Navigation Links
Mouse study shows gene therapy may be possible cure for Hurler syndrome
Date:2/4/2014

al bone growth, heart valve problems, joint disease, intellectual disabilities and death.

Enzyme replacement therapy can be used to treat the disease, but it is only temporary and not curative. Bone marrow transplant using hematopoietic stem cells also has been tested on some patients with mixed results. The transplant procedure can carry severe risks and does not always work.

Pan and her colleagues including Roscoe O. Brady, MD, a researcher at NINDS report that using platelets and megakaryocytes for gene therapy is effective and could reduce the risk of activating cancer-causing oncogenes in hematopoietic stem cells.

The authors said tests showed that human megakaryocytic cells were capable of overexpressing IDUA, revealing their capacity for potential therapeutic benefit. While engineering megakaryocytes and platelets for infusion into their mouse models of Hurler, the scientists report they were able to release IDUA directly into amply sized extracellular spaces or inside micro-particles as the cells matured or activated. The cells were able to produce and package large amounts of functional IDUA and retained the capacity to cross-correct patient cells.

After infusing mouse models of Hurler with the genetically modified cells, researchers said this led to long-term normalization of IDUA levels in the animal's blood with versatile delivery routes and on-target preferential distribution to the liver and spleen. The treatment led to a complete metabolic correction of MPS I in most peripheral organs of the mice.

Researchers cautioned that study results involving laboratory mouse models do not always translate into clinical treatment of human patients, and that additional research is needed for this prospective therapy.


'/>"/>

Contact: Nick Miller
nicholas.miller@cchmc.org
513-803-6035
Cincinnati Children's Hospital Medical Center
Source:Eurekalert

Page: 1 2

Related biology news :

