‘Gene creams’ for genetic diseases?

Extract

People living with inherited (genetic) skin diseases have ­benefitted from recent advances in DNA sequencing technologies that provide new or improved diagnostics. However, developing and delivering new treatments for these genetic skin diseases remains challenging.

The goal of creating topical treatments (treatment that is applied to the skin) that can restore the function of the faulty gene remains largely aspirational. However, recent progress in two fields: (1) the chemistry of topical delivery formulations (lipid nanoparticles) and (2) the molecular biology of gene repair (using a technique called ‘CRISPR-Cas9’), now presents new opportunities to address that unmet need.

In this review, we discuss how lipid nanoparticle delivery vehicles could be used to deliver gene editing tools to develop ‘gene creams’ suitable for the treatment of inherited skin ­diseases. We summarize the historical landscape of topical treatments and advances in gene editing, which may represent an era of new treatments for people with inherited skin diseases.