Patents by Inventor Yongping Yue

Yongping Yue 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: 20210163939
    Abstract: Disclosed herein are methods for systemically editing a gene in a subject and for systemically treating a genetic condition in a subject using a dual-vector CRISPR-Cas therapy. The methods comprise administering to the subject, via systemic administration, a gene editing AAV vector encoding a CRISPR effector protein (e.g., a Cas protein) and a targeting AAV vector providing one or more gRNAs targeted to the gene. In the methods, the ratio of the targeting AAV vector to the gene editing vector is greater than or equal to 2. Also provided are dual-vector systems for editing a gene or treating a genetic disease in a subject.
    Type: Application
    Filed: April 23, 2019
    Publication date: June 3, 2021
    Inventors: DONGSHENG DUAN, CHADY HAKIM, NALINDA B. WASALA, YONGPING YUE
  • Publication number: 20210139550
    Abstract: Disclosed are compositions and methods for treating dystrophinopathies. Compositions include modified dystrophin polynucleotides that encode modified dystrophin proteins having modified hinge 1 (H1) and/or hinge 4 (H4). Also disclosed are methods for treating dystrophinopathies by administering compositions encoding modified dystrophin proteins having modified hinge 1 (H1) and/or modified hinge 4 (H4).
    Type: Application
    Filed: April 3, 2019
    Publication date: May 13, 2021
    Inventors: Dongsheng DUAN, Lakmini WASALA, Yi LAI, Yongping YUE
  • Patent number: 10351611
    Abstract: According to the embodiments described herein, a series of biological materials for treatment/therapy of DMD and/or BMD through the recovery of sarcolemmal nNOS is provided. The biological material comprises the complete dystrophin repeats R16 and R17 or certain domains, sections, or fragments of the dystrophin repeats R16 and R17. In some aspects, such domains, sections, or fragments may be selected from sequence motifs including dystrophin R17 ?1 helix, ?2 and ?3 helices of both R16 and R17, or a combination thereof.
    Type: Grant
    Filed: April 14, 2017
    Date of Patent: July 16, 2019
    Assignee: THE CURATORS OF THE UNIVERSITY OF MISSOURI
    Inventors: Yi Lai, Junling Zhao, Yongping Yue, Dongsheng Duan
  • Publication number: 20190184033
    Abstract: Synthetic nucleic acids encoding mini and microdystrophin genes comprising the membrane binding motifs or domains of the R10-R11-R12 region are provided. Also provided are vectors, host cells, and related methods of using the same to treat a subject suffering from Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) or X-linked dilated cardiomyopathy (XLDC), or for ameliorating one or more adverse effects of DMD, BMD, or XLDC. Also provided are a fusion protein comprising a nNOS binding domain of dystrophin R16-R17 that is operably linked to a syntrophin PDZ domain and synthetic nucleic acids comprising the same that can be used to treat subjects with diseases characterized by loss of sarcolemmal neuronal nitric oxide synthase (nNOS) activity.
    Type: Application
    Filed: June 21, 2017
    Publication date: June 20, 2019
    Applicant: THE CURATORS OF THE UNIVERSITY OF MISSOURI
    Inventors: Dongsheng Duan, Yi Lai, Junling Zhao, Yongping Yue
  • Publication number: 20170349640
    Abstract: According to the embodiments described herein, a series of biological materials for treatment/therapy of DMD and/or BMD through the recovery of sarcolemmal nNOS is provided. The biological material comprises the complete dystrophin repeats R16 and R17 or certain domains, sections, or fragments of the dystrophin repeats R16 and R17. In some aspects, such domains, sections, or fragments may be selected from sequence motifs including dystrophin R17 ?1 helix, ?2 and ?3 helices of both R16 and R17, or a combination thereof.
    Type: Application
    Filed: April 14, 2017
    Publication date: December 7, 2017
    Inventors: Yi Lai, Junling Zhao, Yongping Yue, Dongsheng Duan
  • Patent number: 9624282
    Abstract: According to the embodiments described herein, a series of biological materials for treatment/therapy of DMD and/or BMD through the recovery of sarcolemmal nNOS is provided. The biological material comprises the complete dystrophin repeats R16 and R17 or certain domains, sections, or fragments of the dystrophin repeats R16 and R17. In some aspects, such domains, sections, or fragments may be selected from sequence motifs including dystrophin R17 ?1 helix, ?2 and ?3 helices of both R16 and R17, or a combination thereof.
