Virus-based gene therapy research
Moving in bioRxiv, Biotechnology advances, Frontiers in Genetics, Journal of hepatology, Nature Biotechnology, Nature Communications, Nature Medicine and Nature Reviews Drug Discovery.
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Latest in Virus-based gene therapy research
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Engineered non-LTR retrotransposons, called Gene Writers, enable stable in vivo gene delivery of reporter and CAR-T genes in human cells and mouse models.
- Open access
Cas9 nickase targeting CAG/CTG repeats induces repeat contractions in HD models and improves phenotypes in a mouse model, with over 50% of striatal neurons affected.
A decade after their discovery in 2016, base editors have evolved into versatile tools used in clinical trials for multiple human diseases, including sickle cell disease and leukaemias.
Persistent SIV reservoirs in rhesus macaques are associated with a tissue microenvironment characterized by activated stress responses, metabolic rewiring, and immunosuppressive structures.
- Open access
- 2 cites
The ITCC-P4 platform established 353 molecularly characterized pediatric cancer PDX models for in vivo drug testing, capturing tumor heterogeneity and disease progression.
- Open access
Novel in vivo delivery systems and immune-silent mRNA formulations are advancing mRNA therapeutics 2.0, enabling repeated administration and targeted organ delivery.
AAV gene therapy BBM-H901 is safe and reduces bleeding episodes in 11 adolescents with hemophilia B, with FIX activity reaching an average of 41.8 IU/dl at 52 weeks.
Marsupial genome editing has only been achieved in Monodelphis domestica, despite the availability of high-quality genomes for other species.
Highly efficient retron-based gene editors for mammalian cells and vertebrates achieve editing levels comparable to conventional methods using a genetically encoded cassette.
- 7 cites
One-stage assay FVIII activity accurately reflects AAV-FVIII-SQ function and better predicts bleeding outcomes in hemophilia A gene therapy than chromogenic assays.
Intravenous VCN-01 combined with gemcitabine and nab-paclitaxel improves overall survival in metastatic pancreatic ductal adenocarcinoma, with median OS reaching 10.8 months in the FAS population.
AI-driven methods are transforming gene therapy development into a predictive, data-driven process across multiple stages, from target discovery to post-marketing surveillance.
- Open access
Lenacapavir disrupts HIV-1 capsid maturation by altering interhexamer interactions, preventing functional cone-shaped capsid formation during virion assembly.
- Open access
Novel CRBN molecular glues induce selective VAV1 degradation via a unique non-canonical RT-loop degron, with potency enhanced by conformational restriction strategies.
- Open access
- 4 cites
AAV9 delivery enables mitochondrial adenine base editing in mouse tissues, achieving modest A-to-G conversions over six months.
- Open access
NANITE strategy quadruples genome editing efficiency in cells and triples liver editing levels in mice by enabling enzyme transfer between cells.
- Open access
- 1 cites
Continuous bioprocessing significantly boosts yields and reduces costs in manufacturing AAV and LV vectors for gene therapy, with improvements up to 50% in efficiency.
- Open access
Computational approaches, including machine learning, are increasingly enabling the design of viral vectors for gene therapy, advancing the field significantly.
- Open access
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Topics moving, week to 23 Oct 2026
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