Publications by authors named "Roser M Pujol"
Article Synopsis
- Lentiviral gene therapy can help patients with Fanconi anemia by improving bone marrow function.
- Researchers found that this therapy changes the cells to act more like healthy cells, even fixing some problems in the cells' instructions.
- This study shows that gene therapy might be a good treatment for other diseases with similar problems in the future.
Difficulties in the collection of hematopoietic stem and progenitor cells (HSPCs) from Fanconi anemia (FA) patients have limited the gene therapy in this disease. We have investigated (ClinicalTrials.gov, NCT02931071) the safety and efficacy of filgrastim and plerixafor for mobilization of HSPCs and collection by leukapheresis in FA patients.
View Article and Find Full Text PDFFanconi anemia (FA) is a DNA repair syndrome generated by mutations in any of the 22 FA genes discovered to date. Mutations in FANCA account for more than 60% of FA cases worldwide. Clinically, FA is associated with congenital abnormalities and cancer predisposition.
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