AAV's widespread application is currently confronting critical challenges. This study established a systematic framework of ‘natural discovery–evolutionary analysis–functional optimization’, providing a new paradigm for the development of next‐generation AAV vectors with clinical‐grade tissue specificity, low immunogenicity, and cross‐species ...
Liyu Zhu +11 more
wiley +1 more source
Bioengineered AAV9 and Optimised Microdystrophin Vectors Augment Phenotypic Rescue in a Murine Model of Duchenne Muscular Dystrophy. [PDF]
Senthilkumar MB +14 more
europepmc +1 more source
In the oxygen‐induced retinopathy (OIR) mouse model, two distinct microglial subpopulations play opposing roles: highly glycolytic microglia (HGM) promote neovascular formation via Pkm2, while Mrc1/CD206‐positive phagocytosis‐associated microglia (PAM) facilitate its regression.
Yuan Ma +20 more
wiley +1 more source
Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies. [PDF]
Duan D, Herzog RW.
europepmc +1 more source
Application Strategies of Bone Marrow Mesenchymal Stromal Cells in Bone‐Related Diseases
Engineered BMSCs and vesicles enhance therapy effects for bone diseases via multi‐strategic approaches. ABSTRACT Bone‐related diseases (e.g., osteoporosis, osteoarthritis and fractures) exhibit a rising global incidence, imposing significant burdens on both quality of life and healthcare systems.
Xuemei Long +6 more
wiley +1 more source
The Promise and Pitfalls of AAV-Mediated Gene Therapy for Duchenne Muscular Dystrophy. [PDF]
Kurshakova EV +3 more
europepmc +1 more source
Retinoic Acid Signalling Regulates Zebrafish Tooth Germ Repair Following Injury
Retinoic acid signalling may regulate the repair processes in a tooth germ injury model using Tg(scpp5:Dendra2‐NTR) zebrafish and the nitroreductase (NTR)/metronidazole (MTZ) system. ABSTRACT Although the role of retinoic acid (RA) signalling in odontogenesis is well established, its involvement in the repair of injured tooth germs remains unclear.
Qiqi Liu +4 more
wiley +1 more source
Callus and endosperm green fluorescence reporter-assisted selection system in maize CRISPR/Cas9 gene editing. [PDF]
Ge L +8 more
europepmc +1 more source
Moderate overexpression of wild‐type Met in hepatocytes (Alb‐R26Met mouse model) boosts a strong antioxidant response dependent on the glutathione system while impairing TGF‐β signaling in the liver. This leads to improved liver regeneration and protects against DDC‐induced injury, a model for cholestatic disease sharing features with primary ...
Carlos González‐Corralejo +16 more
wiley +1 more source
Replication-Competent, Tumor-Specific Immuno-Gene Vectors Allow for Exchange of Transgenes and Lead to Viral Persistence Following IV Administration. [PDF]
Rosen LS +12 more
europepmc +1 more source

