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The Analytical Scientist – Defying Uncertainty in Gene Therapy

Defying Uncertainty in Gene Therapy

How new mass spectrometry-based technology is allowing researchers to characterize proteins and gene editing off-target effects with greater sensitivity and reliability

by Hugo Gagnon, CSO, Allumiqs

Recent advances in gene therapy – such as CRISPR/Cas9-based tools – are allowing researchers to introduce or modify genes with unprecedented feasibility. These technologies are hugely exciting from a therapeutic standpoint, particularly in addressing rare and chronic diseases. The ability to potentially heal lethal diseases based on genetic variants, such as muscular dystrophy, cystic fibrosis, and Huntington’s disease, explains the hype around these technologies. In addition, the versatility of gene therapy approaches seems endless.