Delivering the "Blueprints" or "DNA Repairing Kits" Instead of Drugs in the Treatment of Congenital Hemoglobinopathies

Abstract

Hemoglobinopathies, such as β-thalassemia and sickle cell disease (SCD), are among the most common congenital diseases in the world, with high mortality and morbidity rates. The most straightforward approach to correct the main culprit in hemoglobinopathies - the imbalance between the amounts of alpha-like globins/betalike globins- will need to target directly the gene structure or their regulatory elements in order to obtain a sustained therapeutic effect. Keywords: Genetic therapy, nucleic acids delivery strategies, hemoglobinopathies, DNA repairing, DNA editing, viral and non-viral vectors, nanoparticles.