Genome-based therapeutic interventions for β-type hemoglobinopathies.

ABSTRACT: For decades, various strategies have been proposed to solve the enigma of hemoglobinopathies, especially severe cases. However, most of them seem to be lagging in terms of effectiveness and safety. So far, the most prevalent and promising treatment options for patients with β-types hemoglobinopathies, among others, predominantly include drug treatment and gene therapy. Despite the significant improvements of such interventions to the patient's quality of life, a variable response has been demonstrated among different groups of patients and populations. This is essentially due to the complexity of the disease and other genetic factors. In recent years, a more in-depth understanding of the molecular basis of the β-type hemoglobinopathies has led to significant upgrades to the current technol

SUBMITTER: Karamperis K 

PROVIDER: S-EPMC8178887 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature