Pluripotent cell lines and methods of use thereof
AssigneesParkinson's Institute, The Board of Trustees of the Leland Stanford Junior University
InventorsRenee Ann Reijo Pera, J. William Langston, Birgitt Schüle, Theodore D. Palmer, Blake Byers, Ha Nam Nguyen, James Anthony Byrne, Brandon John Cord
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Abstract
Methods of generating cell lines with a sequence variation or copy number variation of a gene of interest, methods of use thereof, and cell lines with a sequence variation or copy number variation of a gene of interest are provided.
Claims65
A method of producing dopaminergic cells from a patient having a diagnosis of Parkinson's disease or Parkinson's-like disease, the method comprising:
- a. obtaining fibroblasts from the patient;
- b. dedifferentiating the fibroblasts;
- and
- c. differentiating the stem cells towards a dopaminergic cell fate.
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A method of determining whether an agent is useful in the treatment of Parkinson's disease or Parkinson's-related disease, the method comprising:
- a. contacting dopaminergic cells produced from induced pluripotent stem cells derived from a patient with Parkinson's disease or Parkinson's-like disease;
- b. detecting a response in the cells;
- and
- c. determining whether the agent is useful in the treatment based on the detected response.
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An induced pluripotent cell line from a patient with Parkinson's disease or Parkinson's-like disease, produced by:
- a. obtaining fibroblasts from a patient with Parkinson's disease or Parkinson's-like disease carrying a genetic mutation which causes the disease;
- b. dedifferentiating the fibroblasts, whereby producing induced pluripotent stem cells;
- and
- c. differentiating the stem cells towards a dopaminergic cell fate, whereby producing an induced pluripotent cell line from a patient with Parkinson's disease or Parkinson's-like disease.
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The cell line of 47 wherein the patient carries a homozygous mutation of LRRK2.
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A human induced pluripotent cell line from a patient with Parkinson's disease wherein the cell line comprises a genetic variation related to Parkinson's disease.
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A human induced pluripotent cell line wherein the cell line comprises a genetic variation in the SNCA gene or the LRRK2 gene.
Dependencies are inferred from the claim text.