News

Correction of Fanconi Anemia Mutations using Digital Genome Engineering

While the transplantation of healthy blood-forming stem cells from a healthy related or unrelated donor is the current standard of care for FA patients, this treatment carries a significant risk of morbidity and mortality caused by the toxic preconditioning regimen...

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

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...

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Extended Funding: Fanconi Anemia Cancer Translational Resource

Fanconi anemia (FA) patients are at exceptionally high risk of developing epithelial cancers. We aim to identify features of these cancers that provide new insight into their origins, and better ways to treat these cancers in the context of FA...

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Pathogenesis, clinical and treatment outcomes, and molecular characteristics of anogenital squamous cell carcinomas in individuals with Fanconi Anemia

Individuals with FA have an increased risk of developing anogenital squamous cell carcinoma (cancer of the vulva, cervix, vagina, or anus), but the best and safest way to treat these cancers in individuals with FA is unknown because only a...

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Severe spermatogenic failure as a sentinel for early diagnosis of late-onset Fanconi Anemia

Through the first systematic evaluation of all known FA genes in a selected group of infertile patients showing mild hematological alterations we expect to diagnose adult-onset FA. Our objective is to diagnose the disease before the appearance of severe complications...

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

Head and neck cancer in adult Fanconi anemia patients is often poorly managed with standard treatments. New drugs that work on controlling the cell cycle and cell division, rather than by damaging the cell’s DNA, may be safer in patients...

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