MISSION

Sickle cell disease (SCD) is a genetic anemia caused by mutations in the gene encoding the adult hemoglobin β-chain and leads to a severe clinical phenotype characterized by anemia, pain crises, and organ damage. Patients with linked mutations that trigger elevated g-globin expression, which is normally expressed only during fetal life, experience a more benign clinical course of the disease. The host lab developed a genome editing strategy that was recently proven successful in increasing g-globin expression in adult red blood cells. This approach is based on the use of the CRISPR/Cas9 system to disrupt g-globin inhibitory regulatory elements. To demonstrate the feasibility of this therapeutic approach, we need to develop protocols that allow the safe and efficient genetic modification of patient hematopoietic stem/progenitor cells. The student will apply state-of-art and novel molecular and cellular biology techniques to assess the CRISPR/Cas9 efficiency and potential toxicity in primary human hematopoietic stem/progenitor cells.

COMPETENCES

Expertise in molecular and cellular biology

LIEU DU STAGE

Laboratory of Chromatin and Gene Regulation During Development

Institut Imagine

24 Boulevard du Montparnasse

75015 Paris

CONTRAT

• Durée du stage : 4-6 mois
• Date de début souhaitée : Septembre 2020
• Gratification : oui

CONTACT

Dr. Annarita MICCIO, annarita.miccio@institutimagine.org