Gene therapy articles from across Nature Portfolio

Experiments in mice reveal an early postnatal window of opportunity for the effective transfer of genes to blood-cell-producing haematopoietic stem cells by injecting mice with gene-carrying lentiviral vectors. This approach showed therapeutic benefit in three mouse models of severe diseases, and could expand the applicability of haematopoietic stem-cell gene therapy in the clinic.

We developed Dual Selective Organ-Targeting Lipid Nanoparticles (Dual SORT LNPs) capable of delivering base editors to multiple organs. Base editor Dual SORT LNPs corrected disease-causing mutations in both the liver and lungs, the primary organs affected in alpha-1 antitrypsin deficiency.

Optimized TALE base editors are used to generate a rat model of mitochondrial disease and then revert the disease mutation in the next generation.

Treatment of cardiomyopathies has historically focused on symptom management. In this Review, Elliott and colleagues discuss how elucidation of the complex cellular and molecular pathways has led to the development of novel therapeutic agents that modify or mitigate the effects of causative genetic variants, enabling tailored treatment for cardiomyopathies.

Benamer et al. develop a knock-in mouse model carrying the E1799del otoferlin mutation to study the atypical DFNB9 variant form of deafness. They show the mouse model exhibits abnormal otoferlin distribution, failure of synaptic transmission in inner hair cells, and profound hearing loss, all of which can be restored with AAV gene therapy.

The average number of bleeding episodes for men with haemophilia B dropped almost tenfold after treatment.

Experiments in mice reveal an early postnatal window of opportunity for the effective transfer of genes to blood-cell-producing haematopoietic stem cells by injecting mice with gene-carrying lentiviral vectors. This approach showed therapeutic benefit in three mouse models of severe diseases, and could expand the applicability of haematopoietic stem-cell gene therapy in the clinic.

We developed Dual Selective Organ-Targeting Lipid Nanoparticles (Dual SORT LNPs) capable of delivering base editors to multiple organs. Base editor Dual SORT LNPs corrected disease-causing mutations in both the liver and lungs, the primary organs affected in alpha-1 antitrypsin deficiency.

Researchers develop a personalized base-editing therapy within 6 months for a newborn with a rare genetic disease — using rapid workflow that could be customized to other patients too.