Repairing mutations in human mitochondria
Posted: March 13, 2012 at 12:11 am
LOS ANGELES Researchers at the UCLA stem cell center and the departments of chemistry and biochemistry and pathology and laboratory medicine have identified, for the first time, a generic way to correct mutations in human mitochondrial DNA by targeting corrective RNAs, a finding with implications for treating a host of mitochondrial diseases.
Mutations in the human mitochondrial genome are implicated in neuromuscular diseases, metabolic defects and aging. There currently are no methods to successfully repair or compensate for these mutations, said study co-senior author Dr. Michael Teitell, a professor of pathology and laboratory medicine and a researcher with the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.
Between 1,000 and 4,000 children per year in the United States are born with a mitochondrial disease and up to one in 4,000 children in the U.S. will develop a mitochondrial disease by the age of 10, according to Mito Action, a nonprofit organization supporting research into mitochondrial diseases. In adults, many diseases of aging have been associated with defects of mitochondrial function, including diabetes, Parkinson's disease, heart disease, stroke, Alzheimer's disease and cancer.
"I think this is a finding that could change the field," Teitell said. "We've been looking to do this for a long time and we had a very reasoned approach, but some key steps were missing. Now we have developed this method and the next step is to show that what we can do in human cell lines with mutant mitochondria can translate into animal models and, ultimately, into humans."
The study appears today in the peer-reviewed journal Proceedings of the National Academy of Sciences.
The current study builds on previous work published in 2010 in the peer-reviewed journal Cell, in which Teitell, Carla Koehler, a professor of chemistry and biochemistry and a Broad stem cell research center scientist, and their team uncovered a role for an essential protein that acts to shuttle RNA into the mitochondria, the energy-producing "power plant" of a cell.
Mitochondria are described as cellular power plants because they generate most of the energy supply within a cell. In addition to supplying energy, mitochondria also are involved in a broad range of other cellular processes including signaling, differentiation, death, control of the cell cycle and growth.
The import of nucleus-encoded small RNAs into mitochondria is essential for the replication, transcription and translation of the mitochondrial genome, but the mechanisms that deliver RNA into mitochondria have remained poorly understood.
The study in Cell outlined a new role for a protein called polynucleotide phosphorylase (PNPASE) in regulating the import of RNA into mitochondria. Reducing the expression or output of PNPASE decreased RNA import, which impaired the processing of mitochondrial genome-encoded RNAs. Reduced RNA processing inhibited the translation of proteins required to maintain the mitochondrial electron transport chain that consumes oxygen during cell respiration to produce energy. With reduced PNPASE, unprocessed mitochondrial-encoded RNAs accumulated, protein translation was inhibited and energy production was compromised, leading to stalled cell growth.
The findings from the current study provide a form of gene therapy for mitochondria by compensating for mutations that cause a wide range of diseases, said study co-senior author Koehler.
Originally posted here:
Repairing mutations in human mitochondria
- Eli and Edythe Broad Center of Regeneration Medicine and ... - October 30th, 2015
- Molecular & Cellular Medicine - October 29th, 2015
- Sickle cell anemia Treatments and drugs - Mayo Clinic - October 29th, 2015
- How are stem cells used in medicine today? - HowStuffWorks - October 29th, 2015
- Oxidative Medicine and Cellular Longevity An Open Access ... - October 29th, 2015
- Department of Regenerative Medicine and Cell Biology - October 29th, 2015
- Sickle Cell Anemia: MedlinePlus - National Library of Medicine - October 29th, 2015
- Personalized RegenerativeMedicine : Dr David Steenblock - October 29th, 2015
- Cell Therapy and Regenerative Medicine - October 29th, 2015
- The Power of Stem Cells | California's Stem Cell Agency - October 29th, 2015
- Herbal medicine | Cancer Research UK - October 29th, 2015
- UR Stem Cell and Regenerative Medicine Institute (SCRMI) - October 29th, 2015
- Penn Medicine News: Genetically Modified "Serial Killer" T ... - October 29th, 2015
- Beta cell regeneration - Center for Regenerative Medicine ... - October 29th, 2015
- The Stem Cell Theory of Cancer - Stanford Medicine Center - October 29th, 2015
- What is a cell? - Genetics Home Reference - October 16th, 2015
- Cell Therapy & Regenerative Medicine - University of Utah ... - October 13th, 2015
- Sickle Cell Anemia: Learn About SCD Symptoms and Treatment - September 26th, 2015
- Research - Stem Cell Biology and Regenerative Medicine ... - September 26th, 2015
- Sickle Cell Disease (Sickle Cell Anemia ... - MedicineNet - September 26th, 2015
- T Cell Therapy (CTL019) | The Children's Hospital of ... - September 15th, 2015
- Knoepfler Lab Stem Cell Blog | Building innovative ... - September 10th, 2015
- Cell culture - Wikipedia, the free encyclopedia - September 6th, 2015
- What are the potential uses of human stem cells and the ... - August 17th, 2015
