Autophagy and gene therapy combine in the treatment of liver disease

作者：Thompson Debra A; Klionsky Daniel J^*

来源：Autophagy, 2013, 9(7): 945-946.

DOI：10.4161/auto.24475

• alpha(1)-antitrypsin deficiency
• lysosome
• macroautophagy
• TFEB

Molecular biology holds the promise not only of increasing our understanding of basic cell biology, but also of advancing our ability to design targeted therapeutic methods for treating a range of diseases. One example that seems to hold tremendous potential is gene therapy, the use of exogenous DNA to replace or suppress a mutant gene in the patient's genome, or to boost the activity of a normal gene. A recent report (highlighted in a punctum in this issue of the journal) has brought autophagy into the gene therapy realm.

• 出版日期2013-7-1