Functional Improvement of dystrophic muscle by utrophin gene editing
Trinath Ghosh, S. K. Tousif Ahamed, Jun-Chao Xu et al.
· Molecular Therapy Advances · 0 citations
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Review
Open access
2026
Artificial intelligence in the rational design of lipid nanoparticles for mRNA therapeutics
This review surveys how artificial intelligence is converting this nonlinear design space from empirical iteration into data-efficient, multi-objective optimization and outlines three priorities for translation: standardized data and metadata, mechanistic endpoints for escape and immunogenicity, and CMC-aware optimizat...
Heyu Zhao, Junchao Xu, Xia Gao et al.
· The Innovation Drug Discover... · 2 citations