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Toward Combined Cell and Gene Therapy for Genodermatoses.

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.

SUBMITTER: De Rosa L

PROVIDER: S-EPMC7197428 | biostudies-literature | 2020 May

REPOSITORIES: biostudies-literature

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Toward Combined Cell and Gene Therapy for Genodermatoses.

De Rosa Laura L Latella Maria Carmela MC Secone Seconetti Alessia A Cattelani Cecilia C Bauer Johann W JW Bondanza Sergio S De Luca Michele M

Cold Spring Harbor perspectives in biology 20200501 5

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 speci ...[more]

PMID: 31653644

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