Toward combined cell and gene therapy for genodermatoses
| Autor: | Laura De Rosa, Johann W. Bauer, Michele De Luca, Sergio Bondanza, Alessia Secone Seconetti, Maria Carmela Latella, Cecilia Cattelani |
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| Jazyk: | angličtina |
| Rok vydání: | 2020 |
| Předmět: |
0301 basic medicine
medicine.medical_specialty Genetic enhancement Genetic Vectors MEDLINE Genes Recessive Biology Skin Diseases General Biochemistry Genetics and Molecular Biology 03 medical and health sciences 0302 clinical medicine medicine Animals Humans Simplexvirus Intensive care medicine Genes Dominant Skin Stem Cells Lentivirus Genetic Therapy Finish line Epidermolysis Bullosa Dystrophica Retroviridae 030104 developmental biology PERSPECTIVES Netherton Syndrome 030220 oncology & carcinogenesis CRISPR-Cas Systems Epidermis Epidermolysis Bullosa Epidermolysis Bullosa Junctional |
| Zdroj: | Cold Spring Harb Perspect Biol |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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