Patents by Inventor Rasmus O. Bak
Rasmus O. Bak 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: 20240124895Abstract: In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.Type: ApplicationFiled: December 5, 2022Publication date: April 18, 2024Applicant: The Board of Trustees of the Leland Stanford Junior UniversityInventors: Daniel P. Dever, Rasmus O. Bak, Ayal Hendel, Waracharee Srifa, Matthew H. Porteus
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Patent number: 11851652Abstract: Provided herein are methods for inducing CRISPR/Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.Type: GrantFiled: March 14, 2022Date of Patent: December 26, 2023Assignees: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR, UNIVERSITY and AGILENT TECHNOLOGIES, INC.Inventors: Matthew H. Porteus, Ayal Hendel, Joe Clark, Rasmus O. Bak, Daniel E. Ryan, Douglas J. Dellinger, Robert Kaiser, Joel Myerson
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Patent number: 11773409Abstract: The present invention relates to a system and method for efficiently modifying the genome of cells to treat diseases via sequential homologous recombination using CRISPR/Cas-mediated genome editing with donor DNA delivered by two or more adeno-associated virus (AAV) vectors.Type: GrantFiled: April 23, 2018Date of Patent: October 3, 2023Assignee: The Board of Trustees of the Leland Stanford Junior UniversityInventors: Rasmus O. Bak, Matthew Porteus, Sriram Vaidyanathan
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Patent number: 11634732Abstract: In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.Type: GrantFiled: October 15, 2021Date of Patent: April 25, 2023Assignee: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITYInventors: Daniel P. Dever, Rasmus O. Bak, Ayal Hendel, Waracharee Srifa, Matthew H. Porteus
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Patent number: 11535846Abstract: Provided herein are methods for inducing CRISPR/Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.Type: GrantFiled: March 14, 2022Date of Patent: December 27, 2022Assignees: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY, AGILENT TECHNOLOGIES, INC.Inventors: Matthew H. Porteus, Ayal Hendel, Joe Clark, Rasmus O. Bak, Daniel E. Ryan, Douglas J. Dellinger, Robert Kaiser, Joel Myerson
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Patent number: 11492646Abstract: In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.Type: GrantFiled: October 15, 2021Date of Patent: November 8, 2022Assignee: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITYInventors: Daniel P. Dever, Rasmus O. Bak, Ayal Hendel, Waracharee Srifa, Matthew H. Porteus
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Publication number: 20220195427Abstract: Provided herein are methods for inducing CRISPR/Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.Type: ApplicationFiled: March 14, 2022Publication date: June 23, 2022Applicants: The Board of Trustees of the Leland Stanford Junior University, Agilent Technologies, Inc.Inventors: Matthew H. Porteus, Ayal Hendel, Joe Clark, Rasmus O. Bak, Daniel E. Ryan, Douglas J. Dellinger, Robert Kaiser, Joel Myerson
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Publication number: 20220195425Abstract: Provided herein are methods for inducing CRISPR/Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.Type: ApplicationFiled: March 14, 2022Publication date: June 23, 2022Applicants: The Board of Trustees of the Leland Stanford Junior University, Agilent Technologies, Inc.Inventors: Matthew H. Porteus, Ayal Hendel, Joe Clark, Rasmus O. Bak, Daniel E. Ryan, Douglas J. Dellinger, Robert Kaiser, Joel Myerson
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Publication number: 20220195426Abstract: Provided herein are methods for inducing CRISPR/Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.Type: ApplicationFiled: March 14, 2022Publication date: June 23, 2022Applicants: The Board of Trustees of the Leland Stanford Junior Univerisity, Agilent Technologies, Inc.Inventors: Matthew H. Porteus, Ayal Hendel, Joe Clark, Rasmus O. Bak, Daniel E. Ryan, Douglas J. Dellinger, Robert Kaiser, Joel Myerson
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Patent number: 11306309Abstract: Provided herein are methods for inducing CRISPR/Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.Type: GrantFiled: October 3, 2017Date of Patent: April 19, 2022Assignees: The Board of Trustees of the Leland Stanford Junior University, Agilent TechnologiesInventors: Matthew H. Porteus, Ayal Hendel, Joe Clark, Rasmus O. Bak, Daniel E. Ryan, Douglas J. Dellinger, Robert Kaiser, Joel Myerson
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Publication number: 20220064676Abstract: In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.Type: ApplicationFiled: October 15, 2021Publication date: March 3, 2022Applicant: The Board of Trustees of the Leland Stanford Junior UniversityInventors: Daniel P. Dever, Rasmus O. Bak, Ayal Hendel, Waracharee Srifa, Matthew H. Porteus
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Publication number: 20220025409Abstract: In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.Type: ApplicationFiled: October 15, 2021Publication date: January 27, 2022Applicant: The Board of Trustees of the Leland Stanford Junior UniversityInventors: Daniel P. Dever, Rasmus O. Bak, Ayal Hendel, Waracharee Srifa, Matthew H. Porteus
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Publication number: 20220025408Abstract: In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.Type: ApplicationFiled: October 15, 2021Publication date: January 27, 2022Applicant: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITYInventors: DANIEL P. DEVER, RASMUS O. BAK, AYAL HENDEL, WARACHAREE SRIFA, MATTHEW H. PORTEUS
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Patent number: 11193141Abstract: In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.Type: GrantFiled: March 21, 2018Date of Patent: December 7, 2021Assignee: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITYInventors: Daniel P. Dever, Rasmus O. Bak, Ayal Hendel, Waracharee Srifa, Matthew H. Porteus
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Publication number: 20200131539Abstract: The present invention relates to a system and method for efficiently modifying the genome of cells to treat diseases via sequential homologous recombination using CRISPR/Cas-mediated genome editing with donor DNA delivered by two or more adeno-associated virus (AAV) vectors.Type: ApplicationFiled: April 23, 2018Publication date: April 30, 2020Inventors: Rasmus O. Bak, Matthew Porteus, Sriram Vaidyanathan
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Publication number: 20190032091Abstract: In certain aspects, the present invention provides methods for inducing a stable gene modification of a target nucleic acid via homologous recombination in a primary cell, such as a primary blood cell and/or a primary mesenchymal cell. In certain other aspects, the present invention provides methods for enriching a population of genetically modified primary cells having targeted integration at a target nucleic acid. The methods of the present invention rely on the introduction of a DNA nuclease such as a Cas polypeptide and a homologous donor adeno-associated viral (AAV) vector into the primary cell to mediate targeted integration of the target nucleic acid. Also provided herein are methods for preventing or treating a disease in a subject in need thereof by administering to the subject any of the genetically modified primary cells or pharmaceutical compositions described herein to prevent the disease or ameliorate one or more symptoms of the disease.Type: ApplicationFiled: March 21, 2018Publication date: January 31, 2019Applicant: The Board of Trustees of the Leland Stanford Junior UniversityInventors: Daniel P. Dever, Rasmus O. Bak, Ayal Hendel, Waracharee Srifa, Matthew H. Porteus
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Publication number: 20180119140Abstract: Provided herein are methods for inducing CRISPR/Cas-based gene regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using modified single guide RNAs (sgRNAs) that enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a modified sgRNA to correct a mutation in a target gene associated with the genetic disease.Type: ApplicationFiled: October 3, 2017Publication date: May 3, 2018Applicants: The Board of Trustees of the Leland Stanford Junior University, Agilent Technologies, Inc.Inventors: Matthew H. Porteus, Ayal Hendel, Joe Clark, Rasmus O. Bak, Daniel E. Ryan, Douglas J. Dellinger, Robert Kaiser, Joel Myerson