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Clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR‐associated (Cas) protein gene editing is poised to transform the treatment of genetic diseases. However, limited progress has been made toward precise editing of DNA via homology‐directed repair (HDR) that requires careful orchestration of complex steps. Herein, dendrimer‐based lipid nanoparticles (dLNPs) are engineered to co‐encapsulate...
mRNA‐mediated protein replacement represents a promising concept for the treatment of liver disorders. Children born with fumarylacetoacetate hydrolase (FAH) mutations suffer from Hepatorenal Tyrosinemia Type 1 (HT‐1) resulting in renal dysfunction, liver failure, neurological impairments, and cancer. Protein replacement therapy using FAH mRNA offers tremendous potential to cure HT‐1, but is currently...
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