Challenges in the gene therapy of bone marrow failure syndromes

Juan Bueren is the Head of the Division of Hematopoietic Innovative Therapies of CIEMAT and CIBER of Rare Diseases. Dr Bueren is also Coordinator of the Mixed Unit of Advanced Therapies CIEMAT/IIS-Jiménez Díaz Foundation and consultant of Rocket Pharmaceuticals Inc, and currently serves as Vice-President of the European Society for Cell and Gene Therapy. Dr Bueren is Scientific Director of gene therapy trials for patients with Fanconi anemia and also for patients with the primary immunodeficiency, leukocyte adhesion deficiency type I, and has participated in the development of therapeutic lentiviral vectors designed as Orphan Drugs by the European Medicines Agency and by the FDA for these two diseases. Both ODs have been licensed to Rocket Pharmaceuticals which is currently developing different global gene therapy programs in Europe and in the USA for Fanconi anemia and LAD-I.

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