title: Autophagy and gene therapy combine in the treatment of liver disease.
abstract: 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.
authors.affiliation: Department of Ophthalmology and Visual Sciences and Department of Biological Chemistry; University of Michigan; Ann Arbor, MI USA.
authors.forename: Debra A
authors.initials: DA
authors.lastname: Thompson
authors.forename: Daniel J
authors.initials: DJ
authors.lastname: Klionsky
mesh_terms: D000818:Animals; D001343:Autophagy; D051778:Basic Helix-Loop-Helix Leucine Zipper Transcription Factors / Q000378:metabolism; D004247:DNA / Q000378:metabolism; D015316:Genetic Therapy; D006801:Humans; D008107:Liver Diseases / Q000235:genetics* / Q000628:therapy*
chemical_list: D051778:Basic Helix-Loop-Helix Leucine Zipper Transcription Factors; D004247:DNA
keywords: TFEB; lysosome; macroautophagy; α1-antitrypsin deficiency