Journal of hepatology · 1 Sept 2026 · Editorial

Optimized lentiviral vectors a solution for paediatric metabolic liver diseases.

Sampaziotis, Gonzalez-Aseguinolaza

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The ITCC-P4 PDX Platform Enables Preclinical Testing of Pediatric Cancers.
Cancer Research · 2 Oct 2026 · Journal Article
The ITCC-P4 platform developed 353 pediatric cancer PDX models, enabling preclinical testing and biomarker discovery for high-risk solid tumors.
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One-stage assay factor VIII activity reflects AAV-derived factor VIII-enhanced thrombin activation and predicts phenotype.
Blood · 1 Oct 2026 · Journal Article
One-stage assay FVIII activity accurately reflects AAV-FVIII-SQ function and better predicts bleeding outcomes in hemophilia A gene therapy than chromogenic assays.
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Towards mRNA therapeutics 2.0.
Nature Reviews Drug Discovery · 1 Oct 2026 · Review
Innovative delivery systems and formulations are key to advancing mRNA therapeutics 2.0, enabling repeated administration and targeted organ delivery beyond the liver.
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https://jclub.com.au/a/42236989
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