Parkinsons disease researchers have used gene-editing tools to introduce the disorders most common genetic mutation into marmoset monkey stem cells and to successfully tamp down cellular chemistry that often goes awry in Parkinsons patients.
The edited cells are a step toward studying the degenerative neurological disorder in a primate model, which has proven elusive. Parkinsons, which affects more than 10 million people worldwide, progressively degrades the nervous system, causing characteristic tremors, dangerous loss of muscle control, cardiac and gastrointestinal dysfunction and other issues.
Marina Emborg
We know now how to insert a single mutation, a point mutation, into the marmoset stem cell, says Marina Emborg, professor of medical physics and leader of University of WisconsinMadison scientists who published their findings Feb. 26 in the journal Scientific Reports. This is an exquisite model of Parkinsons. For testing therapies, this is the perfect platform.
The researchers used a version of the gene-editing technology CRISPR to change a single nucleotide one molecule among more than 2.8 billion pairs of them found in a common marmosets DNA in the cells genetic code and give them a mutation called G2019S.
In human Parkinsons patients, the mutation causes abnormal over-activity of an enzyme, a kinase called LRRK2, involved in a cells metabolism. Other gene-editing studies have employed methods in which the cells produced both normal and mutated enzymes at the same time. The new study is the first to result in cells that make only enzymes with the G2019S mutation, which makes it easier to study what role this mutation plays in the disease.
The metabolism inside our stem cells with the mutation was not as efficient as a normal cell, just as we see in Parkinsons, says Emborg, whose work is supported by the National Institutes of Health. Our cells had a shorter life in a dish. And when they were exposed to oxidative stress, they were less resilient to that.
The mutated cells shared another shortcoming of Parkinsons: lackluster connections to other cells. Stem cells are an especially powerful research tool because they can develop into many different types of cells found throughout the body. When the researchers spurred their mutated stem cells to differentiate into neurons, they developed fewer branches to connect and communicate with neighboring neurons.
We can see the impact of these mutations on the cells in the dish, and that gives us a glimpse of what we could see if we used the same genetic principles to introduce the mutation into a marmoset, says Jenna Kropp Schmidt, a Wisconsin National Primate Research Center scientist and co-author of the study. A precisely genetically-modified monkey would allow us to monitor disease progression and test new therapeutics to affect the course of the disease.
The concept has applications in research beyond Parkinsons.
We can use some of the same genetic techniques and apply it to create other primate models of human diseases, Schmidt says.
The researchers also used marmoset stem cells to test a genetic treatment for Parkinsons. They shortened part of a gene to block LRRK2 production, which made positive changes in cellular metabolism.
We found no differences in viability between the cells with the truncated kinase and normal cells, which is a big thing. And when we made neurons from these cells, we actually found an increased number of branches, Emborg says. This kinase gene target is a good candidate to explore as a potential Parkinsons therapy.
This research was supported by grants from the National Institutes of Health (R24OD019803, P51OD011106 and UL1TR000427).
Share via Facebook
Share via Twitter
Share via Linked In
Share via Email
See the original post here:
Cells carrying Parkinson's mutation could lead to new model for studying disease - University of Wisconsin-Madison
- Embryonic Stem Cell Fact Sheet - University of WisconsinMadison - October 21st, 2022
- The longevity diet: Lots of beans and periodic fasts slow ageing - The New Daily - September 16th, 2022
- See-through zebrafish, new imaging method put blood stem cells in high-resolution spotlight - University of Wisconsin-Madison - August 14th, 2022
- ISCT's New Leader on the Future of Cell and Gene Therapy - The Medicine Maker - August 14th, 2022
- Erik Ranheim to become chair of Department of Pathology and Laboratory Medicine - University of Wisconsin School of Medicine and Public Health - August 5th, 2022
- It's a Holland Hat Trick: College of Health Professor Gets Three Grant Notifications in One Day - University of Utah Health Sciences - August 5th, 2022
- How long-term Covid-19 immunity paves the way for universal Covid-19 vaccines - Vox.com - August 5th, 2022
- Delayed cord blood clamping: a health boost for babies, and potentially for others - La Crosse Tribune - July 19th, 2022
- Neural stem cells: developmental mechanisms and disease modeling - June 13th, 2022
- The benefits and risks of stem cell technology - PMC - June 13th, 2022
- Hunters, Hearing Loss, and New Tech to Fix it - MeatEater - June 13th, 2022
- Cryopreservation of Hematopoietic Stem Cells - January 20th, 2022
- Post from Community: STEM Forward to honor STEM outreach and engineering excellence at the 69th annual celebration of STEM virtual event - Milwaukee... - January 20th, 2022
- Bring in the bulldozers: Cynatas CEO on how heavyweight FujiFilm will turbocharge its ... - Stockhead - January 5th, 2022
- Polymyositis Pipeline to Progress with New and Emerging Drugs for Treatment, Analyzes DelveInsight - GlobeNewswire - December 10th, 2021
- FDA Approval of Jakafi for Chronic GVHD Provides Another Option, But 'More Work Needs to Be Done' - Curetoday.com - October 5th, 2021
