Topical gene editing therapeutics using lipid nanoparticles: 'gene creams' for genetic skin diseases?
Ina Guri-LamceYara AlRokhYoungah KimRuhina MaeshimaCarina GrahamStephen L HartJohn A McGrathJoanna Jackow-MalinowskaPublished in: The British journal of dermatology (2024)
Patients living with inherited skin diseases have benefited from recent advances in DNA sequencing technologies that provide new or improved diagnostics. However, developing and delivering new treatments for the 'genodermatoses' remains challenging. The goal of creating topical preparations that can recover the inherent gene pathology remains largely aspirational. However, recent progress in two fields - the chemistry of topical delivery formulations (lipid nanoparticles) and the molecular biology of gene repair (CRISPR-Cas9, base and prime editing) - presents new opportunities to address this unmet need. In this review, we discuss how lipid nanoparticle delivery vehicles could be used to deliver gene-editing tools to formulate topical 'gene creams' suitable for the treatment of genodermatoses. We summarize the historical landscape of topical therapeutics and advances in gene editing that may herald an era of new therapies for patients with inherited skin disorders.
Keyphrases
- wound healing
- crispr cas
- genome wide
- copy number
- genome editing
- end stage renal disease
- genome wide identification
- soft tissue
- small molecule
- fatty acid
- chronic kidney disease
- single cell
- newly diagnosed
- single molecule
- dna methylation
- circulating tumor
- genome wide analysis
- patient reported outcomes
- cell free
- smoking cessation
- replacement therapy