University of Wisconsin–Madison

Precision Medicine Resource

CRISPR Cas9 embryonic gene editing in the Golos lab. (Image courtesy of the Golos lab)

With a major goal to establish next generation animal models and innovative tools for the assessment of novel precision stem cell therapies, the WNPRC’s Precision Medicine Core is developing unique resources for the WNPRC investigators and broad scientific community, including NHP models for genetic diseases such as Parkinson’s disease, and preclinical models for assessing novel stem cell therapies for bone marrow suppression, genetic blood diseases and AIDS. The Precision Medicine core is composed of two closely interconnected units: The Embryonic Genomic Editing Subunit headed by Dr. Golos is focused on the development of precision disease NHP models with embryonic genomic editing, while the Precision Therapies Subunit headed by Dr. Slukvin is focused on developing stem cell-based therapies utilizing precision disease models.

Contact Information

Developmental Services Supported

The goals of the Precision Medicine Core are to:

  • Provide pluripotent cell, gamete and embryo platforms for CRISPR/Cas9 genomic editing for Precision Medicine NHP model development.
  • Create precision MHC-defined NHP models for gene and stem cell-based therapies.
  • Integrate the NHP stem cell transplantation model into a functional pipeline for translational studies.

Shown are a helical tomotherapy machine and image

The Precision Therapy Subunit of Precision Medicine core supports the recently established NHP bone marrow transplantation program, a key component of the WNPRC Regenerative and Reproductive Medicine program. This program supports NHP studies to prevent solid organ transplant rejection through hematopoietic chimerism (Dixon Kaufman, UW-Madison and Samuel Strober, Stanford University), the development of novel approaches for highly efficient methods of genetic modification of HSCs (Torbett, the Scripps), preclinical evaluation of iPSC-derived blood products and vascular cells (Thomson, Slukvin, Morgridge Institute for Research, UW), and novel stem-cell based therapies for AIDS (Golos, Slukvin, Connor, UW).

An Embryo Genomic Editing Subunit within the Precision Medicine Core will also work with the Stem Cell Resources Unit to develop promising tools for translational research with regenerative medicine therapies.

Activities of the Embryo Genomic Editing Subunit, using the common marmoset model, will include:

  • Construction and in vitro optimization of vectors and other molecular reagents.
  • Production of oocytes and sperm for in vitro fertilization (IVF) or intracytoplasmic sperm injection (ICSI); technical ability to accomplish microinjection and culture of embryos.
  • Transfer of injected embryos to surrogate females and monitoring the progress of pregnancy. Sampling and testing offspring for genetic modification of choice.
  • Project-specific neonatal husbandry tailored to the predicted phenotype of the edited offspring.

The WNPRC Embryo Genomic Editing Subunit will focus on infrastructure and animal-intensive activities including:

  • Maintaining embryo micromanipulation workstations.
  • Maintaining supplies of reagents for IVF and embryo culture.
  • Providing fibroblasts, ESC and iPSC for testing CRISPR/Cas9 targeting vectors.
  • Conducting IVF, ICSI, and microinjection procedures to generate edited NHP embryos.
  • Coordinating collection of samples from pregnancies and from offspring for testing for correct targeting.
  • Developing sperm cryopreservation for experimental and germplasm banking purposes.
  • Leading discussion and analysis of ethical issues arising from genomic editing opportunities.

Key Personnel

Core Leaders:

  • Other Staff:
    • Jenna Kropp Ph.D., Research Associate
    • Saritha D’Souza, Research Associate
    • Matt Raymond, Research Specialist
    • Michael Meyer, M.S., Research Specialist