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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

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…

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

2018 | Research Grant | Agata Smogorzewska

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

Phase 1: Pilot Study of Metformin for Patients with Fanconi Anemia

2018 | Clinical Trial | Akiko Shimamura

Low blood counts due to bone marrow failure are a common complication of Fanconi anemia (FA) which affect health and quality of life. Metformin improves blood counts in mice with FA and may protect against DNA damage. Laboratory studies suggest that Metformin may be protective against aldehydes, whi…

Quercetin chemoprevention for Squamous cell carcinoma in patients with Fanconi anemia

2018 | Clinical Trial | Parinda Mehta

Excessive toxicity from chemotherapy and radiation makes treatment for SCC in FA quite challenging and leads to dismal outcomes, in fact fatal outcomes in most patients. Thus, there is clearly a need for a new approach both for prevention and/or treatment that has fewer and less severe side effects.…

The Fanconi Anemia Cancer Translational Resource

2018 | Research Grant | Raymond Monnat, Jr.

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 patients. As part of this Resource we will develop and c…

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

2017 | Research Grant | Jacob Corn

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 of single stranded DNA after cleavage. By using Cas…

Engineering Fanconi Anemia Hematopoietic Stem Cells from Human iPS Cells

2017 | Research Grant | George Q. Daley, Grant Rowe

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. Progress in FA thera…

Field-Coverage Oral Cancer Chemoprevention via Janus Nanoparticles

2017 | Research Grant | Joerg Lahann, Susan Mallery

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 mouth, occurs at >500 fold higher incidence in ind…

Pilot Study of Metformin for Patients with Fanconi Anemia

2017 | Clinical Trial | Akiko Shimamura

The objective of this proposal is to conduct a pilot study of metformin to treat FA. This pilot study will explore whether Metformin is safe and efficacious in improving hematopoiesis in patients with Fanconi Anemia. We will focus on pediatric and young adult patients with FA to determine safety, to…

TGF-β pathway inhibitors for the treatment of bone marrow failure in Fanconi anemia

2017 | Research Grant | Alan D’Andrea

Fanconi anemia (FA) patients suffer from progressive bone marrow failure due to the defective hematopoietic stem cells (HSCs) in their bone marrow. The mechanisms of why the HSCs in FA patients are defective remain elusive. Recent studies suggest that DNA damage induced by physiological stress or al…

The prevention of DNA damage in Fanconi anemia HSC by ALDH activators

2017 | Research Grant | Daria Mochly-Rosen, Kenneth Weinberg

The cause of injury and loss of blood-forming stem cells (Hematopoietic Stem Cells, HSC) in Fanconi anemia is due to abnormal repair of a specific type of DNA damage in which two DNA strands get permanently knotted to each other. This type of injury probably occurs frequently but is normally repaire…