CRISPR

A single genetic mutation can have profound consequences, as demonstrated in neurodegenerative diseases such as amyotrophic lateral sclerosis or Huntington’s disease. A new study by University of Illinois Urbana-Champaign researchers used a targeted CRISPR technique in the central nervous systems of mice to turn off production of mutant proteins that can cause ALS and Huntington’s disease.

The National Institutes of Health have awarded a $2.2 million grant to researchers at the Carl R. Woese Institute for Genomic Biology. The four-year grant will be used to develop more precise genome editing technologies for gene therapy applications.

Microorganisms possess natural product biosynthetic gene clusters (BGCs) that may harbor unique bioactivities for use in drug development and agricultural applications. However, many uncharacterized microbial BGCs remain inaccessible. Researchers at Illinois previously demonstrated a technique using transcription factor decoys to activate large, silent BGCs in bacteria to aid in natural product discovery.

Researchers used single-molecule imaging to compare the genome-editing tools CRISPR-Cas9 and TALEN. Their experiments revealed that TALEN is up to five times more efficient than CRISPR-Cas9 in parts of the genome, called heterochromatin, that are densely packed. Fragile X syndrome, sickle cell anemia, beta-thalassemia and other diseases are the result of genetic defects in the heterochromatin.

Genome editing of human embryos represents one of the most contentious potential scientific applications today. But what if geneticists could sidestep the controversy by editing sperm and eggs instead?

Just like humans, microbes have equipped themselves with tools to recognize and defend themselves against viral invaders. In a continual evolutionary battle between virus and host, CRISPR-Cas acts as a major driving force of strain diversity in host-virus systems.

Over the years, the democratization of synthetic biology for the production of food has led to products like the Impossible burger, a burger impostor that uses plant tissues instead of meat. Despite this, food companies remain hesitant to utilize synthetic biology due to concerns with genetically modified foods.

With a new CRISPR gene-editing methodology, scientists from the University of Illinois at Urbana-Champaign inactivated one of the genes responsible for an inherited form of amyotrophic lateral sclerosis – a debilitating and fatal neurological disease for which there is no cure. The novel treatment slowed disease progression, improved muscle function and extended lifespan in mice with an aggressive form of ALS.

University of Illinois researchers achieved the highest reported rates of inserting genes into human cells with the CRISPR-Cas9 gene-editing system, a necessary step for harnessing CRISPR for clinical gene-therapy applications.

By chemically tweaking the ends of the DNA to be inserted, the new technique is up to five times more efficient than current approaches. The researchers saw improvements at various genetic locations tested in a human kidney cell line, even seeing 65% insertion at one site where the previous high had been 15%.

Genomic research has unlocked the capability to edit the genomes of living cells; yet so far, the effects of such changes must be examined in isolation. In contrast, the complex traits that are of interest in both fundamental and applied research, such as those related to microbial biofuel production, involve many genes acting in concert. A newly developed system will now allow researchers to fine-tune the activity of multiple genes simultaneously.