    Type: Grant
    Filed: November 26, 2013
    Date of Patent: April 18, 2017
    Assignee: THE CURATORS OF THE UNIVERSITY OF MISSOURI
    Inventors: Yi Lai, Junling Zhao, Yongping Yue, Dongsheng Duan
  • Publication number: 20140234255
    Abstract: According to the embodiments described herein, a series of biological materials for treatment/therapy of DMD and/or BMD through the recovery of sarcolemmal nNOS is provided. The biological material comprises the complete dystrophin repeats R16 and R17 or certain domains, sections, or fragments of the dystrophin repeats R16 and R17. In some aspects, such domains, sections, or fragments may be selected from sequence motifs including dystrophin R17 ?1 helix, ?2 and ?3 helices of both R16 and R17, or a combination thereof.
    Type: Application
    Filed: November 26, 2013
    Publication date: August 21, 2014
    Applicant: THE CURATORS OF THE UNIVERSITY OF MISSOURI
    Inventors: Yi Lai, Junling Zhao, Yongping Yue, Dongsheng Duan
  • Patent number: 8236557
    Abstract: Hybrid adeno-associated virus (AAV) vector systems able to efficiently express therapeutic target genes larger than may be carried in a single AAV vector are provided, wherein a highly recombinogenic foreign DNA sequence is incorporated into two or more ITR-mediated AAV vectors. In one aspect of one embodiment, the novel hybrid AAV vector system is a hybrid dual AAV (hdAAV) vector system. In another aspect of one embodiment, the novel hybrid AAV vector system is a hybrid tri AAV (htAAV) vector system. A method of treating a clinical disease caused at least in part by a defective gene is provided, and comprises (1) providing a hybrid AAV vector system capable of expressing a therapeutic target gene, wherein the therapeutic target gene is capable of replacing, restoring or counteracting the effects of the defective gene; and (2) administering a therapeutic amount of said vector system to a subject wherein said therapeutic target gene is expressed at levels having a therapeutic effect.
    Type: Grant
    Filed: May 28, 2009
    Date of Patent: August 7, 2012
    Assignee: University of Missouri-Columbia
    Inventors: Duan Dongsheng, Arkasubhra Ghosh, Yongping Yue
  • Patent number: 7892824
    Abstract: The present invention provides novel dystrophin mini/micro-genes that retain the essential biological functions of a full-length dystrophin gene. More particularly, the present invention provides to a series of synthetic mini/micro-dystrophin genes capable of restoring neuronal nitric oxide synthase (nNOS) to the sarcolemma. A method as well as a pharmaceutical composition for treatment of Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD), and X-linked Dilated Cardiomyopathy (XLDC) are also provided.
    Type: Grant
    Filed: January 18, 2008
    Date of Patent: February 22, 2011
    Assignee: University of Missouri-Columbia
    Inventors: Dongsheng Duan, Yi Lai, Yongping Yue
  • Publication number: 20100003218
    Abstract: Hybrid adeno-associated virus (AAV) vector systems able to efficiently express therapeutic target genes larger than may be carried in a single AAV vector are provided, wherein a highly recombinogenic foreign DNA sequence is incorporated into two or more ITR-mediated AAV vectors. In one aspect of one embodiment, the novel hybrid AAV vector system is a hybrid dual AAV (hdAAV) vector system. In another aspect of one embodiment, the novel hybrid AAV vector system is a hybrid tri AAV (htAAV) vector system. A method of treating a clinical disease caused at least in part by a defective gene is provided, and comprises (1) providing a hybrid AAV vector system capable of expressing a therapeutic target gene, wherein the therapeutic target gene is capable of replacing, restoring or counteracting the effects of the defective gene; and (2) administering a therapeutic amount of said vector system to a subject wherein said therapeutic target gene is expressed at levels having a therapeutic effect.
    Type: Application
    Filed: May 28, 2009
    Publication date: January 7, 2010
    Inventors: Dongsheng Duan, Arkasubhra Ghosh, Yongping Yue
  • Publication number: 20080249052
    Abstract: The present invention provides novel dystrophin mini/micro-genes that retain the essential biological functions of a full-length dystrophin gene. More particularly, the present invention provides to a series of synthetic mini/micro-dystrophin genes capable of restoring neuronal nitric oxide synthase (nNOS) to the sarcolemma. A method as well as a pharmaceutical composition for treatment of Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD), and X-linked Dilated Cardiomyopathy (XLDC) are also provided.
    Type: Application
    Filed: January 18, 2008
    Publication date: October 9, 2008
    Inventors: Dongsheng Duan, Yi Lai, Yongping Yue