New Integrated Approaches for Cystic Fibrosis
1Department of Biological Science, University of Sannio, Benevento
2Department of Medicine and Surgery, University of Siena – SI– Street Mario Bracci 16, Italy
*Corresponding Author: Amelia Morgillo, Department of Medicine and Surgery, University of Siena – SI– Street Mario Bracci 16, Italy.
October 19, 2023; Published: November 02, 2023
Introduction: Cystic fibrosis, while remaining a rare disease, is the most common autosomal recessive genetic form in the Caucasian population and is a chronic developmental multisystem disease for which until a few years ago there were only supportive therapies, which did not however modify the long-term outcome, inevitably characterized by chronic lung damage, nutritional imbalances and, in severe cases, delayed physical development in affected children and endocrine-metabolic disorders. Today, new drugs are available that act directly on the CFTR protein, which is responsible for clinical signs and symptoms. In this article we wanted to deepen the pharmacology of these new molecules and the trials concerning gene therapy and new pharmacological approaches.
Objective and Materials and Methods: A computerized search was carried out for the articles to be included through the use of international databases such as pubmed, scopus, researchgate, google scholar, by typing in keywords such as: gene therapy for cystic fibrosis and the names of the new drugs and integration with literature data. In addition, data from paper documents such as books and articles have been integrated. The articles relating to the new therapies just approved or in the process of being approved and the related studies were selected.
Results: Although it remains a non-curable disease to date, the therapeutic possibilities for cystic fibrosis are expanding considerably and to date four modulating drugs (correctors and enhancers) CTFR are already available in clinical practice. Furthermore, gene therapy-based methodologies are being developed to directly correct the causal genetic defect, even if they are not yet directly applicable in clinical practice. Although gene therapy in lung diseases has not yet been fully realized, recent efforts in molecular virology aimed at the development of vectors will undoubtedly lead to great benefits in the next decade in this area. From the point of view of safety and efficacy, the current most promising gene transfer systems to the lung for inherited genetic diseases are adeno-associated vectors.
Conclusion: Novel approaches for purifying large amounts of vectors have also made widespread clinical applications more viable. With the help of the appropriate animal model for a given disease and efficient vectors for gene transfer, the field of gene therapy is likely to see great progress in the next decade.
Keywords: CF; CFTR; Viral Vector; Gene Therapy and ATMP
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