Background: Gene therapy is a new approach to discover and treat many diseases. It has attracted
considerable attention from researchers in the last decades. The gene therapy through RNA interference has been
considered one of the most recent and revolutionary approaches used in individualized therapy. In the last years,
we have witnessed the rapid development in the field of the gene silencing and knockdown by topical siRNA. Its
application in gene therapy has become an attractive alternative for drug development.
Methods: This article will address topical delivery of siRNA as a promising treatment for skin disorders. An
update on the advances in siRNA-based nanocarriers as a powerful therapeutic strategy for several skin diseases
will be discussed giving emphasis on in vitro evaluations.
Results: Through the in-depth review of the literature on the use of siRNAs for skin diseases we realize how
widespread this use is. We have also realized that nanoparticles as non-viral vectors are increasingly being explored.
Skin diseases where the use of siRNA has been explored most are skin cancer (melanoma and nonmelanoma),
psoriasis, vitiligo, dermatitis and leprosy. But we also report here other diseases where the use of
siRNA has been growing as acne, alopecia areata, cutaneous leishmaniasis, mycoses, herpes, epidermolysis bullosa
and oculocutaneous albinism. Also highlighted, the first clinical trial of siRNA for cutaneous diseases, aimed
at Pathyounychia Congenita.
Conclusion: The treatment of skin diseases based on topical delivery of siRNA, which act by inhibiting the expression
of target transcripts, offers many potential therapeutic advantages for suppressing genes into the skin.