News

Modeling hematopoietic clonal evolution in Fanconi anemia

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...

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Squamous Cell Carcinoma Chemoprevention Phase 1

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...

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Treating Fanconi Anemia Cancer with Proton Precision Therapy

Grant description: 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...

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

Grant description 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...

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

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...

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Identification of Novel Therapeutic Targets against Fanconi Anemia-associated Squamous Cell Carcinoma

Fanconi anemia patients are dramatically predisposed to early-onset and aggressive head, neck, and anogenital squamous cell carcinoma (SCC). Treating Fanconi SCCs is challenging due to high-frequency recurrence and patient chemosensitivity, yielding an average survival rate of <2 years post-diagnosis. New...

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