Patents by Inventor Rhonda Bassel-Duby

Rhonda Bassel-Duby 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).

  • Publication number: 20240226334
    Abstract: Provided herein are gene therapy methods, vectors and constructs for the treatment of Duchenne Muscular Dystrophy in a subject.
    Type: Application
    Filed: May 24, 2022
    Publication date: July 11, 2024
    Applicant: The Board of Regents of The University of Texas System
    Inventors: Eric N. OLSON, Yu ZHANG, Rhonda BASSEL-DUBY
  • Publication number: 20240165271
    Abstract: Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of dystrophin, which maintains muscle membrane integrity. Provided herein are methods of using adenine base editor (ABE) to modify splice sites of the dystrophin gene, causing skipping or refraining of common DMD exon deletion mutations, restoring dystrophin expression. Also provided herein are methods of using prime editing to reframe the dystrophin open reading frame and restore dystrophin expression.
    Type: Application
    Filed: March 25, 2022
    Publication date: May 23, 2024
    Applicant: The Board of Regents of The University of Texas System
    Inventors: Eric N. OLSON, Francesco CHEMELLO, Rhonda BASSEL-DUBY
  • Publication number: 20220072156
    Abstract: Duchenne muscular dystrophy (DMD) is an inherited X-linked disease caused by mutations in the gene encoding dystrophin, a protein required for muscle fiber integrity. The disclosure reports CRISPR/Cas9-mediated gene editing (Myo-editing) is effective at correcting the dystrophin gene mutation in the mdx mice, a model for DMD. Further, the disclosure reports optimization of germline editing of mdx mice by engineering the permanent skipping of mutant exon (exon 23) and extending exon skipping to also correct the disease by post-natal delivery of adeno-associate virus (AAV). AAV-mediated Myo-editing can efficiently rescue the reading frame of dystrophin in mdx mice in vivo. The disclosure reports means of Myo-editing-mediated exon skipping has been successfully advanced from somatic tissues in mice to human DMD patients-derived iPSCs (induced pluripotent stem cells).
    Type: Application
    Filed: August 17, 2021
    Publication date: March 10, 2022
    Applicant: The Board of Regents of the University of Texas System
    Inventors: Eric N. OLSON, Chengzu LONG, John R. McANALLY, John M. SHELTON, Rhonda BASSEL-DUBY
  • Patent number: 11166949
    Abstract: The present disclosure relates to the identification of Nurr1 as a key regulator of metabolism, and the use of Nurr1 agonist to treat metabolic disorders such as diabetes, obesity, metabolic syndrome and hepatic steatosis.
    Type: Grant
    Filed: April 26, 2017
    Date of Patent: November 9, 2021
    Assignee: The Board of Regents of The University of Texas System
    Inventors: Eric N. Olson, Rhonda Bassel-Duby, Leonela Amoasii
  • Publication number: 20210261962
    Abstract: Duchenne muscular dystrophy (DMD), which affects 1 in 5,000 male births, is one of the most common genetic disorders of children. This disease is caused by an absence or deficiency of dystrophin protein in striated muscle. The major DMD deletion “hot spots” are found between exon 6 to 8, and exons 45 to 53. Here, three DMD mouse models are provided that can be used to test a variety of DMD exon skipping and refraining strategies. Among these are, CRISPR/Cas9 oligonucleotides, small molecules or other therapeutic modalities that promote exon skipping or exon refraining or micro dystrophin mini genes or cell based therapies. Methods for restoring the reading frame of exon 43, exon 45, and exon 52 deletion via CRISPR-mediated exon skipping and refraining in the humanized DMD mouse model, in patient-derived iPSCs and ultimately, in patients using various delivery systems are also contemplated. The impact of CRISPR technology on DMD is that gene editing can permanently correct mutations.
    Type: Application
    Filed: June 21, 2019
    Publication date: August 26, 2021
    Applicant: The Board of Regents of the University of Texas System
    Inventors: Yi-Li MIN, Rhonda BASSEL-DUBY, Eric OLSON
  • Publication number: 20210180023
    Abstract: The present disclosure involves the use of reprogramming factors including AKT1, GATA4, TBX5, MEF2C, HAND2 and either ZNF281 or AS-CL1 to reprogram adult non-cardiomyocytes, such as cardiac fibroblasts into cardiomyocytes, both in vitro and in vivo. Such methods find particular use in the treatment of patients post-myocardial infarction to prevent or limit scarring and to promote myocardial repair.
