Patents by Inventor Charles P. Emerson

Charles P. Emerson 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: 20240141359
    Abstract: The present invention is related to the field of genetic engineering. In particular, the repair, reversion and/or conversion of genetic mutations that are linked to a muscular dystrophy disease. Specifically contemplated are gene editor nuclease proteins or base editor proteins that are targeted to the muscular dystrophy genetic mutations or pathogenic variants. Such gene editor nuclease proteins include, but are not limited to Cas12a nuclease proteins and adenine base editor proteins. Repair, reversion and/or disruption of the genetic mutation or pathogenic variant reduces at least one symptom of a muscular dystrophy disease.
    Type: Application
    Filed: February 23, 2022
    Publication date: May 2, 2024
    Inventors: Charles P. Emerson, JR., Scot A. Wolfe
  • Publication number: 20240018479
    Abstract: Human skeletal muscle stem cells were generated from facioscapulohumeral muscular dystrophy (FSHD) and healthy control iPSC using a transgene-free skeletal muscle differentiation protocol and production of stable iMyoblasts. Analyses revealed that FSHD and healthy control iMyoblasts are embryonic-like myogenic cells that undergo myotube differentiation ex vivo by growth factor depletion and are efficiently transplantable into the tibialis anterior (TA) muscles of NSG mice, where human muscle under-goes embryonic-to-adult myosin isoform switching. The DUX4 FSHD disease gene maintains its hypomethylated disease state inFSHD iPSC and iMyoblast, and its expression is upregulated during myotube differentiation and in muscle xenografts. Consequently, these iMyoblasts accurately exhibit the molecular pathology of human muscular dystrophies and are useful for the development of drug, gene editing and stem cell therapeutics.
    Type: Application
    Filed: October 25, 2021
    Publication date: January 18, 2024
    Inventors: Charles P. Emerson, Dongsheng Guo, Katelyn M. Daman, Jing Yan, Jennifer Chee-Jen Chen, Meng-jiao Shi, Oliver D. King, Lawrence J. Hayward
  • Publication number: 20210230568
    Abstract: The present invention is directed to the filed of gene therapy. In particular, compositions and methods are disclosed that repair gene microduplication mutations by reversion to a wild type sequence. For example, the creation of a double stranded break by a programmable nuclease protein within a microduplication induces the microhomology mediated end joining DNA repair pathway that in the process of DNA repair removes the microduplication mutation and restores the wild type sequence.
    Type: Application
    Filed: May 3, 2019
    Publication date: July 29, 2021
    Inventors: Scot A. Wolfe, Charles P. Emerson, JR., Sukanya Iyer, Sneha Suresh, Christian Mueller, Jennifer Chen, Dongsheng Guo, Oliver King
  • Publication number: 20160160217
    Abstract: Compositions and methods for identifying new treatments for Facioscapulohumeral muscular dystrophy (FSHD), and uses thereof.
    Type: Application
    Filed: February 12, 2016
    Publication date: June 9, 2016
    Inventors: Charles P. Emerson, Jennifer Chen, Oliver D. King
  • Patent number: 9260755
    Abstract: Compositions and methods for identifying new treatments for Facioscapulohumeral muscular dystrophy (FSHD), and uses thereof.
    Type: Grant
    Filed: April 11, 2013
    Date of Patent: February 16, 2016
    Assignee: University of Massachusetts
    Inventors: Charles P. Emerson, Jr., Jennifer Chen, Oliver D. King
  • Publication number: 20130347136
    Abstract: Compositions and methods for identifying new treatments for Facioscapulohumeral muscular dystrophy (FSHD), and uses thereof.
    Type: Application
    Filed: April 11, 2013
    Publication date: December 26, 2013
    Inventors: Charles P. Emerson, JR., Jennifer Chen, Oliver D. King
  • Patent number: 7968527
    Abstract: Methods and compositions for inhibiting FGF signaling are described. Methods of the invention include contacting an FGF-responsive cell with exogenous heparan sulfate 6-O endosulfatase (Sulf1) in an amount effective to modify endogenous heparan sulfate, thereby inhibiting FGF signaling. Methods of the invention also include contacting an FGF-responsive cell with an exogenous Sulf1-modified compound, the exogenous Sulf1-modified compound being characterized by the ability to reduce binding of FGF2 or FGF4 to FGFR1. Compositions comprising exogenous Sulf1-modified compounds are also provided for use in conjunction with methods of the present invention.
