Article: How CRISPR Tools are Unlocking New Ways to Fight Disease

[Webdog]

https://www.ucsf.edu/news/2018/07/411031/crispr-toolkit-unlocks-new-ways-fight-disease

How CRISPR Tools are Unlocking New Ways to Fight Disease
CRISPR-dCas9, Invented at UCSF, Has Turned a Genetic Scalpel into a Swiss Army Knife
By Nina Bai on July 05, 2018

Recent leaps in gene editing technology have brought ideas that just a decade ago seemed like science fiction to the cusp of reality.

The already famous CRISPR system allows scientists to edit faulty genes by cutting and replacing sections of DNA, but new and improved CRISPR techniques have expanded CRISPR’s scalpel into a Swiss Army knife. The new tools give researchers more flexible control of gene function without permanently altering an organism’s genetic code.

These versatile tools are helping to untangle the complex genetics underlying diseases such as cancer and autoimmune disorders. They could identify new targets for drug development or point the way for gene therapies that could one day target genetic defects related to blindness or obesity.

Revealing Hidden Switches
Even as these new CRISPR tools hint at new avenues for gene therapy, they’re already opening the field for other therapies by revealing the complex genetic interactions that underlie diseases.

The most immediate payoff may be in identifying new therapeutic targets, according to Bruce Conklin, MD, professor of medicine at UCSF and senior investigator at Gladstone Institutes, who works with CRISPRi and related techniques in his research.

“The power of these techniques is to test thousands of different drug targets in a single experiment. This has already helped with leads for potentially useful drugs,” he said.

It’s estimated that 98 percent of our DNA does not code for proteins, but instead act as a switchboard for the 2 percent that do. These so-called promotors and enhancers can be cryptic and time-consuming to study with conventional genetic techniques.

With CRISPRa, Marson’s lab can rapidly screen over 20,000 non-coding sites in the genome and study their function – essentially by flipping many switches and seeing which ones turn on the lights. “This is a critical step in identifying the functional significance of genetic elements,” said Marson.

In a recent CRISPRa screen, Marson’s lab, in collaboration with the lab of Jacob Corn, PhD, at UC Berkeley, identified several enhancers associated with inflammation and autoimmune disorders. In fact, one of the newly identified enhancers matched a common genetic variant known to increase the risk of irritable bowel disease, although its mechanism had previously been a mystery.

 

[alktipping]

a common genetic variant known to increase I b s . that should put a spanner in the iapt mus brigade one more illness that psychs can no longer claim as a mental disorder.

 

[TiredSam]

 

[Webdog]

New CRISPR article from UCSF.

The First Genome Surgeons: Scientists Are Preparing to Bring DNA-Editing Tools to the Clinic
https://www.ucsf.edu/news/2018/10/4...-preparing-to-bring-gene-editing-tools-clinic

Like most genome surgery pioneers, he is cautiously optimistic that his efforts will pay off. The rapid rise of CRISPR technology, followed by early therapeutic progress, has given scientists and physicians alike reason to be hopeful — to “feel encouraged,” as Doudna says, “that this is something that in the next few years will be increasingly available to patients.”

At the same time, many important questions remain: How will physicians deliver CRISPR systems to hard-to-reach tissues such as the heart? How will they treat diseases with many underlying and interacting gene mutations? How will they educate patients about the risks and benefits? What exactly are the risks and benefits? What are the proper doses? How will these surgeries be regulated? Who will perform them? Who will pay for them? Who will have access to them?

Edit: Changed link to point to a more reliable website.

 

[wastwater]

I always wondered could this go wrong and spread like an infection that mutates genes

 

[Webdog]

I always wondered could this go wrong and spread like an infection that mutates genes

A decade or so ago, I heard a physics professor say that editing the human genome to reduce the human drive to reproduce and consume resources was the only way our species could survive longterm.

Perhaps he was right.