Pharmacological chaperone therapy for lysosomal storage diseases (Articolo in rivista)

Type

• Articolo in rivista (Classe)
• Prodotto della ricerca (Classe)

Label

• Pharmacological chaperone therapy for lysosomal storage diseases (Articolo in rivista) (literal)

Anno

• 2014-01-01T00:00:00+01:00 (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#doi

• 10.4155/fmc.14.40 (literal)

Alternative label

• Parenti G.; Moracci M.; Fecarotta S.; Andria G. (2014)
  Pharmacological chaperone therapy for lysosomal storage diseases
  in Future medicinal chemistry (Print)
  (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#autori

• Parenti G.; Moracci M.; Fecarotta S.; Andria G. (literal)

Pagina inizio

• 1031 (literal)

Pagina fine

• 1045 (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#url

• http://www.scopus.com/inward/record.url?eid=2-s2.0-84905164552&partnerID=q2rCbXpz (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#numeroVolume

• 6 (literal)

Rivista

• Future medicinal chemistry (Rivista)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#numeroFascicolo

• 9 (literal)

Note

• Scopu (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#affiliazioni

• Telethon Institute of Genetics and Medicine, Naples, Italy; Department of Translational Medical Sciences, Section of Pediatrics, Federico II University, Naples, Italy; Institute of Biosciences and Bioresources, National Research Council (CNR), Naples, Italy (literal)

Titolo

• Pharmacological chaperone therapy for lysosomal storage diseases (literal)

Abstract

• Pharmacological chaperone therapy is an emerging approach to treat lysosomal storage diseases. Small-molecule chaperones interact with mutant enzymes, favor their correct conformation and enhance their stability. This approach shows significant advantages when compared with existing therapies, particularly in terms of the bioavailability of drugs, oral administration and positive impact on the quality of patients' lives. On the other hand, future research in this field must confront important challenges. The identification of novel chaperones is indispensable to expanding the number of patients amenable to this treatment and to optimize therapeutic efficacy. It is important to develop new allosteric drugs, to address the risk of inhibiting target enzymes. Future research must also be directed towards the exploitation of synergies between chaperone treatment and other therapeutic approaches. © 2014 Future Science Ltd. (literal)

Prodotto di

• Studio del Rapporto Struttura-Funzione e progettazione di Enzimi e Proteine (SV.P14.013.001) (Modulo)
• Istituto di Bioscienze e Biorisorse (IBBR) (Istituto)

Autore CNR

• MARCO MORACCI (Persona)

Incoming links:

Autore CNR di

• MARCO MORACCI (Persona)

Prodotto

• Istituto di Bioscienze e Biorisorse (IBBR) (Istituto)
• Studio del Rapporto Struttura-Funzione e progettazione di Enzimi e Proteine (SV.P14.013.001) (Modulo)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#rivistaDi

• Future medicinal chemistry (Rivista)