Patents by Inventor Melissa J. Spencer

Melissa J. Spencer 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: 20240299586
    Abstract: In various embodiments, AAV serotypes capable of transducing skeletal muscle fibers and skeletal muscle stem cells (MuSC) with reduced off target tissue tropism are provided.
    Type: Application
    Filed: May 5, 2022
    Publication date: September 12, 2024
    Applicant: The Regents of the University of California
    Inventors: Melissa J. Spencer, Michael Reza Emami, April D. Pyle, Courtney S. Young, Donald B. Kohn
  • Publication number: 20240156989
    Abstract: The present disclosure provides a method of modifying a mutant dystrophin gene in the genome of a cell. The present disclosure further provides compositions and kits for modifying a mutant dystrophin gene in the genome of a cell.
    Type: Application
    Filed: April 24, 2023
    Publication date: May 16, 2024
    Inventors: April D. Pyle, Courtney S. Young, Melissa J. Spencer
  • Patent number: 11779653
    Abstract: In various embodiments a polyrotaxane carrier for in vivo delivery of a nucleic acid is provided. In certain embodiments the carrier comprises: a multi-arm polyethylene glycol (PEG) backbone comprising at least three arms; at least one cyclic compound having a cavity, where an arm of said multi-arm PEG backbone is threaded into the cavity of said cyclic compound forming an inclusion complex; a bulky moiety capping the terminal of the arm(s) threaded into said cyclic compound where said moiety inhibits dethreading of the cyclodextrin from the arm(s) of said backbone; and where at least one arm of said PEG backbone is free of cyclic compounds; and where said carrier has a net positive charge.
    Type: Grant
    Filed: September 27, 2018
    Date of Patent: October 10, 2023
    Assignee: The Regents of the University of California
    Inventors: Huan Meng, Melissa J. Spencer, April D. Pyle, Courtney S. Young, Xiangsheng Liu, Ying Ji, Michael Reza Emami
  • Patent number: 11666666
    Abstract: The present disclosure provides a method of modifying a mutant dystrophin gene in the genome of a cell. The present disclosure further provides compositions and kits for modifying a mutant dystrophin gene in the genome of a cell.
    Type: Grant
    Filed: February 9, 2017
    Date of Patent: June 6, 2023
    Assignee: The Regents of the University of California
    Inventors: April D. Pyle, Courtney S. Young, Melissa J. Spencer
  • Publication number: 20220177876
    Abstract: Disclosed herein are guide sequences for modifying a dystrophin gene using CRISPR technology. Specifically, the disclosure provides a method of modifying a dystrophin gene in a cell or a subject, which comprises introducing into the cell or subject (a) a Cas protein or a nucleotide sequence encoding the Cas protein; and a single guide RNA (gRNA), or a first gRNA and a second gRNA, wherein the Cas protein is a type II CRISPR/Cas endonuclease. Further disclosed are gRNA nucleic acid sequences.
    Type: Application
    Filed: April 7, 2020
    Publication date: June 9, 2022
    Inventors: Melissa J. Spencer, Courtney S. Young, April D. Pyle, Michael Reza Emami
  • Publication number: 20200376139
    Abstract: In various embodiments a polyrotaxane carrier for in vivo delivery of a nucleic acid is provided. In certain embodiments the carrier comprises: a multi-arm polyethylene glycol (PEG) backbone comprising at least three arms; at least one cyclic compound having a cavity, where an arm of said multi-arm PEG backbone is threaded into the cavity of said cyclic compound forming an inclusion complex; a bulky moiety capping the terminal of the arm(s) threaded into said cyclic compound where said moiety inhibits dethreading of the cyclodextrin from the arm(s) of said backbone; and where at least one arm of said PEG backbone is free of cyclic compounds; and where said carrier has a net positive charge.
    Type: Application
    Filed: September 27, 2018
    Publication date: December 3, 2020
    Inventors: Huan Meng, Melissa J. Spencer, April D. Pyle, Courtney S. Young, Xiangsheng Liu, Ying Ji, Michael Reza Emami
  • Publication number: 20190038776
    Abstract: The present disclosure provides a method of modifying a mutant dystrophin gene in the genome of a cell. The present disclosure further provides compositions and kits for modifying a mutant dystrophin gene in the genome of a cell.
    Type: Application
    Filed: February 9, 2017
    Publication date: February 7, 2019
    Inventors: April D. Pyle, Courtney S. Young, Melissa J. Spencer