The Genetic Revolution for Fanconi Anemia Begins Now 

World-Renown Experts Take On the Root of the Problem

The long-awaited genetic revolution for rare genetic diseases has arrived and with it, the potential to cure diseases like FA within our lifetimes using state-of-the-art gene therapy (gene replacement) and gene editing (gene correction). 

Addressing the root cause of FA will require correction of each one of the 23 genes that may be mutated in individuals with FA. The replacement, or therapy of FA genes, aims to prevent bone marrow failure early in life and reduce the high risk of squamous cell cancers seen in young adults with FA. 

While significant progress has improved bone marrow transplants, the treatment can increase the risk of cancer due to the use of DNA damaging agents during transplant. 

Gene therapy and gene editing would bypass the need for a transplant because an individual’s own stem cells would express the dysfunctional genes. Ongoing gene therapy trials for FA have shown great promise in replacing FA genes to correct hematopoietic stem cells and prevent bone marrow failure. These technologies, however, can be costly and introduce complications due to the nature of removing cells from the body to replace the FA genes. 

This is why the Fanconi Cancer Foundation is proud to develop and fund a ‘Dream Team’ that will advance in vivo (inside the body) gene editing technologies for FA. 

The Dream Team, recruited by FCF in 2023, is comprised of seven world-renowned gene editing experts, and an industry partner, Nanovation Therapeutics. The researchers will work together to investigate new types of gene editing, called base and prime editing, which offer the opportunity to safely correct mutations in all 23 FA genes. The team is expected to leverage their expertise and rapidly translate preclinical research into clinical trials. This represents the beginning of a very promising genetic revolution that will pave the way for a potential cure for FA. 

Thank you to the Kendall and Taylor Atkinson Foundation for the generous contribution to help fund this project. 

Categories: Announcements, Research

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