Gene Therapy

Gene Therapy, Published online: 26 August 2026; doi:10.1038/s41434-026-00641-1 Mapping the global trends and hotspots of research on gene therapy and hair cell regeneration for hearing loss: a comprehensive data-mining-based study

Gene Therapy, Published online: 06 August 2026; doi:10.1038/s41434-026-00638-w Precision correction of the GJB2 c.235delC mutation by prime editing in vitro

Gene Therapy, Published online: 07 August 2026; doi:10.1038/s41434-026-00637-x An engineered helper plasmid generates differential E4orf6 and L4-22/33K gene expression increasing AAV vector production

Gene Therapy, Published online: 06 August 2026; doi:10.1038/s41434-026-00634-0 A gene therapy approach to prevent dilated intercellular space, a hallmark of gastroesophageal reflux disease

Gene Therapy, Published online: 29 July 2026; doi:10.1038/s41434-026-00635-z SACF and GILA for in vitro transformation assessment of CRISPR/Cas9-edited cell therapy candidates: a multi-site study

Gene Therapy, Published online: 29 July 2026; doi:10.1038/s41434-026-00636-y Bone- and muscle-targeted adeno-associated viral vectors enable tissue-selective vitamin D receptor knockdown in mice

Gene Therapy, Published online: 21 July 2026; doi:10.1038/s41434-026-00631-3 rAAV production cost analysis: Indication-specific cost per dose and reduction strategies

Gene Therapy, Published online: 18 July 2026; doi:10.1038/s41434-026-00633-1 Gene augmentation therapy successfully treats mice with complete congenital stationary night blindness (cCSNB), improving retinal function and visual acuity

Gene Therapy, Published online: 16 July 2026; doi:10.1038/s41434-026-00632-2 Reversing cancer cell behavior using AI-guided CRISPR and quantum nanobiology: a systems-based approach to epigenetic reprogramming

Gene Therapy, Published online: 10 July 2026; doi:10.1038/s41434-026-00630-4 FMR1 gene therapy restores translationally relevant phenotypes in a mouse model for fragile X syndrome

Gene Therapy, Published online: 27 June 2026; doi:10.1038/s41434-026-00628-y Recent advancements in improving cross-species applicability of bioengineered AAV capsids

Gene Therapy, Published online: 30 June 2026; doi:10.1038/s41434-026-00629-x Improving the precision of AAV lung gene therapy for SP-B deficiency using computationally derived lung-specific promoters

Gene Therapy, Published online: 18 June 2026; doi:10.1038/s41434-026-00623-3 Assessment of F/HN-pseudotyped lentiviral vector following intravenous delivery to mice

Gene Therapy, Published online: 15 June 2026; doi:10.1038/s41434-026-00627-z Applications of genome editing technologies in the treatment of human diseases

Gene Therapy, Published online: 04 June 2026; doi:10.1038/s41434-026-00626-0 High resolution ES-DMA quantifies AAV capsid DNA content by electrical mobility to mass correlation

Gene Therapy, Published online: 01 June 2026; doi:10.1038/s41434-026-00624-2 AAV8-mediated mouse/human PROC expression rescues thrombophilia in hereditary protein C-deficient mice

Gene Therapy, Published online: 29 May 2026; doi:10.1038/s41434-026-00625-1 Environmental and developmental factors shape anti-AAV immunity in pigs

Gene Therapy, Published online: 25 May 2026; doi:10.1038/s41434-026-00621-5 Research progress and development strategies of antibody-oligonucleotide conjugates

Gene Therapy, Published online: 26 May 2026; doi:10.1038/s41434-026-00622-4 DOCTER: a genetically encoded switchable protein module for ERα-mediated transcriptional regulation

Gene Therapy, Published online: 26 May 2026; doi:10.1038/s41434-026-00620-6 Characterization of CAR-T cell factors that contribute to myeloid cell activation