{
  "patent_number": "US 12735723",
  "country": "US",
  "title": "Treating Muscular Dystrophy with a Self-Complementary AAV Gene Therapy",
  "original_title": "Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy",
  "summary": "This patent describes a method to treat muscular dystrophy by delivering a specific gene using a specially designed, self-complementary adeno-associated virus (AAV) vector.",
  "what_it_does": "This patent describes methods for treating muscular dystrophy by introducing a specific gene into a patient's cells. It uses a self-complementary recombinant adeno-associated virus (rAAV) vector, specifically named scAAVrh74.tMCK.hSGCA, as a delivery vehicle. This vector is designed to express the alpha-sarcoglycan gene (hSGCA) in the patient's body, which is crucial for muscle function. The patent also covers ways to create this rAAV and pharmaceutical mixtures containing it. For example, a patient with a type of muscular dystrophy caused by a faulty alpha-sarcoglycan gene could receive this vector to help their muscles produce the correct protein.",
  "what_it_does_not_cover": [
    "Does not cover gene therapies for muscular dystrophy that use different AAV serotypes (like AAV9 or AAV8) instead of the specified scAAVrh74 vector.",
    "Does not cover gene therapies for muscular dystrophy that deliver genes other than alpha-sarcoglycan (hSGCA), such as the dystrophin gene for Duchenne muscular dystrophy.",
    "Does not cover non-viral methods of gene delivery, such as lipid nanoparticles or electroporation, for treating muscular dystrophy.",
    "Does not cover treatments for muscular dystrophy that do not involve gene therapy, such as physical therapy or steroid medications.",
    "Does not cover the use of this specific scAAVrh74.tMCK.hSGCA vector for treating diseases other than muscular dystrophy."
  ],
  "filed": "2022-11-30",
  "granted": "2026-09-15",
  "expires": null,
  "status": "active",
  "holder": null,
  "holder_url": null,
  "inventors": [],
  "times_cited": 0,
  "tags": [
    "biotech",
    "pharmaceutical",
    "gene_editing"
  ],
  "abstract": "Described herein are methods of treating muscular dystrophy comprising administering a self complementary recombinant AAV (rAAV) scAAVrh74.tMCK.hSGCA vector, methods of expressing alpha-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV, and methods of generating the rAAV.",
  "url": "https://patentbrief.org/patent/us/12735723/self-complementary-adeno-associated-virus-vector-and-its-use-in-treatment-of",
  "markdown_url": "https://patentbrief.org/patent/us/12735723/self-complementary-adeno-associated-virus-vector-and-its-use-in-treatment-of/md",
  "google_patents_url": "https://patents.google.com/patent/US12735723",
  "relatedPatents": []
}