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Drug screening and repurposing in Fanconi anemia therapeutics (acronym: REPAIR-FANC)

2016 | Research Grant | Jordi Surralles

In the field of rare diseases it is extremely difficult to perform clinical trials with new chemical entities without prior information on bioavailability, dose and safety in humans. Drug repurposing is the application of known drugs and compounds to new indications. Thus, the aim of this project is…

How environment shapes the consequences of mutation within the Fanconi anemia pathway

2016 | Research Grant | Georgios Karras, Harvey Lodish

It has long been recognized that gene-environment interactions influence the clinical course of individuals with FA, yet the underlying mechanisms remain elusive. Drawing on this labs deep expertise in protein folding diseases this project proposes a pragmatic approach to address this issue which ca…

Investigating the impact of lentiviral transduction on Fanconi anemia hematopoietic stem cells for improved gene therapy

2016 | Research Grant | Anna Kajaste-Rudnitski

Many have proposed that lentiviral vector (LV)-mediated hematopoietic stem cell (HSC) gene therapy may constitute a new safe and efficient approach for the treatment/prevention of the bone marrow failure (BMF) characteristic of FA patients. To further improve the possibilities of developing a safe a…

Reducing the burden of squamous cell carcinoma in Fanconi anemia - 2016

2016 | Service Grant | Ralf Dietrich, Eunike Velleuer

Eighty percent of FA patients survive beyond age 20, carry a high risk for squamous cell carcinoma (SCC) of the oropharyngeal and anogenital regions connected with high morbidity and mortality. In addition to intrinsic genetic instability, HSCT and a number of other factors contribute to the pathoge…

Targeting lipid metabolism in FA for the prevention and treatment of squamous cell carcinoma

2016 | Research Grant | Susanne Wells

Dry and moist skin (e.g., in the mouth) plays a critical role in maintaining a barrier against environmental insults and cancer. Based on new data, this team believes that individuals with FA may have an impaired barrier and this may be why they are at risk for SCC. Specifically, data shows that FA …

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…