1. Dietary treatment shows potential in a mouse model of Alzheimers disease
2. The mouse that RORed
3. H. pylori vaccine shows promise in mouse studies
4. New book on mouse models of cancer from Cold Spring Harbor Laboratory Press
5. Mouse studies reveal promising vitamin D-based treatment for MS
6. Elevated levels of copper in amyloid plaques associated with neurodegeneration in mouse models of AD
7. New agent inhibits HCV replication in mouse models -- No resistance seen
8. Cat and mouse: A single gene matters
9. New mouse viruses could aid hepatitis research
10. Omega-3s from fish vs. fish oil pills better at maintaining blood pressure in mouse model
11. UT Southwestern scientists make mouse model of human cancer, demonstrate cure
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:12/11/2014)... , Dec. 09, 2014 Research and Markets ... ( http://www.researchandmarkets.com/research/twhjll/biometrics_market ) has announced ... the APAC Region 2015-2019"  report to their offering. ... this market is advances in technology. With continuous ... biometric solutions to the latest standard that meets ...
(Date:12/10/2014)... -- You,ve been here before: you desperately need to sign ... site key or the answer to your secret question. What,s ... Today, Hoyos Labs , a digital infrastructure ... put an end to the frustration that comes with usernames, ... leverages a user,s smartphone to acquire his or her biometrics ...
(Date:12/10/2014)... , Dec. 08, 2014 Research and Markets ... addition of the "Biometrics Market in Japan ... http://photos.prnewswire.com/prnh/20130307/600769 The ... such as rural banking and upgradation of the ... witnessed in the market. Besides the aforementioned projects, ...
Breaking Biology News(10 mins):Biometrics Market in the APAC Region 2015-2019: Key Vendors are 3M Cogent, NEC, Safran and Suprema 2The Password is Finally Dead: Launch of 1U Mobile App Eliminates Need for All Usernames and Passwords 2The Password is Finally Dead: Launch of 1U Mobile App Eliminates Need for All Usernames and Passwords 3Biometrics Market in Japan 2014-2018: Key Vendors are DDS, Fujitsu, Hitachi and NEC 2
... bodies are replaced through cell division. Researchers at Karolinska ... how genetic information remains intact despite this continuous exchange ... issue of the scientific journal Molecular Cell. , An ... of which die and are replaced by cell division ...
... down to two basic missions: eat, and avoid being eaten. ... faster it grows big enough to transform into a frog; ... of becoming someone else's dinner. , Scientists have known ... risk, but new research by a University of Michigan graduate ...
... a step toward personalized medicine, Howard Hughes Medical Institute ... new technique to identify previously unknown genetic mutations that ... of the genes ?inside acute myeloid leukemia (AML) cells, ... to zero in on molecular abnormalities that might be ...
Cached Biology News:A protein complex that untangles DNA 2Time of day tempers tadpoles' response to predators 2New strategy rapidly identifies cancer targets 2New strategy rapidly identifies cancer targets 3
(Date:12/19/2014)... Boston, MA (PRWEB) December 18, 2014 ... software, announced the appointment of Siri Bryant as the ... the new Director of Core Implementations. These two new ... provide outstanding customer service by ensuring that iLab continues ... , “In the last four years, iLab has been ...
(Date:12/19/2014)... The empty capsules market is experiencing growth ... technological innovations in the empty capsules market. The ... growth of the empty capsules market. , Get ... of Report , The report will enrich both ... to gauge the pulse of the market which ...
(Date:12/19/2014)... , Dec. 18, 2014  Decision Resources Group finds ... biomaterial market will grow moderately through 2023 as the ... growing awareness of dental biomaterials all spur procedure volumes. ... as China and ... dental procedures within reach for a larger proportion of ...
(Date:12/19/2014)... 18, 2014   ISN has released its ... Indicators publication series. With data aggregated from ... in 2013 within its online contractor management platform, ISNetworld, ... performance indicator (KPI) statistics. The ... publication is available in two industry specific volumes: ...
Breaking Biology Technology:iLab Solutions Announces Two New Leadership Positions to Meet the Needs of Its Growing Customer Base 2Empty Capsules Market Hit to Reach $1,820 Million by 2019 - New Research Report by MarketsandMarkets 2Empty Capsules Market Hit to Reach $1,820 Million by 2019 - New Research Report by MarketsandMarkets 3Empty Capsules Market Hit to Reach $1,820 Million by 2019 - New Research Report by MarketsandMarkets 4Asia Pacific Dental Biomaterial Market Will Grow Strongly Through 2023 2Asia Pacific Dental Biomaterial Market Will Grow Strongly Through 2023 3ISN Releases Third Edition of US Contractor Health & Safety Performance Indicators Publication Series 2ISN Releases Third Edition of US Contractor Health & Safety Performance Indicators Publication Series 3
... Inc . of Marlborough, MA was ... 5,000 fastest-growing private companies in the country for the ... ranked #2626 after being ranked #575 in 2008 and #2116 ... the most important segment of the economy – America,s independent-minded ...
... ThermoGenesis Corp. (Nasdaq: KOOLD ), a leading ... adult stem cells, said today it has sold two BioArchive® ... which operates cord blood banks in Taiwan, Hong Kong, Thailand ... and a storage capacity for cryopreserving and archiving cord blood ...
... Aug. 30 Dr. Kyong S. Choe, of Kwangju, South ... Edition for his outstanding contributions and achievements in the field ... CHOE Dr. Choe is currently practicing ... where he practices periodontics and prosthodontics targeting implants and full ...
Cached Biology Technology:21st Century Biochemicals Marks Its Third Consecutive Year on the Inc. 5000 2ThermoGenesis Announces Sale of BioArchive Systems In Asia as Company Increases Presence In Key Market 2ThermoGenesis Announces Sale of BioArchive Systems In Asia as Company Increases Presence In Key Market 3Dr. Kyong S. Choe Recognized by Strathmore's Who's Who Worldwide Publication 2
...
...
...
... Sartolab P20 plus,for Reliable Sterile Filtration of ... to a,membrane pump or tube pump, quickly ... of media,and aqueous solutions. The combination ... an automatic de-aeration,ensures high flow rates and ...
Biology Products: