Patents by Inventor Karen Bulaklak

Karen Bulaklak 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).

  • DUAL AAV VECTOR-MEDIATED DELETION OF LARGE MUTATIONAL HOTSPOT FOR TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
    Publication number: 20230392132
    Abstract: Disclosed herein are therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use. The compositions and methods may include gRNAs targeting exons 44 and 55. The compositions and methods may also Include a mutant inverted terminal repeat (ITR) to generate a self-complementary vector genome.
    Type: Application
    Filed: October 21, 2021
    Publication date: December 7, 2023
    Inventors: Charles A. Gersbach, Karen Bulaklak
  • A HIGH-THROUGHPUT SCREENING METHOD TO DISCOVER OPTIMAL GRNA PAIRS FOR CRISPR-MEDIATED EXON DELETION
    Publication number: 20230349888
    Abstract: Disclosed herein are methods of using probes for high-throughput screening of guide RNA (gRNA) efficiency for Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRiSPR-associated (Cas)-based genome editing systems. Further disclosed herein is a humanized transgenic mouse model that recapitulates the severe DMD pathology of human patients. The mouse model may be used for determining the feasibility of CRISPR-based therapies for the correction of the human dystrophin gene by gene editing and methods of use.
    Type: Application
    Filed: April 27, 2021
    Publication date: November 2, 2023
    Inventors: Charles A. Gersbach, Veronica Gough, Karen Bulaklak
  • AAV VECTOR-MEDIATED DELETION OF LARGE MUTATIONAL HOTSPOT FOR TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
    Publication number: 20220184229
    Abstract: Disclosed herein are therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use.
    Type: Application
    Filed: April 14, 2020
    Publication date: June 16, 2022
    Inventors: Charles A. Gersbach, Karen Bulaklak, Jacqueline N. Robinson-Hamm