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Effective AAV‐mediated gene therapy in a mouse model of ...
Ethylmalonic encephalopathy (EE) is an invariably fatal disease, characterized by the accumulation of hydrogen sulfide (H 2 S), a highly toxic compound. ETHE1, encoding sulfur dioxygenase (SDO), which takes part in the mitochondrial pathway that converts sulfide into harmless sulfate, is mutated in EE.The main source of H 2 S is the anaerobic bacterial flora of the colon, although in trace ...
Effective AAV‐mediated gene therapy in a mouse model of ...
The paper explained. PROBLEM: Ethylmalonic encephalopathy (EE) is an invariably fatal disease characterized by the accumulation of hydrogen sulfide (H 2 S), a highly toxic compound. The main source of H 2 S is the anaerobic bacterial flora of the colon, although it is also produced by tissues in trace amounts, where it acts as a “gasotransmitter”. EE is caused by mutations in ETHE1, a gene ...
Effective AAV-mediated gene therapy in a mouse model of ...
**Corresponding author: Tel: þ39 02 2394 2630; Fax: þ39 02 23942619; E-mail: zeviani@istituto-besta.it 1008 2012 The Authors. Published by John Wiley and Sons, Ltd on behalf of EMBO. This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and
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