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New genomic editing methods produce better disease models from patient-derived iPSCs

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PUBLIC RELEASE DATE:

8-Sep-2014

Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, September 8, 2014Highly valuable for modeling human diseases and discovering novel drugs and cell-based therapies, induced pluripotent stem cells (iPSCs) are created by reprogramming an adult cell from a patient to obtain patient-specific stem cells. Due to genetic variation, however, iPSCs may differ from a patient's diseased cells, and researchers are now applying new and emerging genomic editing tools to human disease modeling, as described in a comprehensive Review article published in Stem Cells and Development, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Stem Cells and Development website until September 30, 2014.

In "Genomic Editing Tools to Model Human Diseases with Isogenic Pluripotent Stem Cells," Ihor Lemischka, Huen Suk Kim, Jeffrey Bernitz, and Dung-Fang Lee, Icahn School of Medicine at Mount Sinai (New York, NY), provide a detailed overview of the development of patient-specific iPSCs for modeling a disease. The authors describe the many factors that need to be considered when generating an iPSC-based disease model comprised of cells that are genetically identical, and they discuss the advantages and limitations of the three leading genomic editing tools: zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and the most recent, the clustered regularly interspaced short palindromic repeat (CRISPR) system.

"As our appreciation of iPSCs as primarily therapeutic screens and disease models matures, we look to advanced gene editing tools to assist in appropriate experimental design. Ihor Lemischka and colleagues provide a much needed examination of the advantages and shortcomings of such techniques," says Editor-in-Chief Graham C. Parker, PhD, The Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine, Detroit, MI.

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About the Journal

Stem Cells and Development is an authoritative peer-reviewed journal published 24 times per year in print and online. The Journal is dedicated to communication and objective analysis of developments in the biology, characteristics, and therapeutic utility of stem cells, especially those of the hematopoietic system. Complete tables of content and a free sample issue may be viewed on the Stem Cells and Development website.

About the Publisher

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New genomic editing methods produce better disease models from patient-derived iPSCs

Editing DNA could be genetic medicine breakthrough – SFGate

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A new way to make powerful changes at will to the DNA of humans, other animals and plants, much like how a writer changes words in a story, could usher in a transformation in genetic medicine.

Scientists are not just excited about this recently discovered technique because it can snip and edit DNA with precision. It can also do the job more easily and cheaply than other gene-editing methods, making possible research that has historically been difficult, experts say.

Now some of the biologists who unlocked this tool, derived from the immune system of bacteria, are forming companies around it. Although this molecular system, known as Crispr, is not fully understood, researchers believe it can be harnessed to create therapies for intractable genetic diseases.

One of those scientists, UC Berkeley Professor Jennifer Doudna, was part of the team that in 2012 first demonstrated the technique. It is now employed by two companies she has co-founded: Caribou Biosciences in Berkeley, and Editas Medicine in Cambridge, Mass. The latter started last year with $43 million in venture capital. Another company, the aptly named Crispr Therapeutics in Switzerland, has $25 million in the bank, and other biotechnology companies are experimenting with the procedure.

"In principle, this is a technology that could enable correction of genetic mutations that would otherwise lead to disease," said Doudna, a professor of chemistry and biochemistry and molecular biology, in a telephone interview. She was among several experts who spoke at a UC Berkeley conference on the subject last month.

But because the method is in its infancy and has little precedent with the agencies that regulate medicines, it will almost certainly be a long time before a Crispr-based therapy makes it to market.

Its potential risks also concern some bioethicists.

"In the very worst case, technologies that can cause permanent inheritable changes in people bring you very close to the risk of modern eugenics," said Pete Shanks, a consultant who blogs about the topic for the Center for Genetics and Society, a bioethics watchdog organization in Berkeley. "Pretty much everyone agrees that we should avoid that. How we do that, comes the question."

The technique operates on the recent discovery that bacteria, like humans, have an immune system that remembers viruses that have attacked before. To protect themselves, bacteria chop up and incorporate short fragments of foreign invaders' genetic code so they know to destroy a virus should it strike again. It is their equivalent of developing vaccines.

Those new fragments in the bacterial genome add up to an "unusual structure," first reported in the late 1980s by scientists who called them "clustered regularly interspaced short palindromic repeats" - Crispr for short. But Crispr's role, to fight infections, wasn't confirmed until 2007.

