Research

1 Fanconi Hope-funded Research Activities

1.1 “Observational study for long term health implications of individuals affected by Fanconi anaemia” funded by Fanconi Hope. (Manchester University)

The principals in this study are:-

Read more about the Observational Study

1.2. International Fanconi Anaemia Gene Therapy Working Group

Since 2010, Fanconi Hope and the Fanconi Anemia Research Fund (FARF) have jointly organised and co-funded the International FA Gene Therapy Working Group, set up to bring together leading Gene Therapy and Fanconi Anaemia experts from across the globe to create an action plan for gene therapy trials in Fanconi Anaemia. The group is chaired by Prof Jakub Tolar from the University of Minnesota. Read more about the International Fanconi Anaemia Gene Therapy Working Group.

1.3 Previous Fanconi Hope-funded Research

Read about Previous Fanconi-Hope-funded Research

2. Relevant Non-Fanconi Hope-funded research and trials

2.1 Eurofancolen Gene Therapy Trial (2014+) led by Dr Juan Bueren at CIEMAT in Spain:
Phase I/II Gene therapy trial of Fanconi anemia patients with a new Orphan Drug consisting of a lentiviral vector carrying the FANCA gene: A Coordinated International Action.  Read more about the Eurofancolen Gene Therapy Trial.

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