Supported projects

D201702 – Preclinical development of new lentiviral vectors and gene therapy strategies to treat sickle cell disease

Scientific leader
Annarita Miccio

Sickle cell disease is the most common genetic disease worldwide and is caused by a point mutation in the gene coding for the adult hemoglobin β-chain. So far, the only curative treatment is represented by transplantation of normal hematopoietic stem cells from compatible donors that, however, are available only to a fraction of the patients. Gene therapy approaches aimed at correcting patient hematopoietic stem cells represent a valuable alternative therapeutic strategy.

Patients with linked mutations that trigger elevated g-globin expression, which are normally expressed only during fetal life, experience a more benign clinical course of the disease. The aim of this project is to develop novel lentiviral- and genome editing-based approaches aimed to: (i) deliver into patient cells an optimized therapeutic g-globin-like gene; (ii) reactivate endogenous g-globin expression. Evaluation of the efficacy and safety of these approaches will be performed to select the best therapeutic strategy for sickle cell disease.

Funding
Institut Imagine, DIM