On February 9th 2016 the Congress "ADVANCES OF GENE THERAPY IN MONOGENIC DISEASES AFECTING THE HEMATOPOIETIC SYSTEM" was held in Madrid.

Gene therapy of patients suering from a variety of monogenic diseases has experienced a remarkable progress both in terms of safety and ecacy. Such an improvement is particularly evident in approaches based on the ex vivo correction of hematopoietic stem cells (HSCs) with novel retroviral and lentiviral vectors. This workshop aims to share current protocols of HSC gene therapy with specialists treating patients suering from monogenic diseases aecting the hematopoietic system. Specic emphasis will be made on primary immunodeciencies, hemoglobinopathies and bone marrow failure syndromes.

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