Patents by Inventor Matthew H. PORTEUS
Matthew H. PORTEUS 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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Publication number: 20240093242Abstract: The present disclosure provides methods and compositions for treating SCID-X1 in subjects, comprising genetically modifying cells from the subjects ex vivo by integrating a full-length, codon-optimized IL2RG cDNA at the endogenous IL2RG locus.Type: ApplicationFiled: August 3, 2021Publication date: March 21, 2024Applicant: The Board of Trustees of the Leland Stanford Junior UniversityInventors: Mara Pavel-Dinu, Matthew H. Porteus
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Patent number: 11903969Abstract: Methods and compositions for modifying allogeneic donor ?? T cells for use in the treatment of high risk leukemias are provided.Type: GrantFiled: November 13, 2020Date of Patent: February 20, 2024Assignee: The Board of Trustees of the Leland Stanford Junior UniversityInventors: Matthew H. Porteus, Alice Bertaina, Volker Andreas Wiebking
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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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Publication number: 20230357798Abstract: The present disclosure provides methods and compositions for treating X-CGD in subjects, comprising genetically modifying cells from the subjects ex vivo by integrating a functional, codon-optimized CYBB cDNA at the endogenous CYBB locus.Type: ApplicationFiled: October 12, 2021Publication date: November 9, 2023Applicant: The Board of Trustees of the Leland Stanford Junior UniversityInventors: Mara Pavel-Dinu, Matthew H. Porteus
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Publication number: 20230348932Abstract: A method for scarless genome editing is disclosed. In particular, the method provides scarless genome modification by using homology directed repair (HDR) steps to genetically modify cells and remove unwanted sequences. This method can be used for genome editing, including introducing mutations, deletions, or insertions at any position in the genome without leaving silent mutations, selection marker sequences, or other additional undesired sequences in the genome.Type: ApplicationFiled: May 18, 2023Publication date: November 2, 2023Applicant: The Board of Trustees of the Leland Stanford Junior UniversityInventors: Kazuya Ikeda, Matthew H. Porteus
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Publication number: 20230265440Abstract: The present disclosure provides methods and compositions for treating lysosomal storage disorders in subjects, comprising genetically modifying cells from the subjects ex vivo by integrating therapeutic transgenes into the CCR5 locus.Type: ApplicationFiled: June 25, 2021Publication date: August 24, 2023Applicant: The Board of Trustees of the Leland Stanford Junior UniversityInventors: Natalia Gomez-Ospina, Matthew H. Porteus, Samantha Glynne Scharenberg, Alvaro Amorin
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Publication number: 20230250423Abstract: The invention provides methods for generating a genetically modified human neural stem cell, genetically modified human neural stem cells, and pharmaceutical compositions comprising the genetically modified human neural stem cells. Also provided are associated kits. The invention also provides methods for preventing or treating a neurodegenerative disease or a neurological injury in a human subject using genetically modified human neural stem cells.Type: ApplicationFiled: January 3, 2023Publication date: August 10, 2023Inventors: Matthew H. PORTEUS, Eric J. KILDEBECK, Daniel P. DEVER, Joseph T. CLARK, Ann TSUKAMOTO, Nobuko UCHIDA
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Patent number: 11692202Abstract: A method for scarless genome editing is disclosed. In particular, the method provides scarless genome modification by using homology directed repair (HDR) steps to genetically modify cells and remove unwanted sequences. This method can be used for genome editing, including introducing mutations, deletions, or insertions at any position in the genome without leaving silent mutations, selection marker sequences, or other additional undesired sequences in the genome.Type: GrantFiled: January 10, 2020Date of Patent: July 4, 2023Assignee: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITYInventors: Kazuya Ikeda, Matthew H. Porteus
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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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Publication number: 20230085945Abstract: Compositions and methods are provided for depletion of pluripotent cells. In one embodiment of the invention, methods are provided for depletion of pluripotent cells from a mixed population of differentiated cells and stem cells, to provide a population of cells substantially free of pluripotent stem cells.Type: ApplicationFiled: February 19, 2021Publication date: March 23, 2023Inventors: Matthew H. Porteus, Michael K. Cromer, Renata Martin, Jonas Fowler, Kyle Loh
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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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Publication number: 20220356450Abstract: The present disclosure provides methods and compositions for genetically modifying hematopoietic stem and progenitor cells (HSPCs), in particular by replacing the HBA1 or HBA2 locus in the HSPCs with a transgene encoding a therapeutic protein.Type: ApplicationFiled: May 9, 2022Publication date: November 10, 2022Applicant: THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITYInventors: MATTHEW H PORTEUS, MICHAEL KYLE CROMER, DANIEL P. DEVER, JOAB CAMARENA
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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: 20220280571Abstract: The disclosure provide methods and compositions that use gene editing or gene therapy to treat alpha thalassemia major. The gene editing may be performed ex vivo in fetal cells or cells obtained after birth to improve production of globin, with those cells then delivered to the fetus. In other embodiments, gene editing reagents are delivered to the fetus or the patient after birth in vivo to edit genes of the alpha-globin cluster and improve globin production. Gene editing system such as CRISPR, TALENs, or ZFNs are used to increase production of alpha, zeta, or theta globin and/or to decrease production of gamma globin. Globin production may be improved by inserting a copy of globin gene or mutating a globin gene to change its expression. Any of the gene editing strategies may be performed in conjunction with delivering to a fetus or patient after birth a therapeutic blood transfusion. Exemplary patients after birth are patients no older than one year of age.Type: ApplicationFiled: July 31, 2020Publication date: September 8, 2022Inventors: Tippi MacKenzie, Beeke Wienert, Michael Kyle Cromer, Matthew H. Porteus
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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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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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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