{
  "patent_number": "US 20140248702",
  "country": "US",
  "title": "How CRISPR Nickases Precisely Edit Genes in Human Cells",
  "original_title": "CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes",
  "summary": "This patent describes an engineered CRISPR-Cas system that uses a modified Cas9 protein, called a nickase, to cut only one strand of DNA in a eukaryotic cell, enabling more precise gene editing or altering gene expression.",
  "what_it_does": "This patent details a method for altering gene expression or editing the genome within a eukaryotic cell, like a human cell. It involves introducing an engineered CRISPR-Cas system that includes a guide RNA and a special Type-II Cas9 protein. Crucially, this Cas9 protein has specific mutations in its cutting part, called the catalytic domain, turning it into a 'nickase' that only cleaves one strand of the DNA molecule (Claim 1). The system also includes at least two nuclear localization signals (NLSs) to ensure it gets into the cell's nucleus where the DNA is located. This single-strand cut can then be repaired by the cell, often with the help of a provided homologous recombination (HR) template, allowing for precise changes to the DNA sequence (Claim 2, 15). For example, this could be used to correct a single faulty letter in a gene that causes a genetic disease.",
  "what_it_does_not_cover": [
    "Does not cover CRISPR-Cas systems that create double-strand breaks in DNA, as this system specifically uses a nickase to cleave only one strand.",
    "Does not cover gene editing or expression alteration in prokaryotic cells, as the claims specify a 'eukaryotic cell'.",
    "Does not cover naturally occurring CRISPR-Cas systems, as the claims require an 'engineered, non-naturally occurring' system.",
    "Does not cover Cas9 proteins that lack specific mutations in their catalytic domain, which are necessary to make it a nickase.",
    "Does not cover CRISPR-Cas systems that do not include at least two nuclear localization signals (NLSs)."
  ],
  "filed": "2014-04-22",
  "granted": null,
  "expires": "2034-04-22",
  "status": "active",
  "holder": "Massachusetts Institute of Technology",
  "holder_url": "https://patentbrief.org/company/massachusetts-institute-of-technology",
  "inventors": [
    {
      "name": "Le Cong",
      "url": "https://patentbrief.org/inventor/le-cong"
    },
    {
      "name": "Feng Zhang",
      "url": "https://patentbrief.org/inventor/feng-zhang"
    }
  ],
  "times_cited": 268,
  "tags": [
    "biotech",
    "pharmaceutical",
    "gene_editing",
    "software"
  ],
  "abstract": "The invention provides for systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR/Cas system.",
  "url": "https://patentbrief.org/patent/us/20140248702/crispr-cas-nickase-systems-methods-and-compositions-for-sequence-manipulation-in",
  "markdown_url": "https://patentbrief.org/patent/us/20140248702/crispr-cas-nickase-systems-methods-and-compositions-for-sequence-manipulation-in/md",
  "google_patents_url": "https://patents.google.com/patent/US20140248702",
  "relatedPatents": []
}