    Type: Application
    Filed: June 4, 2018
    Publication date: June 17, 2021
    Applicant: The Board of Regents of the University of Texas System
    Inventors: Eric N. OLSON, Huanyu ZHOU, Rhonda BASSEL-DUBY
  • Publication number: 20200275641
    Abstract: Duchenne muscular dystrophy (DMD), which affects 1 in 5,000 male births, is one of the most common genetic disorders of children. This disease is caused by an absence or deficiency of dystrophin protein in striated muscle. The major DMD deletion “hot spots” are found between exon 6 to 8, and exons 45 to 53. Here, a “humanized” mouse model is provided that can be used to test a variety of DMD exon skipping strategies. Among these are, CRISPR/Cas9 oligonucleotides, small molecules or other therapeutic modalities that promote exon skipping or micro dystrophin mini genes or cell based therapies. Methods for restoring the reading frame of exon 44 deletion via CRISPR-mediated exon skipping in the humanized mouse model, in patient-derived iPS cells and ultimately, in patients using various delivery systems are also contemplated. The impact of CRISPR technology on DMD is that gene editing can permanently correct mutations.
    Type: Application
    Filed: May 13, 2020
    Publication date: September 3, 2020
    Applicant: The Board of Regents of the University of Texas System
    Inventors: Yi-Li MIN, Rhonda BASSEL-DUBY, Eric OLSON
  • Publication number: 20200260698
    Abstract: CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene and absence or deficiency of dystrophin protein in striated muscle. Provided herein are compositions and methods for treating DMD caused by mutations in the dystrophin Actin Binding Domain 1 (ABD-1). The compositions and method described herein can be used to remove mutant sequences in dystrophin ABD-1 to generate a corrected DMD protein that, while lacking one or more exons (e.g., exons 3-9), retains important functional properties.
    Type: Application
    Filed: August 17, 2018
    Publication date: August 20, 2020
    Applicant: The Board of Regents of the University of Texas System
    Inventors: Viktoriia KYRYCHENKO, Eric N. OLSON, Rhonda BASSEL-DUBY
  • Patent number: 10687520
    Abstract: Duchenne muscular dystrophy (DMD), which affects 1 in 5,000 male births, is one of the most common genetic disorders of children. This disease is caused by an absence or deficiency of dystrophin protein in striated muscle. The major DMD deletion “hot spots” are found between exon 6 to 8, and exons 45 to 53. Here, a “humanized” mouse model is provided that can be used to test a variety of DMD exon skipping strategies. Among these are, CRISPR/Cas9 oligonucleotides, small molecules or other therapeutic modalities that promote exon skipping or micro dystrophin mini genes or cell based therapies. Methods for restoring the reading frame of exon 44 deletion via CRISPR-mediated exon skipping in the humanized mouse model, in patient-derived iPS cells and ultimately, in patients using various delivery systems are also contemplated. The impact of CRISPR technology on DMD is that gene editing can permanently correct mutations.
    Type: Grant
    Filed: March 7, 2018
    Date of Patent: June 23, 2020
    Assignee: The Board of Regents of the University of Texas System
    Inventors: Yi-Li Min, Rhonda Bassel-Duby, Eric Olson
  • Publication number: 20200046854
    Abstract: Duchenne muscular dystrophy (DMD) is an inherited X-linked disease caused by mutations in the gene encoding dystrophin, a protein required for muscle fiber integrity. The disclosure reports CRISPR/Cpf1-mediated gene editing (Myo-editing) is effective at correcting the dystrophin gene mutation in the mdx mice, a model for DMD. Further, the disclosure reports optimization of germline editing of mdx mice by engineering the permanent skipping of mutant exon and extending exon skipping to also correct the disease by post-natal delivery of adeno-associated virus (AAV). AAV-mediated Myo-editing can efficiently rescue the reading frame of dystrophin in mdx mice in vivo. The disclosure reports means of Myo-editing-mediated exon skipping has been successfully advanced from somatic tissues in mice to human DMD patients-derived iPSCs (induced pluripotent stem cells).