- Researchers May Have Found The Gene Responsible For Cellular Aging - Anti Aging News - December 4th, 2020
- Osteoporosis treatments could be on the way after scientists identify aging gene - iNews - December 4th, 2020
- Stem Cell Assay Market In-Depth Analysis and Forecast 2017-2025 - Khabar South Asia - December 4th, 2020
- Stem Cell Assay Market In-Depth Analysis and Forecast 2017-2025 - Royal Sutton News - October 29th, 2020
- Stem Cell Assay Market to Witness Growth Acceleration During 2017-2025 - Cole of Duty - June 4th, 2020
- Clinical prospects for stem cells begin to emerge - April 28th, 2020
- Answering your sweet onion question and the science of why onions make you cry - Green Bay Press Gazette - April 28th, 2020
- New technique developed to treat hardening of internal organs - WNDU-TV - March 4th, 2020
- Scientists Grapple with US Restrictions on Fetal Tissue Research - The Scientist - March 4th, 2020
- Deer hunter or Bambi lover: What you should know about chronic wasting disease - Ohio's Country Journal and Ohio Ag Net - February 14th, 2020
- Cord Tissue Cell Therapy | Wisconsin Stem Cell Therapy - January 29th, 2020
- The selling of CTE: How the 'Concussion' doctor has built a career on distorted science - Stars and Stripes - January 24th, 2020
- nation briefs - Daily Herald - January 5th, 2020
- Addressing Disparities in Cancer: Factors Influencing Care, Access and Outcomes - OncoZine - December 11th, 2019
- Ausman family thankful for recoveries, support from family and community - Chippewa Herald - November 28th, 2019
- Lab: Nightmares may help the brain prepare for frightening situations in real life - Metro Newspaper UK - November 28th, 2019
- Stem Cell Assay Market Demand with Leading Key Players and New Investment Opportunities Emerge To Augment Segments in Sector By 2025 - The Denton... - November 28th, 2019
- Development of the dopaminergic system - from stem cells ... - April 15th, 2019
- Stem Cell Therapy for Back Conditions Oconomowoc, WI - January 26th, 2019
- Wisconsin Stem Cell Clinic - Forward Healthy Lifestyles - December 31st, 2018
- Knee Conditions Milwaukee, WI | Wisconsin Stem Cell Therapy - November 1st, 2018
- Stem Cells for Hip Brookfield, WI | Wisconsin Stem Cell ... - October 26th, 2018
- Geron Official Affirms iPS Cells Are the Future of Stem ... - October 13th, 2018
- Laws Governing Stem Cell Research Wisconsin Right to Life - June 23rd, 2018
- Knee Conditions | Milwaukee, WI | Wisconsin Stem Cell - October 16th, 2017
- Sponsors World Stem Cell Summit - October 15th, 2017
- Stem Cell Therapy - Cendant Cellular Therapies - Denver ... - October 15th, 2017
- Madison man is first Hmong-American to get an MD-Ph.D. - Post-Bulletin - July 10th, 2017
- Stem cells | | News | UW-Madison - November 20th, 2016
- Public Outreach | Stem Cell and Regenerative Medicine Center - November 20th, 2016
- Chapter 34: Stem Cells - The Hastings Center - September 28th, 2016
- Wisconsin Stem Cell Therapy | Stem Cell Treatments - October 10th, 2015
- 1. Embryonic Stem Cells [Stem Cell Information] - October 1st, 2015
- WISCONSIN STEM CELL SYMPOSIUM - BTC Institute - October 1st, 2015
- How Stem Cells Work - HowStuffWorks - October 1st, 2015
- Variations in cell programs control cancer and normal stem ... - September 8th, 2015
- Local students take part in stem cell camp | Local | apg ... - August 1st, 2015
- Wisconsin Roundup: VC Funding, StartUp Health, Stem Cells ... - July 2nd, 2015
- Stem cell expert: Bart Starr treatment shows need for ... - June 29th, 2015
- Study Reveals How Imaging Technology Can Track Stem Cell ... - June 11th, 2015
- Wisconsin Stem Cell Now Issues and Legislation - June 11th, 2015
- UW-Madison Heart Stem Cell Study Among American Heart ... - May 28th, 2015
- Whitehead Institute - News - 2008 - Human blood stem cells ... - May 1st, 2015
- Science meets art: 2015 Cool Science Images unveiled - April 11th, 2015
- Kentucky Fan Gets Life-Saving Stem Cell Donation From Univ. of Wisconsin Student - April 3rd, 2015
- Blood ties: Ky. basketball fan gets Wisconsin assist - April 2nd, 2015
- Wisconsin Stem Cell Roundtable (WiSCR) - Welcome to Stem ... - March 13th, 2015
- New insights into 3-D genome organization and genetic variability - March 10th, 2015
- UW Stem Cell Researchers Building a 'Retinal Patch' to ... - February 27th, 2015
- New Insights into 3D Genome Organization and Genetic Variability - February 18th, 2015
- First contracting human muscle grown in laboratory - January 14th, 2015
- Eye Researchers Awarded Grant for Stem-Cell Research in ... - January 3rd, 2015
- UW-Madison Stem Cell & Regenerative Medicine Center - December 27th, 2014
- Baby cells learn to communicate using the lsd1 gene - December 16th, 2014
- Advances in lymphoma and multiple myeloma treatment seek to improve outcomes for patients - December 9th, 2014
- Research to Prevent Blindness, Inc. and the International Retinal Research Foundation Announce Catalyst Awards for AMD ... - December 4th, 2014
- James A. Thomson - Wisconsin Stem Cell Research Program - December 1st, 2014
- Programs - Cell and Regenerative Biology, University of ... - November 18th, 2014
- Morgridge Scientists Find Way to 'Keep the Lights on' for Cell Self-Renewal - November 13th, 2014
- BioEden the specialist tooth stem cell bank announce ... - November 12th, 2014
- Survival rates in pediatric umbilical cord transplants may indicate a new standard of care - October 30th, 2014
- UW-Madison senior Maria Estevez discusses women pursuing STEM fields - October 27th, 2014
- Cashton man goes from winning state award to battling cancer - October 12th, 2014
- Career Opportunities | Stem Cell and Regenerative Medicine ... - September 26th, 2014