{
  "patent_number": "US 12252707",
  "country": "US",
  "title": "How to Make Animals That Can Turn On CRISPR Gene Editing",
  "original_title": "Delivery, Engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications",
  "summary": "This patent describes creating genetically modified non-human animals, like mice, where a key CRISPR gene-editing protein (Cas9) can be turned on only in specific cells or at specific times using a special genetic switch.",
  "what_it_does": "The patent details a \"non-human transgenic animal\" (Claim 1), meaning an animal whose genes have been changed. This animal has a special genetic package, called an \"exogenous conditional expression cassette,\" inserted into its own DNA, specifically \"knocked into an endogenous genomic locus\" (Claim 1), like the \"Rosa26 locus\" (Claim 3) in mice. This cassette contains the instructions for making a \"Cas9 protein,\" which is a molecular scissor used in CRISPR gene editing. The clever part is that the Cas9 protein is only made when another enzyme, \"Cre recombinase,\" is present (Claim 1). This allows scientists to control exactly when and where the Cas9 protein is active. The Cas9 protein itself is designed with two \"nuclear localization signals\" (NLS), one at each end (N-terminus and C-terminus), to help it get into the cell's nucleus where the DNA is (Claim 1). For example, a researcher could use this to study how turning on Cas9 in only certain brain cells affects a neurological disease.",
  "what_it_does_not_cover": [
    "Does not cover genetically modifying human beings.",
    "Does not cover Cas9 protein expression that is always on or not dependent on Cre recombinase.",
    "Does not cover Cas9 proteins that lack two nuclear localization signals (NLS), one at the N-terminus and one at the C-terminus.",
    "Does not cover gene editing systems that use different proteins instead of Cas9 (e.g., other CRISPR enzymes or TALENs).",
    "Does not cover inserting the Cas9 cassette into just any random spot in the genome; it must be \"knocked into an endogenous genomic locus.\""
  ],
  "filed": "2021-04-30",
  "granted": "2025-03-18",
  "expires": "2041-04-30",
  "status": "active",
  "holder": "Massachusetts Institute of Technology",
  "holder_url": "https://patentbrief.org/company/massachusetts-institute-of-technology",
  "inventors": [
    {
      "name": "Yang Zhou",
      "url": "https://patentbrief.org/inventor/yang-zhou"
    },
    {
      "name": "Randall Jeffrey Platt",
      "url": "https://patentbrief.org/inventor/randall-jeffrey-platt"
    },
    {
      "name": "Guoping Feng",
      "url": "https://patentbrief.org/inventor/guoping-feng"
    },
    {
      "name": "Feng Zhang",
      "url": "https://patentbrief.org/inventor/feng-zhang"
    }
  ],
  "times_cited": 0,
  "tags": [
    "biotech",
    "gene_editing",
    "pharmaceutical",
    "research_tools"
  ],
  "abstract": "The invention provides for delivery, engineering and optimization of systems, methods and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also 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 to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.",
  "url": "https://patentbrief.org/patent/us/12252707/delivery-engineering-and-optimization-of-systems-methods-and-compositions-for-se",
  "markdown_url": "https://patentbrief.org/patent/us/12252707/delivery-engineering-and-optimization-of-systems-methods-and-compositions-for-se/md",
  "google_patents_url": "https://patents.google.com/patent/US12252707",
  "relatedPatents": []
}