Durata: 24 mesi (dal 01/09/2012 al 31/08/2014) Progetto n°: 01/2012 FFC Finanziato da: Fondazione per la Ricerca sulla Fibrosi Cistica-Bando 2012-Nonsense mutations are the leading cause of approximately 30% of inherited diseases, including cystic fibrosis (CF). They promote premature translational termination and following loss of CFTR protein by introduction of premature termination codons (PTCs). In the last few years, it has been demonstrated that drugs (like aminoglycoside antibiotics) can be designed and produced to suppress this process by the ribosomal read-through mechanism where these drugs mask PTC synthetizing a full-lengh CFTR protein. The rationale supporting of this project is to optimize the ribosomal read-through molecules leading to restoration of CFTR production in cystic fibrosis caused by stop mutation. This study may introduce new hopes for the development of a pharmacologic approach to the cure of CF.
L’approccio read-through per il trattamento della fibrosi cistica causata da mutazioni di stop Durata: 24 mesi (dal 01/09/2012 al 31/08/2014) Progetto n°: 01/2012 FFC Finanziato da: Fondazione per la Ricerca sulla Fibrosi Cistica-Bando 2012
BORGATTI, Monica
Primo
Funding Acquisition
;
2012
Abstract
Durata: 24 mesi (dal 01/09/2012 al 31/08/2014) Progetto n°: 01/2012 FFC Finanziato da: Fondazione per la Ricerca sulla Fibrosi Cistica-Bando 2012-Nonsense mutations are the leading cause of approximately 30% of inherited diseases, including cystic fibrosis (CF). They promote premature translational termination and following loss of CFTR protein by introduction of premature termination codons (PTCs). In the last few years, it has been demonstrated that drugs (like aminoglycoside antibiotics) can be designed and produced to suppress this process by the ribosomal read-through mechanism where these drugs mask PTC synthetizing a full-lengh CFTR protein. The rationale supporting of this project is to optimize the ribosomal read-through molecules leading to restoration of CFTR production in cystic fibrosis caused by stop mutation. This study may introduce new hopes for the development of a pharmacologic approach to the cure of CF.I documenti in SFERA sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
