[Genome Editing in Therapy of Genodermatoses]

Area of Science:

• Genetics
• Dermatology
• Molecular Biology

Background:

• Genodermatoses are inherited skin diseases with limited treatment options.
• Epidermolysis bullosa is a primary focus due to the applicability of various therapeutic methods.

Approach:

• This review examines gene therapy approaches, emphasizing genome editing using programmable nucleases.
• Methods are classified based on DNA repair pathways: canonical non-homologous end joining (cNHEJ) and homologous recombination (HDR).
• In vitro applications on diverse cell types are detailed, alongside in vivo strategies.

Key Points:

• Genome editing strategies are tailored to specific mutation types, inheritance patterns, and the surrounding DNA sequence.
• Animal models generated via genome editing are crucial for preclinical research.
• The review summarizes the development and application of genome editing for previously untreatable genodermatoses.

Conclusions:

• Genome editing represents a significant advancement in the therapeutic landscape for genodermatoses.
• The precise control offered by programmable nucleases opens avenues for correcting genetic defects in skin disorders.
• Continued research and application of these techniques hold promise for curing inherited skin diseases.