Patents by Inventor Carrie Miceli

Carrie Miceli 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).

  • Identification of small molecules that facilitate therapeutic exon skipping
    Patent number: 10188633
    Abstract: This invention relates, e.g., to a method for enhancing exon skipping in a pre-mRNA of interest, comprising contacting the pre-mRNA with an effective amount of a small molecule selected from the compounds shown in Table 1, or a pharmaceutically acceptable salt, hydrate, solvate, or isomer thereof, and, optionally, with an antisense oligonucleotide that is specific for a splicing sequence in the pre-mRNA Methods for treating Duchenne muscular dystrophy (DMD) are disclosed.
    Type: Grant
    Filed: March 30, 2016
    Date of Patent: January 29, 2019
    Assignee: THE REGENTS OF THE UNIVERSITY OF CALIFORNIA
    Inventors: Stanley F. Nelson, M. Carrie Miceli, Miriana Moran
  • Identification of structurally similar small molecules that enhance therapeutic exon skipping
    Patent number: 9982260
    Abstract: This invention relates, e.g., to a method for enhancing exon skipping in a pre-mRNA of interest, comprising contacting the pre-mRNA with an effective amount of a compound such as, for example, Perphenazine, Flupentixol DiHCl, Zuclopenthixol or Corynanthine HCl, or a compound which shares a similar 2-D structure and activity level with one of these compounds, or a pharmaceutically acceptable salt, hydrate, solvate, or isomer of the compound, and, optionally, with an antisense oligonucleotide that is specific for a splicing sequence in the pre-mRNA Methods for treating Duchenne muscular dystrophy (DMD) are disclosed.
    Type: Grant
    Filed: September 30, 2014
    Date of Patent: May 29, 2018
    Assignee: The Regents of The University of California
    Inventors: Genevieve C. Kendall, Stanley F. Nelson, M. Carrie Miceli
  • IDENTIFICATION OF STRUCTURALLY SIMILAR SMALL MOLECULES THAT ENHANCE THERAPEUTIC EXON SKIPPING
    Publication number: 20160257952
    Abstract: This invention relates, e.g., to a method for enhancing exon skipping in a pre-mRNA of interest, comprising contacting the pre-mRNA with an effective amount of a compound such as, for example, Perphenazine, Flupentixol DiHCl, Zuclopenthixol or Corynanthine HCl, or a compound which shares a similar 2-D structure and activity level with one of these compounds, or a pharmaceutically acceptable salt, hydrate, solvate, or isomer of the compound, and, optionally, with an antisense oligonucleotide that is specific for a splicing sequence in the pre-mRNA Methods for treating Duchenne muscular dystrophy (DMD) are disclosed.
    Type: Application
    Filed: September 30, 2014
    Publication date: September 8, 2016
    Inventors: Genevieve C. KENDALL, Stanley F. NELSON, M. Carrie MICELI
  • IDENTIFICATION OF SMALL MOLECULES THAT FACILITATE THERAPEUTIC EXON SKIPPING
    Publication number: 20160220538
    Abstract: This invention relates, e.g., to a method for enhancing exon skipping in a pre-mRNA of interest, comprising contacting the pre-mRNA with an effective amount of a small molecule selected from the compounds shown in Table 1, or a pharmaceutically acceptable salt, hydrate, solvate, or isomer thereof, and, optionally, with an antisense oligonucleotide that is specific for a splicing sequence in the pre-mRNA Methods for treating Duchenne muscular dystrophy (DMD) are disclosed.
    Type: Application
    Filed: March 30, 2016
    Publication date: August 4, 2016
    Inventors: Stanley F. Nelson, M. Carrie Miceli, Miriana Moran
  • IDENTIFICATION OF SMALL MOLECULES THAT FACILITATE THERAPEUTIC EXON SKIPPING
    Publication number: 20140080896
    Abstract: This invention relates, e.g., to a method for enhancing exon skipping in a pre-mRNA of interest, comprising contacting the pre-mRNA with an effective amount of a small molecule selected from the compounds shown in Table 1, or a pharmaceutically acceptable salt, hydrate, solvate, or isomer thereof, and, optionally, with an antisense oligonucleotide that is specific for a splicing sequence in the pre-mRNA Methods for treating Duchenne muscular dystrophy (DMD) are disclosed.
    Type: Application
    Filed: September 13, 2013
    Publication date: March 20, 2014
    Applicant: The Regents of the University of california
    Inventors: Stanley F. Nelson, Carrie Miceli, Miriana Moran