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Targeting LNK(SH2B3) to ameliorate hematopoietic stem/progenitor defects in Fanconi Anemia

2016 | Research Grant | Wei Tong

Dr. Tong recently discovered a novel gene called “LNK” that regulates bone marrow cell survival and growth. She found that loss of LNK in a mouse model of FA restores normal bone marrow functions and increases stem cell longevity. This new grant will allow Dr. Tong to explore the mechanisms underlyi…

The Role of Aldehydes in Fanconi Anemia Oral Squamous Cell Carcinoma

2016 | Research Grant | Ashley Kamimae-Lanning, KJ Patel

Fanconi anemia (FA) patients are at high risk of developing head and neck cancer and have limited options for treatment due to their sensitivity to chemotherapy and radiation. We have recently found that FA model mice with mutations in certain aldehyde detoxifying enzymes (ALDH2, ADH5) develop oral …

Towards improved clinical management of FA-related cancer via a novel functional genomics approach

2016 | Research Grant | Josephine Dorsman

This project seeks to identify cellular pathways that could be targeted to treat or prevent cancer in FA. The investigators take a novel approach using a high-resolution CRISPR knockout library to screen for fitness genes and FANCA-specific cancer vulnerabilities. They will take advantage of their o…

A porcine model of Fanconi anemia

2015 | Research Grant | William Fleming, Markus Grompe

A major limitation in FA research is the absence of an animal model that faithfully recapitulates the clinical features of this disease in humans. While mice have the characteristic DNA repair defects, they do not spontaneously develop the progressive anemia or acute leukemia seen in many patients. …

Bridge Funding for Metformin Studies

2015 | Research Grant | Markus Grompe

In the past funding period of the OHSU Fanconi Anemia Program Project we succeeded in discovering new drug targets and small molecules for FA therapy. All of these candidates have clinical potential and we are on the threshold of new interventions for this severe disease. The key goal of the project…

Deciphering the endocrine-specific role of FA proteines in pancreas islets

2015 | Research Grant | Nicholas Woods

Since many FA patients also suffer from diabetes and insulin resistance, the research proposed in this application is designed to determine the function of Fanconi anemia proteins in the endocrine pancreas. This study will analyze the dynamic features of FA protein expression and response to glucose…

Identifying the nature of the endogenous aldehydes-induced DNA damage that Fanconi anemia DNA repair pathways counteract

2015 | Research Grant | Rui Yu

All living cells form formaldehyde and acetaldehyde through normal metabolism (also from food and alcohol consumption). However, these aldehydes also induce severe DNA damage, which must be tolerated or repaired to prevent diseases. Currently, ~1 billion people in the world have difficulty in detoxi…

The effects of loss of Fanconi gene function on the behavior and therapeutic responses of head and neck cancers

2015 | Research Grant | Ian Mackenzie

In an attempt to explain the high frequency of head and neck cancers in FA patients we have examined differences in the behaviour and signaling pathways of oral epithelial cells (cells that line the oral cavity) lacking Fanconi gene function. Experimental reduction of expression of FA-__A and FA-__C…