A review in Genes & Diseases says robust non-clinical safety assessment is critical for CRISPR-based gene therapies. It highlights genotoxic, delivery, and immunological risks and recommends risk-based development programs.
Scientists have developed multiple breakthrough delivery systems for gene editing and mRNA therapies, including a simple amino acid supplement that increases delivery 20-fold and a CRISPR gene drive that reverses antibiotic resistance in bacterial populations.
Scientists have developed two breakthrough approaches to dramatically enhance gene editing and mRNA therapy delivery: a simple amino acid supplement that increases CRISPR efficiency to nearly 90 percent, and a self-replicating CRISPR system that spreads between cells like a virus.
Transposase systems are emerging as efficient alternatives to CRISPR-Cas9 for gene editing in biopharmaceutical manufacturing and plant breeding, with studies showing up to 90% efficiency and heritability rates while offering advantages in size and integration capabilities.
Researchers developed pancreatic-targeted lipid nanoparticles (AH-LNP) that enable precise mRNA delivery to the pancreas through capsule-filter-mediated accumulation. The platform demonstrated efficacy in genome editing and cancer immunotherapy across multiple animal models including non-human primates.
