Ranucci, Giusy
(2018)
PROSPECTIVE DEVELOPMENTS TOWARDS NEW THERAPIES IN WILSON'S DISEASE.
[Tesi di dottorato]
| Tipologia del documento: |
Tesi di dottorato
|
| Lingua: |
English |
| Titolo: |
PROSPECTIVE DEVELOPMENTS TOWARDS NEW THERAPIES IN WILSON'S DISEASE |
| Autori: |
| Autore | Email |
|---|
| Ranucci, Giusy | giusyranucci@hotmail.it |
|
| Data: |
10 Dicembre 2018 |
| Numero di pagine: |
90 |
| Istituzione: |
Università degli Studi di Napoli Federico II |
| Dipartimento: |
Scienze Mediche Traslazionali |
| Dottorato: |
Medicina clinica e sperimentale |
| Ciclo di dottorato: |
31 |
| Coordinatore del Corso di dottorato: |
| nome | email |
|---|
| BEGUINOT, FRANCESCO | beguino@unina.it |
|
| Tutor: |
| nome | email |
|---|
| IORIO, RAFFAELE | [non definito] |
|
| Data: |
10 Dicembre 2018 |
| Numero di pagine: |
90 |
| Parole chiave: |
WILSON'S DISEASE, DRUG DISCOVERING, RARE |
| Settori scientifico-disciplinari del MIUR: |
Area 06 - Scienze mediche > MED/38 - Pediatria generale e specialistica |
[error in script]
[error in script]
| Depositato il: |
19 Dic 2018 15:24 |
| Ultima modifica: |
26 Giu 2020 20:07 |
| URI: |
http://www.fedoa.unina.it/id/eprint/12548 |
Abstract
Wilson's disease (WD) is an autosomal recessive disorder of copper metabolism, caused by mutations in the ATP7B gene. A clear demand for novel WD treatment strategies has emerged. Although therapies using zinc salts and copper chelators can effectively cure WD, these drugs exhibit limitations in a substantial pool of WD patients who develop intolerance and/or severe side effects. Several lines of research have indicated intriguing potential for novel strategies and targets for development of new therapies. Here, we review these new approaches, which comprise correction of ATP7B mutants and discovery of new compounds that circumvent ATP7B-deficiency, as well as cell and gene therapies. We also discuss whether and when these new therapeutic strategies will be translated into clinical use, according to the key requirements for clinical trials that remain to be met. Finally, we discuss the hope for the current rapidly developing research on molecular mechanisms underlying WD pathogenesis and for the related potential therapeutic targets to provide a solid foundation for the next generation of WD therapies that may lead to an effective, tolerable and safe cure.
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