Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain
the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in
Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles
and JavaScript.
Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a functional copy. Alternatively DNA encoding a therapeutic protein drug may be introduced.
Primary human hepatocyte (PHH) transplantation could be an alternative to liver transplantation. Here, the authors use the mRNA-LNP platform to express growth factors in the liver in a controlled manner to drastically improve PHH engraftment, thus, reducing disease burden and enhancing overall liver function.
Sickle cell disease is a blood disorder that originates from a single point mutation in the HBB gene that codes for hemoglobin. Here, Moiani et al. developed an efficient TALEN-mediated HBB correction process that is compatible with gene therapy applications.
Recombinases generated by phage-assisted evolution enhance the efficiency of the prime-editing-assisted targeted integration of large genes in mammalian cells.
An interim analysis of a single-arm trial in 5 children with hereditary deafness shows that binaural AAV gene therapy is safe and leads to hearing improvement up to 13–26 weeks of follow-up.
Cryo-electron microscopy structures of the prime editor bound to a prime editing guide RNA and target DNA, in the pre-initiation, initiation and elongation and termination states, provide insights into the mechanism by which prime editing occurs.
Researchers in India fought to develop what could have been the first therapy to use gene-editing to halt a rare neurodegenerative disease. The efforts hold lessons for the messy state of modern drug development.
Haemophilia A is caused by variants in the gene that encodes coagulation factor VIII (FVIII). Sequencing of this gene in the 1980s was the initial step in developing replacement therapy with recombinant FVIII, and thereby removing the risk of blood-borne infections from plasma-derived FVIII.
Final results of two studies — whose preliminary data led to regulatory approval of the gene therapy exagamglogene autotemcel — describe highly effective treatment of sickle-cell disease and transfusion-dependent β-thalassemia.