News

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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The 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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Use of triplex-forming PNAs as a strategy for correction of the FA phenotype (continued)

As a monogenic blood disorder with potential survival disadvantage, Fanconi anemia has long been considered an attractive target for conventional gene therapy but success has been elusive. Consequently, there has been increasing interest in developing techniques to catalyze correction of...

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Defining tractable approaches for gene editing of Fanconi Anemia hematopoietic stem cells

My lab has developed a rapid and efficacious Cas9-based approach to introduce programmed sequence changes to human cells with ease. This work takes advantage of our discovery that Cas9 is extremely long-lived on its target DNA, yet releases a flap...

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Engineering Fanconi Anemia Hematopoietic Stem Cells from Human iPS Cells

Current therapy for FA is limited to allogeneic stem cell transplantation (SCT), a process associated with significant morbidity and mortality, particularly when an ideally matched donor is not readily available. Therefore, novel therapies that improve or replace SCT are needed....

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Field-Coverage Oral Cancer Chemoprevention via Janus Nanoparticles

By virtue of their inability to repair a specific form of DNA damage, persons with Fanconi Anemia (FA) are appreciably more susceptible to development of certain cancers. Oral squamous cell carcinoma (OSCC), which arises from the lining cells of the...

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