Fanconi Anemia News and Research

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Fanconi anemia or FA, is a rare, inherited blood disorder that leads to bone marrow failure. FA causes your bone marrow to stop making enough new blood cells for your body to work normally. FA also can cause your bone marrow to make many abnormal blood cells. This can lead to serious health problems such as cancer.

High-dose folic acid appears safe from formaldehyde toxicity

Folic acid – an essential vitamin added to flour and cereals and prescribed at high doses for cancer patients and people with blood disorders – does not elevate toxic formaldehyde in the body, according to a new Cornell University study on mice and human cancer patients who consumed high doses.

2 Sep 2026

New insights into alcohol-related DNA damage and cancer risk

Alcohol consumption leads to the formation of a toxic compound called acetaldehyde, which damages DNA.

15 Dec 2025

Fanconi anemia patients show striking metabolic differences

Experts at Cincinnati Children's have uncovered striking metabolic differences in people with Fanconi anemia (FA), a rare genetic disorder that causes bone marrow failure and dramatically increases cancer risk.

29 Nov 2025

New antibody therapy offers hope for fragile patients with Fanconi anemia

An antibody treatment developed at Stanford Medicine successfully prepared patients for stem cell transplants without toxic busulfan chemotherapy or radiation, a phase 1 clinical trial has shown.

22 Jul 2025

Maternal iron deficiency disrupts embryonic sex determination in mice

Developmentally expressed genes play a fundamental role in the formation of a wide range of organs. The Y chromosome-located mammalian sex-determining gene Sry, required for the formation of testis, is activated only within a narrow time window during embryogenesis.

11 Jun 2025

Scientists identify early life opportunity for in vivo stem cell gene therapy

A team of scientists from the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, has identified a unique window shortly after birth in which circulating blood stem cells can be effectively targeted with gene therapy directly in the body.

28 May 2025

Newly identified gene mutation causes severe form of Fanconi anemia

Fanconi anemia is an aggressive, life-threatening disorder. Most individuals living with this rare genetic condition, characterized by bone marrow failure and cancer predisposition, survive into adulthood only with bone marrow transplantation and regular cancer screening.

11 May 2025

DDX41: A unique player in myeloid leukemogenesis

A new editorial paper was published in Oncotarget's Volume 15 on July 2, 2024, entitled, "DDX41 and its unique contribution to myeloid leukemogenesis."

19 Jul 2024

Stand Up To Cancer unveils new gastroesophageal cancer research teams and awards

Stand Up To Cancer (SU2C) today announced three Research Teams focused on bringing new therapies to clinical trials for the treatment of gastroesophageal cancer (GEC).

15 Nov 2023

Detection and targeting of splicing deregulation reveal a potential strategy for pAML therapy

Pediatric acute myeloid leukemia or pAML is a childhood blood cancer, one that has proved confounding to clinicians and researchers, with a high relapse rate and relatively few identified genetic mutations (compared to the adult version) that might explain its cause.

8 Mar 2023

Repressing a protein that facilitates DNA repair to potentiate chemotherapy

Chemotherapy kills tumor cells by causing damage to them. One of the most effective ways of causing damage is to prevent the two DNA strands from separating so that the cellular machinery cannot read the instructions written in the genes.

15 Jun 2021

Study may lead to new treatments that eliminate cancer cells without harming other body cells

Normal cells usually have multiple solutions for fixing problems. For example, when DNA becomes damaged, healthy white blood cells can use several different strategies to make repairs.

28 May 2021

Insights into elusive protein complex can help find novel treatments for chromosomal disorders

The cells in our body are constantly fighting off the threat of cancer by repairing damaged DNA. In a new study, scientists from Tokyo University of Science investigate the structure of an elusive protein complex that plays a key role in the activation of the "Fanconi anemia pathway" involved in DNA repair, and report on the factors governing its stability.

17 Mar 2021

Scientists examine DNA repair pathways and their impact on cancer evolution

DNA repair pathways exist to correct molecular damage caused by internal and external factors. However, any damage to these pathways can result in the creation of tumors and specific types of cancers.

17 Feb 2021

SU2C announces $3.25 million in grants to find new treatments for head and neck cancers

Stand Up To Cancer today announced $3.25 million in grants from four national nonprofits to fund research to find new treatments for head and neck cancers, which are newly diagnosed in about 65,000 Americans every year.

21 Jan 2021

Inherited DNA defects cause squamous cell carcinoma in kids with Fanconi anemia

Physicians and scientists at Cincinnati Children's Hospital Medical Center used new stem cell technology to regenerate and study living patient-specific skin in the lab.

24 Nov 2020

Study uncovers the mechanism behind bone marrow failure in Fanconi anemia

Fanconi anaemia (FA) is a genetic disease affecting small children characterized by bone-marrow failure, developmental abnormalities and predisposition to multiple forms of cancer.

14 Oct 2020

New repair mechanism for alcohol-induced DNA damage discovered

Researchers of the Hubrecht Institute in Utrecht, The Netherlands, and the MRC Laboratory of Molecular Biology in Cambridge, United Kingdom, have discovered a new way in which the human body repairs DNA damage caused by a degradation product of alcohol.

4 Mar 2020

Researchers discover unexpected survival mechanism of a subset of cancer cells

Embedded at the end of chromosomes are structures called "telomeres" that in normal cells become shorter as cells divide. As the shortening progresses it triggers cell proliferation arrest or death. Cancer cells adopt different strategies to overcome this control mechanism that keeps track of the number of times that a cell has divided.

28 May 2019

WVU researcher studies stubborn blood disease that strikes children

Treating a stubborn blood disease that strikes children may come down to tweaking energy production in stem cells, suggests research out of West Virginia University.

14 May 2019