News

Transforming Treatment of Inherited Bone Marrow Failure in Fanconi Anemia by Precise In Vivo Genome Editing

Fanconi anemia (FA) is a rare inherited bone marrow failure syndrome characterized by chromosomal abnormalities and increased susceptibility to cancer. Allogeneic bone marrow transplantation is currently the only treatment for bone marrow failure, but its use is limited by the...

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Development of In Utero Therapies for Fanconi Anemia

Correcting FA mutations in all cells of the body may prevent issues such as bone marrow failure and cancer in people with FA. Since mutations in FA genes start during the gestational process, the ideal time to correct genes may...

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Building a Fanconi Anemia Data Commons

Studying a rare disease such as Fanconi anemia is challenging: data must be captured from as many treatment locations as possible. No single institution can have a breakthrough; it requires sharing as much data as possible from as many individuals...

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Building Collaborative Partnerships to Understand Fanconi Anemia Tumor Pathogenesis, Prevention, and Treatment

This team will facilitate collaborations between all current and future participants of the Fanconi Anemia Cancer Consortium (FACC). The goal is to gather information and samples from past, present, and future patients to fully understand how tumors develop in FA...

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Cytology-Based DNA Analysis to Investigate the Malignant Potential of Oral Lesions in Patients with Fanconi Anemia

Researchers will run samples collected by brushing lesions through an automated system to detect abnormal DNA content and malignant changes. By identifying high-risk lesions, closer follow-up and early intervention can be used to prevent malignancies. The goal is to establish...

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Development and Characterization of FA-HNSCC PDX Models

Laboratory models of FA head and neck cancer are lacking and represent a major barrier to identifying more effective treatments. We helped pioneer the creation of patient-derived xenografts (PDX) from head and neck cancers and have developed the first PDX...

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