Supported Research

Transforming Treatment of Inherited Bone Marrow Failure in Fanconi Anemia by Precise In Vivo Genome Editing

2024 | Research Grant | Instituto de Investigación Sanitaria Fundación Jiménez Díaz; Harvard University; ETH Zurich; St. Vincent's Institute Fitzroy; Fred Hutchinson Cancer Center; University of Minnesota; Medical Center - University of Freiburg, Institute for Transfusion Medicine and Gene Therapy

Amount Funded: $1,258,190

Fanconi anemia (FA) is a rare inherited bone marrow failure syndrome characterized by chromosomal abnormalities and increased susceptibility to cancer. Allogeneic bone marrow transplantation is currently the only treatment for bone marrow failure, but its use is limited by the need for a suitable donor and potential side effects.

Base and prime editing will be implemented to target frequent mutations described in individuals with FA and will be combined with optimized viral and non-viral delivery systems to target HSCs in vivo to provide a promising avenue for the treatment of this inherited bone marrow failure syndrome.

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