Literature DB >> 23590992

Autophagy and gene therapy combine in the treatment of liver disease.

Debra A Thompson1, Daniel J Klionsky.   

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.

Entities:  

Keywords:  TFEB; lysosome; macroautophagy; α1-antitrypsin deficiency

Mesh:

Substances:

Year:  2013        PMID: 23590992      PMCID: PMC3722329          DOI: 10.4161/auto.24475

Source DB:  PubMed          Journal:  Autophagy        ISSN: 1554-8627            Impact factor:   16.016


  19 in total

1.  TFEB regulates autophagy: an integrated coordination of cellular degradation and recycling processes.

Authors:  Carmine Settembre; Andrea Ballabio
Journal:  Autophagy       Date:  2011-11-01       Impact factor: 16.016

2.  PGC-1α rescues Huntington's disease proteotoxicity by preventing oxidative stress and promoting TFEB function.

Authors:  Taiji Tsunemi; Travis D Ashe; Bradley E Morrison; Kathryn R Soriano; Jonathan Au; Ruben A Vázquez Roque; Eduardo R Lazarowski; Vincent A Damian; Eliezer Masliah; Albert R La Spada
Journal:  Sci Transl Med       Date:  2012-07-11       Impact factor: 17.956

3.  Rapamycin reduces intrahepatic alpha-1-antitrypsin mutant Z protein polymers and liver injury in a mouse model.

Authors:  Shalesh Kaushal; Mani Annamali; Keith Blomenkamp; David Rudnick; Donna Halloran; Elizabeth M Brunt; Jeffrey H Teckman
Journal:  Exp Biol Med (Maywood)       Date:  2010-06

4.  Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

Authors:  Alessandro Aiuti; Federica Cattaneo; Stefania Galimberti; Ulrike Benninghoff; Barbara Cassani; Luciano Callegaro; Samantha Scaramuzza; Grazia Andolfi; Massimiliano Mirolo; Immacolata Brigida; Antonella Tabucchi; Filippo Carlucci; Martha Eibl; Memet Aker; Shimon Slavin; Hamoud Al-Mousa; Abdulaziz Al Ghonaium; Alina Ferster; Andrea Duppenthaler; Luigi Notarangelo; Uwe Wintergerst; Rebecca H Buckley; Marco Bregni; Sarah Marktel; Maria Grazia Valsecchi; Paolo Rossi; Fabio Ciceri; Roberto Miniero; Claudio Bordignon; Maria-Grazia Roncarolo
Journal:  N Engl J Med       Date:  2009-01-29       Impact factor: 91.245

5.  Effect of gene therapy on visual function in Leber's congenital amaurosis.

Authors:  James W B Bainbridge; Alexander J Smith; Susie S Barker; Scott Robbie; Robert Henderson; Kamaljit Balaggan; Ananth Viswanathan; Graham E Holder; Andrew Stockman; Nick Tyler; Simon Petersen-Jones; Shomi S Bhattacharya; Adrian J Thrasher; Fred W Fitzke; Barrie J Carter; Gary S Rubin; Anthony T Moore; Robin R Ali
Journal:  N Engl J Med       Date:  2008-04-27       Impact factor: 91.245

6.  The endoplasmic reticulum stress response in aging and age-related diseases.

Authors:  Marishka K Brown; Nirinjini Naidoo
Journal:  Front Physiol       Date:  2012-07-16       Impact factor: 4.566

7.  A lysosome-to-nucleus signalling mechanism senses and regulates the lysosome via mTOR and TFEB.

Authors:  Carmine Settembre; Roberto Zoncu; Diego L Medina; Francesco Vetrini; Serkan Erdin; SerpilUckac Erdin; Tuong Huynh; Mathieu Ferron; Gerard Karsenty; Michel C Vellard; Valeria Facchinetti; David M Sabatini; Andrea Ballabio
Journal:  EMBO J       Date:  2012-02-17       Impact factor: 11.598

8.  Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha-1-anti-trypsin deficiency.

Authors:  Nunzia Pastore; Keith Blomenkamp; Fabio Annunziata; Pasquale Piccolo; Pratibha Mithbaokar; Rosa Maria Sepe; Francesco Vetrini; Donna Palmer; Philip Ng; Elena Polishchuk; Simona Iacobacci; Roman Polishchuk; Jeffrey Teckman; Andrea Ballabio; Nicola Brunetti-Pierri
Journal:  EMBO Mol Med       Date:  2013-02-04       Impact factor: 12.137

9.  Autophagy master regulator TFEB induces clearance of toxic SERPINA1/α-1-antitrypsin polymers.

Authors:  Nunzia Pastore; Andrea Ballabio; Nicola Brunetti-Pierri
Journal:  Autophagy       Date:  2013-04-12       Impact factor: 16.016

Review 10.  Autophagy and heterophagy dysregulation leads to retinal pigment epithelium dysfunction and development of age-related macular degeneration.

Authors:  Kai Kaarniranta; Debasish Sinha; Janusz Blasiak; Anu Kauppinen; Zoltán Veréb; Antero Salminen; Michael E Boulton; Goran Petrovski
Journal:  Autophagy       Date:  2013-04-09       Impact factor: 16.016

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