Understanding Brain Research: CRISPR-Cas9, Optogenetics, and More

Understand the gene editing breakthrough by watching the video Genome Editing with CRISPR – Cas9

Some of today’s hottest new technologies are at work at MIT’s McGovern Institute for Brain Research. Heard of CRISPR-Cas9? It’s among the new techniques accelerating biomedical research worldwide. McGovern investigator Feng Zhang, a bioengineer with appointments at the Broad Institute and the MIT’s Brain and Cognitive Science department, developed key components of the editing tool used on the genomes of living cells and organisms.

What does CRISPR-Cas9 do? First, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) can replace damaged genes with good ones by using RNA to target a bad area and an enzyme called Cas9 to snip it out; it can also target many genes at once. Zhang, who has been called the “most transformative biologist of his generation,” is using the technique to study complex disorders, such as psychiatric and neurological diseases, that are caused by multiple genetic and environmental risk factors and are difficult to model using conventional methods.

Watch Genome Editing with CRISPR – Cas9, one of McGovern’s explainer videos, to see the technique in action.

Advanced imaging is an important tool for brain research. Find out why McGovern investigator Rebecca Saxe, pictured with her infant son, decided to create this mother and child MRI

During Zhang’s graduate studies at Stanford, he contributed to a project led by neuroscience professor Karl Deisseroth and graduate student Ed Boyden ’99, MEng ’99 on the development of optogenetics, a method of slipping light-sensitive proteins into neurons. As a result, Boyden and other scientists use light to activate specific neural circuits and reveal which circuits control specific behaviors, a way to search for the roots of illnesses such as schizophrenia.

Boyden, who also has appointments at the MIT Media Lab and the departments of brain and cognitive science and biological engineering, recently won a Breakthrough Prize for the development of optogenetics, which he is using to explore potential treatments for epilepsy, Parkinson’s disease, and mood disorders.

Watch What is optogenetics? to understand the technique.

Zhang and Boyden are among the McGovern’s 20 principal investigators who work to understand how the brain functions and to address brain disorders. Founded in 2000 thanks to a gift from Patrick J. McGovern and Lore Harp McGovern, the McGovern Institute researchers work on developmental disorders along the lifespan. Autism and dyslexia, for example, appear in early childhood. Psychiatric diseases such as depression and schizophrenia are typically diagnosed in teens or early adulthood. And Alzheimer’s disease, Parkinson’s disease, stroke, and macular degeneration tend to be challenges of later life.

“Statistics say one in four families are going to be affected by a brain disorder of some form,” says McGovern director Robert Desimone in a recent video. And that’s one reason the McGovern Institute’s multi-disciplinary neuroscientists are working on these widespread problems. “I think in my lifetime I will see serious improvements in our treatments of brain disorders.”

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