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Research ArticleLactic acidosis
Phenylbutyrate Therapy for Pyruvate Dehydrogenase Complex Deficiency and Lactic Acidosis
- Rosa Ferriero1,
- Giuseppe Manco2,
- Eleonora Lamantea3,
- Edoardo Nusco1,
- Maria I. Ferrante4,
- Paolo Sordino4,
- Peter W. Stacpoole5,
- Brendan Lee6,7,
- Massimo Zeviani3 and
- Nicola Brunetti-Pierri1,8,*
- 1Telethon Institute of Genetics and Medicine, Naples 80131, Italy.
- 2Institute of Protein Biochemistry, Naples 80131, Italy.
- 3Besta Institute, Milan 20126, Italy.
- 4Stazione Zoologica Anton Dohrn, Naples 80121, Italy.
- 5Departments of Medicine and Biochemistry and Molecular Biology, University of Florida College of Medicine, Gainesville, FL 32610, USA.
- 6Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
- 7Howard Hughes Institute, Baylor College of Medicine, Houston, TX 77030, USA.
- 8Department of Pediatrics, Federico II University of Naples, Naples 80131, Italy.
- ↵*To whom correspondence should be addressed. E-mail: brunetti{at}tigem.it
See allHide authors and affiliations
Science Translational Medicine 06 Mar 2013:
Vol. 5, Issue 175, pp. 175ra31
DOI: 10.1126/scitranslmed.3004986
Vol. 5, Issue 175, pp. 175ra31
DOI: 10.1126/scitranslmed.3004986
Rosa Ferriero
1Telethon Institute of Genetics and Medicine, Naples 80131, Italy.
Giuseppe Manco
2Institute of Protein Biochemistry, Naples 80131, Italy.
Eleonora Lamantea
3Besta Institute, Milan 20126, Italy.
Edoardo Nusco
1Telethon Institute of Genetics and Medicine, Naples 80131, Italy.
Maria I. Ferrante
4Stazione Zoologica Anton Dohrn, Naples 80121, Italy.
Paolo Sordino
4Stazione Zoologica Anton Dohrn, Naples 80121, Italy.
Peter W. Stacpoole
5Departments of Medicine and Biochemistry and Molecular Biology, University of Florida College of Medicine, Gainesville, FL 32610, USA.
Brendan Lee
6Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX 77030, USA.
7Howard Hughes Institute, Baylor College of Medicine, Houston, TX 77030, USA.
Massimo Zeviani
3Besta Institute, Milan 20126, Italy.
Nicola Brunetti-Pierri
1Telethon Institute of Genetics and Medicine, Naples 80131, Italy.
8Department of Pediatrics, Federico II University of Naples, Naples 80131, Italy.
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