Genetic engineering Genomic editing
Is it true that there is genomic editing that can permanently rid people of hereditary diseases?
Hi! In theory - yes. CRISPR can cure a number of genetic diseases ranging from blood diseases like sickle cell anemia to cancer.
But only subsequent mutations are not canceled by anyone
Genetic diseases can be diagnosed early in pregnancy, but many monogenic diseases continue to cause significant neonatal and childhood morbidity and mortality. However, early intervention through intrauterine gene editing could correct the genetic defect, which would potentially ensure the normal development of organs, improvement of functional state or cure. Intrauterine administration of nanoparticles containing peptide nucleic acids (PNA) and donor DNA corrects the disease-causing mutation in the β-globin gene in a mouse model of human β-thalassemia, leading to a steady postnatal increase in hemoglobin levels in the blood to the normal range, a decrease in the number of reticulocytes, a reversal of splenomegaly and an improvement in survival without detected non-target mutations in partially homologous loci.
@argentum Thanks for the answers and links to sources. The question of further mutations in the body is also interesting. Wouldn't changing the genome do even more harm than good?