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A Porcine Model of Fanconi Anemia (2019)

2019 | Research Grant | William Fleming, Markus Grompe

A limitation in FA research is the absence of a live model that displays key features of the disease. Researchers at OHSU will create the first large animal model of FA to test potential therapies for people with FA.

Complete DNA sequencing of the Fanconi anemia patient tumors

2019 | Research Grant | Agata Smogorzewska

Fanconi anemia patients have a very high predisposition to head, neck, and anogenital squamous cell carcinoma (SCC). To understand how these cancers develop, Dr. Smogorzewska and team are cataloging genetic changes that are present in the cancers. They are using the latest DNA sequencing technologie…

CRISPR transcriptional screens for re-activation of HDR in FA patient-derived cells

2019 | Research Grant | Jacob Corn

There is a great deal of excitement about the potential of CRISPR genome editing to treat disorders such as Fanconi anemia. But the mutations that cause FA also reduce the effectiveness of genome editing. Dr. Corn and colleagues will search for potential ways to re-activate genome editing in FA pati…

Gefitinib/Afatinib therapy for Head and Neck Squamous Cell Carcinomas in Fanconi anemia

2019 | Research Grant | Jordi Minguillón, Jordi Surralles

Beyond surgical resection, there is no effective therapy nowadays to treat FA patients that develop solid tumors, so a drug able to kill tumor cells but nontoxic for FA patients is of critical relevance. Gefitinib and afatinib candidates are approved drugs and are already being used in cancer therap…

High-priority agents for Fanconi anemia-associated oral cancer treatment and prevention

2019 | Research Grant | Frank G. Ondrey

Oral cancer in Fanconi patients is a severe disease requiring innovative treatment and prevention measures. The potential use of FDA-approved diabetes drugs is a practical strategy for Fanconi patients for oral cancer prevention or treatment studies, once these preliminary studies are conducted. The…

Metabolic alterations in glucose utilization and carnitine biosynthesis impact nutritional status in individuals with FA

2019 | Research Grant | Adam Nelson, Lindsey Romick-Rosendale

Persons with FA, as well as their family members, voice concern about being underweight and unable to build muscle mass. These concerns have an obvious physical impact, not only on the person's appearance to others and themselves and the negative impact on immune function and host defense but also h…

Modeling hematopoietic clonal evolution in Fanconi anemia

2019 | Research Grant | George Q. Daley, Grant Rowe

FA confers a high risk of developing leukemia at a young age, and patients with FA who develop leukemia are difficult to treat with have poor long-term outcomes. Leukemia develops as a consequence of the acquisition of genetic mutations within blood stem cells in the bone marrow that cooperate to dr…

Squamous Cell Carcinoma Chemoprevention Phase 1

2019 | Research Grant | Markus Grompe

Head and neck cancer is a major issue for the FA population. There is no suitable preclinical model to study potential strategies for preventing or delaying squamous cell carcinoma in FA. Using a cancer-prone mouse model developed by a lab in the United Kingdom, researchers will test small molecules…

Synthetic Lethal Approaches to Treatment of FA Gene Mutant Head and Neck Cancer

2019 | | Barbara Burtness, Gary Kupfer

Typical treatments for cancer involve conditioning that damages DNA and can therefore be harmful to people with FA. Researchers are testing drugs using patient-derived xenografts (tumors from patients that are grown in mice) to find a way to kill cancer cells without harming normal cells.

Treating Fanconi Anemia Cancer with Proton Precision Therapy

2019 | Research Grant | Mathieu Sertorio, Yi Zheng

Treatment of blood complications in FA kids using bone marrow transplantation has seen great improvement over the past decade. However, after a successful bone marrow transplantation, those kids affected with FA must now face nonblood malignancies in subsequence adolescent life which defy convention…

Development of a safe, completely non-genotoxic anti-Kit antibody-based conditioning regimen for hematopoietic stem cell transplantation in Fanconi Anemia

2018 | Research Grant | Agnieszka Czechowicz

Bone marrow transplantation has been used for 60+ years to treat >1,000,000 patients suffering from many types of blood or immune diseases, and this therapy is the best current treatment for FA patients experiencing blood problems. This procedure relies on replacing sick blood-forming stem cells in …

Direct in vivo gene correction of hematopoietic stem cell populations in Fanconi anemia

2018 | Research Grant | Kevin Haworth, Hans-Peter Kiem

Fanconi Anemia (FA) is characterized by fragile bone marrow and the inability to repair DNA damage which accumulates in repopulating stem cells, leading to marrow failure. Current treatments often involve bone marrow transplantation however the majority of patients will not have an appropriately mat…