Anne Vonada
Oregon Health & Science University, OR·Medicine & Clinical Research
Many inherited liver diseases trace to a single faulty gene that disrupts the body's chemistry, and severe cases are often treated by replacing the entire organ. A less drastic option is to transplant only healthy liver cells, called hepatocytes.
- Fields
Medicine & Clinical Research
Genetics
Microbiology & Immunology
- Topics
hepatocyte transplantation
cell therapy
liver disease
- Grants
- 1 known grant, through Apr 2030
What they work on
- Render donor hepatocytes resistant to acetaminophen by knocking out Cypor, the gene needed to convert the drug into a toxic form, and test drug-driven selection of donor cells in mouse models of alkaptonuria and gyrate atrophy.
- Generate immune-stealthy hepatocytes by knocking out class I and then class II HLA genes, the surface markers immune cells use to spot foreign tissue, and test them in allotransplantation models including under inflammatory conditions.
- Combine selective expansion and immune evasion in fully enhanced donor hepatocytes and test whether they correct disease without immune suppression in a phenylketonuria mouse model.
More about the research
Compared with liver-directed gene therapy, cell therapy offers durability, the ability to give repeat doses, and applicability to many disorders at once, in effect "one drug for multiple diseases." Two obstacles block wider use: too few donor cells take hold in the liver to reach the replacement level needed for clinical benefit, and because donor cells come from other people they are attacked by the recipient's immune system unless it is suppressed with drugs.
Why it matters
Patients with inherited metabolic liver diseases such as phenylketonuria, alkaptonuria and gyrate atrophy may face whole-organ transplantation or lifelong restriction. Donor liver cells engineered to expand in the recipient and avoid immune attack could make cell therapy practical for several such diseases at once, and could remove the need for long-term immune-suppressing drugs after transplant.
Known grants
From NIH and NSF research grants. Labs often have other funding we can't see.
Closest labs
Jayakar Nayak
Stanford University, CA·Medicine & Clinical Research
Identify which biomaterial platform, a scaffold that carries cells, best supports transplanting and engrafting human airway basal stem cells moved from culture into living airway tissue.
Bharat Bhushan
University of Pittsburgh, PA·Pharmacology
Determine the role of EGFR in liver injury and regeneration after acetaminophen overdose using mice lacking EGFR in hepatocytes and receptor-activation strategies, and identify how EGFR worsens injury.
Rita Perlingeiro
University of Minnesota, MN·Biomedical Engineering
Determine how the host muscle environment affects engraftment of PSC-derived myogenic progenitors in an immune-deficient FKRP mutant mouse model of dystroglycanopathy.
The school
Oregon Health & Science University: 24 labs on PI Pages, $545M a year on research, 54 research doctorates a year.
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