(Press-News.org) The seeds of greenhouse-grown corn could hold the key to treating a rare, life-threatening childhood genetic disease, according to researchers from Simon Fraser University.
SFU biologist Allison Kermode and her team have been carrying out multidisciplinary research toward developing enzyme therapeutics for lysosomal storage diseases - rare, but devastating childhood genetic diseases – for more than a decade.
In the most severe forms of these inherited diseases, untreated patients die in early childhood because of progressive damage to all organs of the body.
Currently, enzyme treatments are available for only six of the more than 70 diverse types of lysosomal storage diseases.
"In part because mammalian cell cultures have been the system of choice to produce these therapeutics, the enzymes are extremely costly to make, with treatments typically ranging from $300,000 to $500,000 per year for children, with even higher costs for adults," says Kermode, noting the strain on healthcare budgets in Canada and other countries is becoming an issue.
Greenhouse-grown maize may become a platform for making alpha-L-iduronidase, an enzyme used to treat the lysosomal storage disease known as mucopolysaccharidosis I, according to research published in this week's Nature Communications.
The findings could ultimately change how these enzyme therapeutics are made, and substantially reduce the costs of treating patients. The novel technology manipulates processes inside the maize seed that "traffick" messenger RNAs to certain parts of the cell as a means of controlling the subsequent sugar processing of the therapeutic protein.
In this way, the researchers have been able to produce the enzyme drug in maize seeds. The product could ultimately be used as a disease therapeutic, although it is still "early days," says Kermode, and several research goals remain to be accomplished before this can become a reality.
Kermode says the success of the work underscores the power of multidisciplinary research that included contributions from SFU chemistry professor David Vocadlo, and from UBC Medical Genetics professor Lorne Clarke. It further underscores the importance of connections between SFU and Australia's Griffith University, through collaborative researchers Mark von Itzstein and Thomas Haselhorst.
"In 2005, we had the basis of our story worked out," says Kermode. "Taking it to the next level involved their precise analyses to determine the sugar residues on the therapeutic enzyme produced by the modified maize seeds.
"When we first looked at the sugar analysis data we were amazed at how well the 'mRNA-trafficking strategy' had worked, and the high fidelity of the process for controlling the sugar-processing of the therapeutic protein. This is critical as sugar processing influences the characteristics of a protein (enzyme) therapeutic, including its safety, quality, half-life in the bloodstream, and efficacy. The work could well extend to forming a platform for the production of other protein therapeutics."
Kermode also credits SFU research associate Xu He, the first author of the Nature Communications paper. Her funding sources included NSERC Strategic grants and a Michael Smith Foundation for Health Research Senior Scholar Award, and in related research, a Canadian Society for Mucopolysaccharide and Related Diseases grant.
###
Simon Fraser University is Canada's top-ranked comprehensive university and one of the top 50 universities in the world under 50 years old. With campuses in Vancouver, Burnaby and Surrey, B.C., SFU engages actively with the community in its research and teaching, delivers almost 150 programs to more than 30,000 students, and has more than 120,000 alumni in 130 countries.
Simon Fraser University
Public Affairs/Media Relations (PAMR)
778.782.3210 www.sfu.ca/pamr
Contact:
Allison Kermode, 778.782.3982; kermode@sfu.ca (note: limited availability today)
Marianne_Meadahl, PAMR, 778.782.9017; Marianne_Meadahl@sfu.cav
Simon Fraser University: Engaging Students. Engaging Research. Engaging Communities.
Growing corn to treat rare disease
2012-09-21
ELSE PRESS RELEASES FROM THIS DATE:
LifeShield Builds Web Presence for Regional Authorized Dealers
2012-09-21
LifeShield Security authorized dealers throughout the country can now connect with consumers on a regional level through locally relevant websites that help to identify customers and prospects. Sites also offer special wireless home security deals to each region that are constantly changing.
"Local dealers understand the security challenges particular to their own regions and therefore can be more successful at recruiting new customers," said Shannon Dominello, CMO, LifeShield. "Regional sites provide a legitimate local web presence for each dealer that ...
Fear can be erased from the brain
2012-09-21
Newly formed emotional memories can be erased from the human brain. This is shown by researchers from Uppsala University in a new study now being published by the academic journal Science. The findings may represent a breakthrough in research on memory and fear.
Thomas Ågren, a doctoral candidate at the Department of Psychology under the supervision of Professors Mats Fredrikson and Tomas Furmark, has shown, that it is possible to erase newly formed emotional memories from the human brain.
