Our unit was born with the idea that novel gene therapy strategies can be developed for the treat of bone marrow failure in different inherited bone marrow failure syndromes (IBMFS). Although allogeneic hematopoietic stem cell transplantation is the only curative treatment for IBMFS such as Fanconi Anemia (FA) and Diamond Blackfan Anemia (DBA), donor availability and the extensive comorbidities associated with the specific complications in both diseases highlight the need for new treatments for the patients. In this context, we are developing different strategies based on the use of lentiviral mediated gene therapy to correct HSCs from FA and DBA patients with very promising results. In parallel we are also developing novel gene editing approaches to finally correct specific mutations in both IBMFS maintaining the endogenous regulation of the gene. In both cases cellular stress analysis in HSCs associated to the different gene therapy strategies is being analyzed to finally obtained the most safe and efficient gene therapy alternative for IBMFS patients