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Editing DNA could be genetic medicine breakthrough - SFGate

Want to know more about marijuana? Science cafes (yes, plural) to the rescue

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I bet youve got opinions about marijuana; everybody does.

I bet youve got opinions about marijuana; everybody does.

But I also bet you dont have much knowledge about it; few of us do.

How addictive is dope? Does it have health benefits and if so, which ones, and in what context? How about health drawbacks, physical or mental?

I dont know and neither do you, and no wonder. The effects of bogarting that joint (to use terminology from my youth) can be tough to pin down; thats true even for a man who has done at least as much of that pinning-down as anybody in New Hampshire.

When youre talking about smoked marijuana, its not easy to do good research. Its tough to interpret. Its tough to run the studies. Its tough to have a placebo. Its tough to get people in your studies that arent already smoking marijuana. Its tough to try to dose it, said Alan Budney, a professor at the Geisel School of Medicine at Dartmouth College, who has written lots of papers about cannabis use disorders in adults and adolescents.

But New Hampshire is ramping up to allow medical marijuana, so it behooves us as residents of the state to know more.

Happily, Budneys going to help us learn.

He is one of two panelists who will answer all your questions about the science of marijuana at Science Cafe New Hampshire in Nashua, a week from Wednesday, Sept. 17. Stacy Gruber, director of the Cognitive and Clinical Neuroimaging Core at McLean Hospitals Brain Imaging Center in Cambridge, will also be there; her lab has done a slew of research into the results of substance use and abuse, especially cannabis.

As always, I will moderate the free, two-hour session, which starts at 6 p.m. at Killarneys Irish Pub.

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Want to know more about marijuana? Science cafes (yes, plural) to the rescue

Concordia Chemistry and Biochemistry Ice Bucket Challenge – Video

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Concordia Chemistry and Biochemistry Ice Bucket Challenge
Concordia #39;s departments of Chemistry and Biochemistry accept the ALS Association Ice Bucket Challenge and nominate all departments in the Faculty of Arts and Science as well as Richard J. Renaud.

By: Concordia University - Sir George Williams Campus

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Concordia Chemistry and Biochemistry Ice Bucket Challenge - Video

Mesa College lands $3.7 million

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San Diego Mesa College has received more than $3.7 million in grants to support underrepresented students wanting to pursue careers in biomedical or behavioral science as well helping improve the academic success of low-income Hispanic students.

The college was awarded $2.62 million from the U.S. Department of Education to improve retention and academic success for low-income Hispanic students under a five-year Title V grant. Mesa was one of about 20 colleges and universities selected for the funding through a competitive process.

Mesa Colleges grant proposal, called Proyecto Exito or Project Success, calls for the college to offer more tutoring, mentoring, summer programs and career development. Officials say they will be redesigning courses, strengthening student support and expanding training for faculty and staff.

This is the first Title V grant received by Mesa College. About 32 percent of the colleges fall enrollment was of Hispanic or Latino descent, officials said.

Mesa is thrilled to receive this grant, which will enable the college to create the conditions that lead to higher student retention and completion for our Latino students, college President Pamela Luster said in a statement. Our student population mirrors that of the greater San Diego area, and as such our commitment to our growing population of Latino students is critical. Their success is our success, and as educated citizens our students contribute to greater economic advances for our community and the region.

Julianna Barnes, vice president of student services, said in a statement that the grant will strengthen the college's institutional capacity to better support Latino students and will level the odds for success for all students.

Mesa also recently learned it would be getting nearly $1.1 million over five years from the National Institutes of Health to extend a program that helps underrepresented groups studying biomedical or behavioral sciences to pursue careers in research.

The Bridges to the Baccalaureate Program has been in place since 2005 and is the result of a partnership between Mesa College and UC San Diego. Officials say 97 of the 102 Mesa College students participating in the program have transferred or plan to transfer to a four-year college or university and some students are en route to earning a doctorate.

The program focuses on academic research and provides for faculty mentors, tutors, counselors and training workshops.

Students participate in an eight-week, full-time undergraduate summer internship at UC San Diego that includes six hours a day of laboratory training with top research scientists. They also attend lectures and seminars with faculty from UCSD, the Salk Institute for Biological Sciences and the Sanford-Burnham Medical Research Institute.

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Mesa College lands $3.7 million