- One type of airway cell can regenerate another lung cell type - April 13th, 2015
- Limber Lungs: One Type of Airway Cell Can Regenerate Another Lung Cell Type - April 13th, 2015
- Regenestem Network Announces Plans to Attend the 23rd Annual World Congress on Anti-Aging Medicine May 7-9, 2015 - April 6th, 2015
- 'Open' stem cell chromosomes reveal new possibilities for diabetes - April 3rd, 2015
- Key mechanism identified in tumor-cell proliferation in pediatric bone cancers - April 3rd, 2015
- UCI team gets $5 million to create stem cell treatment for Huntington's disease - March 28th, 2015
- Sungduan: Growth factors - March 28th, 2015
- Cleveland Clinic Researchers First to Demonstrate Significant Blocking of Opioid Tolerance With Mesenchymal Stem Cell ... - March 25th, 2015
- Johns Hopkins Medicine, based in Baltimore, Maryland - March 23rd, 2015
- stem cell medicine Jakarta tangerang serpong bsd bintaro - Video - March 21st, 2015
- Global Stem Cells Group to Hold Practical Adipose-Derived Stem Cell Harvesting, Isolation and Re-integration Training ... - March 20th, 2015
- Boston Stem Cell Biotech Start-up Asymmetrex Will Present Essential Technologies for Stem Cell Medical Engineering at ... - March 18th, 2015
- A Single-Cell Breakthrough: newly developed technology dissects properties of single stem cells - March 18th, 2015
- BrainStorm Cell Therapeutics to Present at 3rd Annual Regen Med Investor Day on March 25 in New York - March 18th, 2015
- Global Stem Cells Group to Participate in the 25th Argentine Congress of Aesthetic Medicine in Buenos Aires April 9-10 ... - March 17th, 2015
- Boosting A Natural Protection Against Alzheimer's Disease - March 13th, 2015
- Media portray unrealistic timelines for stem cell therapies - March 12th, 2015
- Stem cells lurking in tumors can resist treatment - March 12th, 2015
- Building custom blood cells to battle sickle cell disease - March 11th, 2015
- Johns Hopkins researchers engineer custom blood cells - March 10th, 2015
- Targazyme Inc. Receives Orphan Drug Designation to TZ101 for Use With Regulatory T Cells to Prevent & Reduce the ... - March 10th, 2015
- Mast cell - Wikipedia, the free encyclopedia - March 9th, 2015
- Achieving gender equality in science, engineering and medicine - March 9th, 2015
- Seven strategies to advance women in science - March 9th, 2015
- British biotech firm sets crowdfunding record with heart drug - March 9th, 2015
- A good night's sleep keeps your stem cells young - March 9th, 2015
- Howe's recovery shows stem-cell advances - March 1st, 2015
- Translational Regenerative Medicine: Market Prospects 2015-2025 - February 28th, 2015
- Stem cellrecruiting hydrogels based on self-assembling peptides for tissue regeneration - February 25th, 2015
- Sertoli cell - Wikipedia, the free encyclopedia - February 17th, 2015
- Global Stem Cells Group Announces Alliance with Regenerative Technology - February 11th, 2015
- Biotech firm Cell Therapy claims crowdfunding record with heart drug - February 10th, 2015
- Two UC San Diego Scientists Receive Stem Cell Technology Grants - February 3rd, 2015
- Regenestem Network and Gilberto Hernandez Falcon, M.D. Open Stem Cell Clinic in Yucatan - January 31st, 2015
- Global Stem Cells Group and Regenestem Network Announce Launch of New Stem Cell and Regenerative Medicine Clinic in ... - January 29th, 2015
- Asymmetrex Scheduled to Present Unique Perspectives in Stem Cell Biology and Recent Advances in Technologies for Adult ... - January 29th, 2015
- Gordie Howe's stem cell therapy raises concerns among medical experts - January 29th, 2015
- Gordie Howe's stem cell therapy raises concerns among experts - January 29th, 2015
- BioTime Announces Issuance of 14 New Patents in the Fields of Regenerative Medicine, Stem Cell Technology, and Cancer ... - January 27th, 2015
- Keeping the Kraken asleep - January 27th, 2015
- Helmholtz International Fellow Award for Prof. Amanda Fisher from London - January 27th, 2015
- Regenestem Network, a division of Global Stem Cells Group, Announces Launch of New Stem Cells and Regenerative ... - January 22nd, 2015
- Scientists announce revolutionary culturing technique for liver and pancreas - January 22nd, 2015
- New Type of Cell Found to Repair Lung Injury in Mice - January 18th, 2015
- Stem cells derived from amniotic tissues have immunosuppressive properties - January 16th, 2015
- Global Stem Cells Group to Move Headquarters to Larger Miami Lakes Office Complex - January 14th, 2015
- Peter S. Kim Named the Virginia and D.K. Ludwig Professor of Biochemistry at Stanford - January 13th, 2015
- Renowned professor's book addresses stem cell biology & regenerative medicine - January 13th, 2015
- Circadian rhythms regulate skin stem cell metabolism and expansion, study finds - January 7th, 2015
- Cord Blood Banking Leader, Cryo-Cell International, Continues to Support the Advancement of Regenerative Medicine - January 6th, 2015
- Scientists Develop Pioneering Method to Define Stages of Stem Cell Reprogramming - January 6th, 2015
- Pioneering method developed to define stages of stem cell reprogramming - January 6th, 2015
- The Irvine Stem Cell Treatment Center Announces Adult Stem Cell Public Seminars in Orange County, California - January 5th, 2015
- Ten years in, California's stem cell program is getting a reboot - January 4th, 2015
- Two-thirds of cancer cases are "bad luck," study says - January 1st, 2015
- 'Bad Luck' of Random Mutations Plays Predominant Role in Cancer, Study Shows - January 1st, 2015