When a person learns something, a lasting long-term memory is created with the ...
Move to less impoverished neighborhoods boosts physical and mental health
2012-09-21
Moving from a high-poverty to lower-poverty neighborhood spurs long-term gains in the physical and mental health of low-income adults, as well as a substantial increase in their happiness, despite not improving economic self-sufficiency, according to a new study published in the Sept. 20 issue of Science by researchers at the University of Chicago and partners at other institutions.
Although moving into less disadvantaged neighborhoods did not raise incomes for the families that moved, these families experienced important gains in well-being in other ways. Moving from ...
A mother’s nutrition--before pregnancy--may alter the function of her children’s genes
2012-09-21
Bethesda, MD—Everyone knows that what mom eats when pregnant makes a huge difference in the health of her child. Now, new research in mice suggests that what she ate before pregnancy might be important too. According to a new research report published online in The FASEB Journal, what a group of female mice ate—before pregnancy—chemically altered their DNA and these changes were passed to her offspring. These DNA alterations, called "epigenetic" changes, drastically affected the pups' metabolism of many essential fatty acids. These results could have a profound impact on ...
Treating disease by the numbers
2012-09-21
Mathematical modeling being tested by researchers at the School of Science at Indiana University-Purdue University Indianapolis (IUPUI) and the IU School of Medicine has the potential to impact the knowledge and treatment of several diseases that continue to challenge scientists across the world.
The National Science Foundation recently recognized the work led by Drs. Giovanna Guidoboni, associate professor of mathematics in the School of Science, and Alon Harris, professor of ophthalmology and director of clinical research at the Eugene and Marilyn Glick Eye Institute, ...
ORNL research uncovers path to defect-free thin films
2012-09-21
A team led by Oak Ridge National Laboratory's Ho Nyung Lee has discovered a strain relaxation phenomenon in cobaltites that has eluded researchers for decades and may lead to advances in fuel cells, magnetic sensors and a host of energy-related materials.
The finding, published in Nano Letters, could change the conventional wisdom that accommodating the strain inherent during the formation of epitaxial thin films necessarily involves structural defects, said Lee, a member of the Department of Energy lab's Materials Science and Technology Division. Instead, the researchers ...
Once usability becomes secure
2012-09-21
Risk increases with comfort: "Single Sign-On" permits users to access all their protected Web resources, replacing repeated sign-ins with passwords. However, attackers also know about the advantages such a single point of attack offers to them. Andreas Mayer, who is writing his PhD thesis as an external doctoral candidate at the Chair for Network and Data Security (Prof. Dr. Jörg Schwenk) at Ruhr-Universität Bochum, has now been able to significantly increase the security of this central interface for the simpleSAMLphp framework.
In the past, no protection against targeted ...
As painkiller overdoses mount, researchers outline effective approaches to curb epidemic
2012-09-21
WASHINGTON—Prescription painkillers are responsible for more fatal overdoses in the United States than heroin and cocaine combined. And while most states have programs to curb abuse and addiction, a new report from Brandeis University shows that many states do not fully analyze the data they collect.
Experts from the Prescription Drug Monitoring Program Center of Excellence at Brandeis University's Heller School for Social Policy and Management systematically assessed prescription drug monitoring programs and found a patchwork of strategies and standards. Their report ...
Walking to the beat could help patients with Parkinson's disease
2012-09-21
Walking to a beat could be useful for patients needing rehabilitation, according to a University of Pittsburgh study. The findings, highlighted in the August issue of PLOS One, demonstrate that researchers should further investigate the potential of auditory, visual, and tactile cues in the rehabilitation of patients suffering from illnesses like Parkinson's Disease—a brain disorder leading to shaking (tremors) and difficulty walking.
Together with a team of collaborators from abroad, Ervin Sejdic, an assistant professor of engineering in Pitt's Swanson School of Engineering, ...
Business plan competitions may be key to job growth
2012-09-21
A new study of high-tech startups that participated in the Rice Business Plan Competition (RBPC) shows that these entrepreneurs have a much higher rate of success than typical new ventures and are therefore more likely to contribute to job growth.
The study by the Rice Alliance for Technology and Entrepreneurship spans the 11-year life of the RBPC, the world's richest and largest business plan competition, which comprises teams of graduate students from throughout the world. The comprehensive and longitudinal study offers insights into the experiential factors that can ...