IMAGINE INSTITUT DES MALADIES GENETIQUES NECKER ENFANTS MALADES FONDATION

Forschungsprojekte

• Assessing efficacy and safety of genome EDITing approaches for Sickle Cell Disease Sickle cell disease (SCD) is one of the most prevalent monogenic diseases in Europe. A single amino acid subs…
• Cellular Homeostasis ANd AGing in Connective TissuE Disorders The increased longevity in developed countries not necessarily goes hand in hand with amelioration of health…
• Inborn errors of translation reinitiation in humans with Mendelian susceptibility to mycobacterial disease Mendelian susceptibility to mycobacterial disease (MSMD) is characterized by severe infections with weakly vi…
• NAD+ International Scientist-training Nicotinamide adenine dinucleotide (NAD+) is a central redox cofactor and the limiting substrate of key metabo…
• New Prime Editing and non-viral delivery strategies for Gene Therapy Genome editing technologies based on CRISPR/Cas systems allow targeted genomic modification with unprecedente…