The ITCC-P4 platform developed 353 pediatric cancer PDX models, enabling preclinical testing and biomarker discovery for high-risk solid tumors.
- Open access
Virus-based gene therapy research
Moving in bioRxiv, Biotechnology advances, Blood, Journal of hepatology, Nature Communications, Nature Medicine, Cancer Research, Frontiers in Genetics.
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Latest in Virus-based gene therapy research
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Innovative delivery systems and formulations are key to advancing mRNA therapeutics 2.0, enabling repeated administration and targeted organ delivery beyond the liver.
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, meeting primary efficacy endpoints in the FAS population.
Lenacapavir disrupts HIV-1 capsid maturation by altering interhexamer interactions, preventing functional cone-shaped capsid formation during virion assembly.
- Open access
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.
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
Viral and non-viral vectors for in vivo CAR-T therapy show distinct advantages and limitations, impacting safety, efficacy, and scalability in clinical applications.
- Open access
- 1 cites
Liver-directed lentiviral gene therapy achieves long-lasting metabolic correction in MMA mice, with over 80% hepatocyte transduction and therapeutic effects lasting more than a year.
- Open access
- 2 cites
Suprachoroidal delivery of AL-001 achieves sustained aflibercept expression and superior efficacy with minimal inflammation in a nonhuman primate model of wet age-related macular degeneration.
- 1 opens in JClub
- Open access
Computational design methods have enabled the creation of optimized viral vectors, advancing gene therapy with significant improvements in vector properties.
- Open access
Engineering vector plasmids and cell lines has significantly enhanced rAAV production, overcoming capacity bottlenecks in the HEK293 system to meet clinical demand.
- Open access
- 1 cites
Viral exposures beyond oncogenesis can enhance cancer immunity, with endogenous retroviruses and microbiome-associated phages playing protective roles.
Gene therapy has achieved clinical breakthroughs for hereditary hematological disorders, with approved treatments for hemoglobinopathies and hemophilia, moving toward curative potential.
- Open access
Noncanonical P gene mRNA editing in Cedar virus produces a U protein essential for efficient virion production, challenging previous gene expression models.
- Open access
- 1 cites
A copper-based covalent organic framework combined with oncolytic virus enhances radiotherapy efficacy by inducing cuproptosis in radioresistant tumors, achieving durable immune responses.
- Open access
Recombinant tick-borne encephalitis viruses with stable reporter genes enable effective antiviral testing, with Nluc showing superior stability over eGFP in diverse cell types.
- Open access
-based CD45 radioimmunotherapy achieves effective, targeted conditioning with minimal non-hematologic toxicity in primates, enabling high-efficiency gene-edited HSPC engraftment.
- Open access
Long-term BCL11A silencing via shmiR vector in Sickle Cell Disease shows durable HbF induction and sustained clinical benefits over 48 months.
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