided are methods of making such non-human animal models. The non-human animal models can be used for screening compounds for activity in inhibiting or reducing NPSCL or ameliorating NPSCL-like symptoms or screening compounds for activity potentially harmful in promoting or exacerbating NPSCL as well as to provide insights in to the mechanism of NPSCL and potentially new therapeutic and diagnostic targets.Type: ApplicationFiled: December 17, 2019Publication date: April 9, 2020Applicant: Regeneron Pharmaceuticals, Inc.Inventors: Charleen Hunt, Jason Mastaitis, Guochun Gong, Ka-Man Venus Lai, Jesper Gromada, Aris N. Economides
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Patent number: 10548302Abstract: Provided are non-human animals comprising a mutation in the Fbn1 gene to model neonatal progeroid syndrome with congenital lipodystrophy (NPSCL). Also provided are methods of making such non-human animal models. The non-human animal models can be used for screening compounds for activity in inhibiting or reducing NPSCL or ameliorating NPSCL-like symptoms or screening compounds for activity potentially harmful in promoting or exacerbating NPSCL as well as to provide insights in to the mechanism of NPSCL and potentially new therapeutic and diagnostic targets.Type: GrantFiled: July 28, 2017Date of Patent: February 4, 2020Assignee: Regeneron Pharmaceuticals, Inc.Inventors: Charleen Hunt, Jason Mastaitis, Guochun Gong, Ka-Man Venus Lai, Jesper Gromada, Aris N. Economides
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Publication number: 20190284572Abstract: Non-human animal cells and non-human animals comprising CRISPR/Cas synergistic activation mediator system components and methods of making and using such non-human animal cells and non-human animals are provided. Methods are provided for using such non-human animals to increase expression of target genes in vivo and to assess CRISPR/Cas synergistic activation mediator systems for the ability to increase expression of target genes in vivo.Type: ApplicationFiled: March 19, 2019Publication date: September 19, 2019Inventors: Charleen Hunt, Suzanne Hartford, Guochun Gong, Brian Zambrowicz
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Publication number: 20190032156Abstract: Methods and compositions are provided for assessing CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo or ex vivo. The methods and compositions employ non-human animals comprising a CRISPR reporter such as a genomically integrated CRISPR reporter for detecting and measuring CRISPR/Cas-induced repair of a coding sequence for a catalytically inactive reporter protein through recombination with an exogenous donor nucleic acid. Methods and compositions are also provided for making and using these non-human animals.Type: ApplicationFiled: July 31, 2018Publication date: January 31, 2019Inventors: Guochun Gong, Charleen Hunt, Suzanne Hartford, Jose Rojas, David Frendewey, Brian Zambrowicz, Andrew J. Murphy
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Publication number: 20190032092Abstract: Methods and compositions are provided for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo or ex vivo. The methods and compositions employ non-human animals comprising a CRISPR reporter such as a genomically integrated CRISPR reporter for detecting and measuring targeted excision of a sequence between two CRISPR/Cas nuclease cleavage sites or disruption of a sequence near a CRISPR/Cas nuclease cleavage site and/or measuring CRISPR/Cas-induced recombination of the CRISPR reporter with an exogenous donor nucleic acid to convert the coding sequence for a first reporter protein to the coding sequence for a different second reporter protein. Methods and compositions are also provided for making and using these non-human animals.Type: ApplicationFiled: July 31, 2018Publication date: January 31, 2019Inventors: Guochun Gong, Charleen Hunt, Susannah Brydges, Suzanne Hartford, David Frendewey, Brian Zambrowicz, Andrew J. Murphy