Can Genome Editing Combat Disease?

by Nancy DuVergne Smith on April 23, 2015 · 1 comment

in Engineering, Health, Research

Feng Zhang applies his engineering background to problems in human health.

Feng Zhang applies his engineering background to problems in human health.

Genome editing is hot—especially at the Broad Institute of MIT and Harvard. Through a special two-day Faculty Forum Online, you can learn how a new gene editing process may transform genetic engineering and open new paths to fight disease. The Broad’s Genome Engineering 3.0 Workshop is available to MIT alumni free via webcast on May 8‒9.

The workshop is organized by Feng Zhang, who won a 2014 patent for the CRISPR-Cas9 method, and his lab at the Broad Institute. Zhang is the W.M. Keck Career Development Assistant Professor of Brain and Cognitive Sciences and Biological Engineering and a member of the McGovern Institute for Brain Research and the Broad Institute. While in graduate school at Stanford, he co-developed a revolutionary technology called optogenetics, now used by neuroscientists worldwide, and he used this and other tools to study animal models of depression and schizophrenia. His work at the Broad focuses on development of synthetic biology tools, like the CRISPR-Cas9 method, to study neuropsychiatric disease. Visit his McGovern page to view terrific short videos on his work and genome editing.

The CRISPR system has the potential to radically alter the current understanding of genetic engineering and how it could be applied to the treatment of diseases. In essence, it is a search-and-replace method for altering DNA.

Here’s how the Broad describes this breakthrough:

CRISPRs (Clustered Regularly Interspaced Short Palindromic Repeats) have been harnessed as genome-editing tools in a wide range of species. The engineered CRISPR-Cas9 system allows researchers to mutate or change the expression of genes in living cells. The family of Cas9 nucleases—the centerpiece of this genome-editing system—recognizes DNA targets in complex with RNA guides. Researchers can now use these tools to home in on specific genes within the genome and cut the DNA at those precise targets. The cuts modify the activity of the targeted genes, allowing researchers to study the genes’ function.

Want to know more?

Register today for the May 8-9 Faculty Forum Online to listen to keynotes by MIT faculty and leading researchers, technical talks, and lively debates about the future of biotechnology, ethics, intellectual property, and academia vs. industry.

{ 1 comment… read it below or add one }

Oona Houlihan May 9, 2015 at 7:04 am

Looking at what genetic engineering has achieved over the years (decades by now and still increasing in speed) and at the same time how much has been learned about epigenetics and how certain traits influence each other across various chromosomes even I still am skeptical because … evolution generally took thousands if not millions of years to “find out” if certain genetic changes were “here to stay” … and: genomic evolution has a tendency to preserve “unwanted” pieces of “code” for”ever” – q.v. the resistance genes in bacteria that are “never needed” (until … they’re needed). But what we humans tend to do, as always in our short history of technology (three hundred years is nothing against an evolutionary timescale) is to find the “one size fits all solution” – until we find that diversity has its merits even if INDIVIDUALLY it seems like a disadvantage.


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