Approaches to mitochondrial gene therapy

Current Gene Therapy
Gerard G M D'Souza, Volkmar Weissig

Abstract

Since their discovery during the end of the 80's the number of diseases found to be associated with defects in the mitochondrial genome has grown significantly. Organs affected by mutations in mitochondrial DNA (mtDNA) include in decreasing order of vulnerability the brain, skeletal muscle, heart, kidney and liver. Hence neuromuscular and neurodegenerative diseases represent the two largest groups of mtDNA diseases. Despite major advances in understanding mtDNA defects at the genetic and biochemical level, there is however no satisfactory treatment available to the vast majority of patients. This is largely due to the fact that most of these patients have respiratory chain defects, i.e. defects that involve the final common pathway of oxidative metabolism, making it impossible to bypass the defect by administering alternative metabolic carriers of energy. Conventional biochemical treatment having reached an impasse, the exploration of gene therapeutic approaches for patients with mtDNA defects is warranted. For now mitochondrial gene therapy appears to be only theoretical and speculative. Any possibility for gene replacement is dependent on the development of an efficient mitochondrial transfection vector. In this review we des...Continue Reading

Citations

Dec 21, 2006·Pharmaceutical Research·Gerard G M D'SouzaVolkmar Weissig
Mar 11, 2005·Expert Opinion on Biological Therapy·Robert W Taylor
Nov 22, 2005·Expert Opinion on Drug Delivery·Volkmar Weissig
Jan 25, 2011·Journal of Controlled Release : Official Journal of the Controlled Release Society·Young-Wook WonYong-Hee Kim
Feb 2, 2010·Medical Hypotheses·M A Dani, S U Dani
Jul 31, 2007·Journal of Controlled Release : Official Journal of the Controlled Release Society·Richard W HorobinVolkmar Weissig
Aug 25, 2010·Biochimica Et Biophysica Acta·Gerard G M D'SouzaAnee Shah
Jul 16, 2008·Biochimica Et Biophysica Acta·Roza KucharczykJean-Paul di Rago
Jul 13, 2006·Annual Review of Biomedical Engineering·Vladimir P Torchilin
Mar 31, 2005·Rejuvenation Research·Konstantin Khrapko
Dec 21, 2007·Antioxidants & Redox Signaling·Jeffrey S Armstrong
Sep 1, 2006·Acta Neurologica Scandinavica·J Finsterer
Aug 11, 2006·American Journal of Physiology. Cell Physiology·Shaharyar M KhanRussell H Swerdlow
Sep 6, 2006·Journal of Liposome Research·Volkmar WeissigGerard G M D'Souza

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