Marzo 2006 - Volume XXV - numero 3

Medico e Bambino


Focus

Prospettive di nuove terapie della fibrosi cistica

MASSIMO CONESE1, LUCIA PALMIERI, ELENA COPRENI Istituto per il Trattamento Sperimentale della Fibrosi Cistica, Ospedale S. Raffaele, Milano

1Dipartimento di Scienze Biomediche, Università di Foggia

Indirizzo per corrispondenza: conese.massimo@hsr.it

NEW THERAPIES OF CYSTIC FIBROSIS

Key words: Adenoassociate virus, Bone marrow, Cardiac glycosides, Curcumin, Nanoparticles, Pharmacogenomics, Stem cells

Although to date symptomatic therapies for cystic fibrosis (CF) - based on the eradication/ control of opportunistic infections and facilitation of mucus excretion - have rapidly and significantly increased patients survival, an aetiological therapy is still far from being available. There are several ongoing studies with the aim of correcting the underlying defect of the disease ranging from gene therapy to studies on molecules which will help the mutated CFRT gene to correctly express its protein or to make it work better. Stem cell therapy is a promising technique since it does not require the use of vectors, fundamental in gene therapy. Pharmacogenomics will help developing new drugs and identifying new efficacy indicators of these drugs.

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M. Conese, L. Palmieri, E. Copreni Prospettive di nuove terapie della fibrosi cistica. Medico e Bambino 2006;25(3):155 https://www.medicoebambino.com/?id=0603_155.pdf


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