    Type: Grant
    Filed: February 11, 2005
    Date of Patent: June 28, 2011
    Assignee: Boston Biomedical Research Institute
    Inventors: Charles P. Emerson, Jr., Xingbin Ai
  • Patent number: 6797816
    Abstract: Nucleic acid sequences encoding members of a new subfamily of sulfatases and polypeptides encoded thereby are provided. Compositions and methods of modulating sulfatases of this new subfamily to modify growth properties and differentiation of cells, as well as the ability of cells to prevent viral entry and to prevent recruitment of lymphocytes to a site of inflammation are also provided. The compositions and methods are useful in treating cancer and inhibiting metastases, promoting differentiation of stem cells into muscle, neural and renal cells and inhibiting viral infection and inflammation. In addition, functional embryonic techniques for identification and characterization of developmental regulatory genes such as these sulfatases are provided.
    Type: Grant
    Filed: March 13, 2003
    Date of Patent: September 28, 2004
    Assignee: Trustees of the University of Pennsylvania
    Inventors: Charles P. Emerson, Gurtej Kaur Dhoot
  • Publication number: 20030158084
    Abstract: Nucleic acid sequences encoding members of a new subfamily of sulfatases and polypeptides encoded thereby are provided. Compositions and methods of modulating sulfatases of this new subfamily to modify growth properties and differentiation of cells, as well as the ability of cells to prevent viral entry and to prevent recruitment of lymphocytes to a site of inflammation are also provided. The compositions and methods are useful in treating cancer and inhibiting metastases, promoting differentiation of stem cells into muscle, neural and renal cells and inhibiting viral infection and inflammation. In addition, functional embryonic techniques for identification and characterization of developmental regulatory genes such as these sulfatases are provided.
    Type: Application
    Filed: March 13, 2003
    Publication date: August 21, 2003
    Inventors: Charles P. Emerson, Gurtej Kaur Dhoot
  • Patent number: 6562956
    Abstract: Nucleic acid sequences encoding members of a new subfamily of sulfatases and polypeptides encoded thereby are provided. Compositions and methods of modulating sulfatases of this new subfamily to modify growth properties and differentiation of cells, as well as the ability of cells to prevent viral entry and to prevent recruitment of lymphocytes to a site of inflammation are also provided. The compositions and methods are useful in treating cancer and inhibiting metastases, promoting differentiation of stem cells into muscle, neural and renal cells and inhibiting viral infection and inflammation. In addition, functional embryonic techniques for identification and characterization of developmental regulatory genes such as these sulfatases are provided.
    Type: Grant
    Filed: September 22, 2000
    Date of Patent: May 13, 2003
    Assignees: Trustees of the University of Pennsylvania, The Royal Veterinary College
    Inventors: Charles P. Emerson, Gurtej Kaur Dhoot
  • Patent number: 5681735
    Abstract: A transcription control element is provided for controlling gene expression in myogenic cells. The transcription control element comprises an isolated DNA segment having an enhancer activity in cultured cells and in non-cultured myogenic cells. The transcription control element is isolated from upstream regions of genes encoding bHLH myogenic regulatory proteins. Specifically, an enhancer element from the upstream region of human myoD and an enhancer element from the upstream region of a quail qmf1 are provided. These myoblast-specific transcription control elements are capable of significantly increasing the levels of gene expression in myogenic cells and are intended to be applied in gene therapy, using myoblast transfer and microinjection techniques, wherein myoblast-specific gene expression is desired or required.
    Type: Grant
    Filed: October 7, 1994
    Date of Patent: October 28, 1997
    Assignee: Fox Chase Cancer Center
    Inventors: Charles P. Emerson, David J. Goldhamer