Toward Combined Cell and Gene Therapy for Genodermatoses
- Laura De Rosa1,
- Maria Carmela Latella1,
- Alessia Secone Seconetti1,
- Cecilia Cattelani2,
- Johann W. Bauer3,
- Sergio Bondanza1 and
- Michele De Luca2
- 1Holostem Terapie Avanzate S.r.l., Center for Regenerative Medicine “Stefano Ferrari,” 41125 Modena, Italy
- 2Center for Regenerative Medicine “Stefano Ferrari,” Department of Life Sciences, University of Modena and Reggio Emilia, 41125 Modena, Italy
- 3EB House Austria and Department of Dermatology, University Hospital of the Paracelsus Medical University, 5020 Salzburg, Austria
- Correspondence: michele.deluca{at}unimore.it
Abstract
To date, more than 200 monogenic, often devastating, skin diseases have been described. Because of unmet medical needs, development of long-lasting and curative therapies has been consistently attempted, with the aim of correcting the underlying molecular defect. In this review, we will specifically address the few combined cell and gene therapy strategies that made it to the clinics. Based on these studies, what can be envisioned for the future is a patient-oriented strategy, built on the specific features of the individual in need. Most likely, a combination of different strategies, approaches, and advanced therapies will be required to reach the finish line at the end of the long and winding road hampering the achievement of definitive treatments for genodermatoses.