    Type: Application
    Filed: November 28, 2017
    Publication date: February 13, 2020
    Applicant: The Board of Regents of the University of Texas System
    Inventors: Yu ZHANG, Chengzu LONG, Rhonda BASSEL-DUBY, Eric OLSON
  • Publication number: 20190364862
    Abstract: CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene. In vivo AAV-mediated delivery of gene-editing components machinery has been shown to successfully remove mutant sequence to generate an exon skipping in the cardiac and skeletal muscle cells of postnatal mdx mice, a model of DMD. Using different modes of AAV9 delivery, the restoration of dystrophin protein expression in cardiac and skeletal muscle of mdx mice was achieved. Here, a humanized mouse model for DMD is created to help test the efficacy of genome editing to cure DMD. Additionally, to facilitate the analysis of exon skipping strategies in vivo in a non-invasive way, a reporter luciferase knock-in version of the mouse model was prepared. These humanized mouse models provide the ability to study correcting of mutations responsible for DMD in vivo.
    Type: Application
    Filed: December 8, 2017
    Publication date: December 5, 2019
    Applicant: The Board of Regents of the University of Texas System
    Inventors: Leonela AMOASII, Chengzu LONG, Rhonda BASSEL-DUBY, Eric OLSON
  • Publication number: 20190134021
    Abstract: The present disclosure relates to the identification of Nurr1 as a key regulator of metabolism, and the use of Nurr1 agonist to treat metabolic disorders such as diabetes, obesity, metabolic syndrome and hepatic steatosis.
    Type: Application
    Filed: April 26, 2017
    Publication date: May 9, 2019
    Applicant: The Board of Regents of the University of Texas System
    Inventors: Eric N. OLSON, Rhonda BASSEL-DUBY, Leonela AMOASII
  • Publication number: 20180271069
    Abstract: Duchenne muscular dystrophy (DMD), which affects 1 in 5,000 male births, is one of the most common genetic disorders of children. This disease is caused by an absence or deficiency of dystrophin protein in striated muscle. The major DMD deletion “hot spots” are found between exon 6 to 8, and exons 45 to 53. Here, a “humanized” mouse model is provided that can be used to test a variety of DMD exon skipping strategies. Among these are, CRISPR/Cas9 oligonucleotides, small molecules or other therapeutic modalities that promote exon skipping or micro dystrophin mini genes or cell based therapies. Methods for restoring the reading frame of exon 44 deletion via CRISPR-mediated exon skipping in the humanized mouse model, in patient-derived iPS cells and ultimately, in patients using various delivery systems are also contemplated. The impact of CRISPR technology on DMD is that gene editing can permanently correct mutations.
    Type: Application
    Filed: March 7, 2018
    Publication date: September 27, 2018
    Inventors: Yi-Li MIN, Rhonda BASSEL-DUBY, Eric OLSON
  • Publication number: 20160058889
    Abstract: Duchenne muscular dystrophy (DMD) is an inherited X-linked disease caused by mutations in the gene encoding dystrophin, a protein required for muscle fiber integrity. The disclosure reports CRISPR/Cas9-mediated gene editing (Myo-editing) is effective at correcting the dystrophin gene mutation in the mdx mice, a model for DMD. Further, the disclosure reports optimization of germline editing of mdx mice by engineering the permanent skipping of mutant exon (exon 23) and extending exon skipping to also correct the disease by post-natal delivery of adeno-associate virus (AAV). AAV-mediated Myo-editing can efficiently rescue the reading frame of dystrophin in mdx mice in vivo. The disclosure reports means of Myo-editing-mediated exon skipping has been successfully advanced from somatic tissues in mice to human DMD patients-derived iPSCs (induced pluripotent stem cells).
    Type: Application
    Filed: August 11, 2015
    Publication date: March 3, 2016
    Inventors: Eric N. OLSON, Chengzu LONG, John R. MCANALLY, John M. SHELTON, Rhonda BASSEL-DUBY
  • Publication number: 20040003424
    Abstract: The present invention provides methods for creating conditionally-immortal cell lines. These transgenic cell lines can be grown indefinitely in culture while maintaining a relatively undifferentiated stated. Upon appropriate switch signal, the cells cease replicating and differentiate much like adult cells. The switch is facilitated by the inactivation of a transforming gene, such as large T antigen. A convenient methodology for such inactivation is Cre-Lox mediated excision of the gene. Cardiac cells are provided as an example of useful a transgenic cell line.
    Type: Application
    Filed: March 4, 2003
    Publication date: January 1, 2004
    Applicant: Board of Regents, The University of Texas System
    Inventors: Eric Olson, Rhonda Bassel-Duby, David W. Markham, Igor I. Rybkin, R. Sanders Williams