Toward Combined Cell and Gene Therapy for Genodermatoses

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...

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Bibliographic Details
Published in:Cold Spring Harbor perspectives in biology 2020-05, Vol.12 (5), p.a035667
Main Authors: De Rosa, Laura, Latella, Maria Carmela, Secone Seconetti, Alessia, Cattelani, Cecilia, Bauer, Johann W, Bondanza, Sergio, De Luca, Michele
Format: Article
Language:English
Subjects:
Animals
CRISPR-Cas Systems
Epidermis - metabolism
Epidermolysis Bullosa - therapy
Epidermolysis Bullosa Dystrophica - therapy
Epidermolysis Bullosa, Junctional - therapy
Gene therapy
Genes, Dominant
Genes, Recessive
Genetic Therapy - methods
Genetic Vectors
Humans
Lentivirus - genetics
Netherton Syndrome - therapy
PERSPECTIVES
Retroviridae - genetics
Simplexvirus
Skin - metabolism
Skin diseases
Skin Diseases - genetics
Skin Diseases - therapy
Stem Cells - cytology
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Summary: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.
ISSN:1943-0264
1943-0264
DOI:10.1101/cshperspect.a035667