Lifeboat Foundation

Scientists have categorized different types of CRISPR systems into two classes based on how their Cas nucleases function. In class 1 (types I, III, and IV), different Cas proteins form a complex machinery to identify and cut foreign DNA; in class 2 CRISPR systems (types II, V, and VI), a single Cas protein effector recognizes and cleaves DNA.⁹

After characterizing CRISPR’s role as a defense mechanism in bacteria, researchers soon realized that they could harness this system for gene manipulation in any cell. All they needed to do was design a CRISPR gRNA sequence that bound to a specific DNA sequence and triggered the Cas nuclease, which would then cut precisely at that location. With CRISPR, researchers routinely knock out gene function by cutting out a DNA fragment, or they insert a desired genetic sequence into the genome by providing a reference DNA template along with the CRISPR components. While editing eukaryotic cells has been the focus for tackling diseases, many researchers now use CRISPR to edit bacterial communities.

“It’s almost like back to the beginning or back to the origins. There’s some irony in bringing CRISPR back to where it came from,” said Rodolphe Barrangou, a functional genomics researcher at North Carolina State University, who helped characterize the immune function of CRISPR and has been working with it for more than 20 years.

Nanomedicine to Cure All!

Aortic aneurysms are bulges in the aorta, the largest blood vessel that carries oxygen-rich blood from the heart to the rest of the body. Smoking, high blood pressure, diabetes, or injury can all increase the risk of aneurysms, which tend to occur more often in Caucasian male smokers over the age of 65.

“The soft tissues that make up blood vessels act essentially like rubber bands, and it’s the elastic fibers within these tissues that allow them to stretch and snap back,” says Professor Anand Ramamurthi, chair of the Department of Bioengineering in Lehigh University’s P.C. Rossin College of Engineering and Applied Science. “These fibers are produced primarily before and just after birth. After that, they don’t regenerate or undergo natural repair after injury. So when they become injured or diseased, the tissue weakens and causes an aneurysm, which can grow over time. After about seven to 10 years, it typically reaches the rupture stage.”

Continue reading “Nanomedicine research aims to transform treatment of aortic aneurysms” »

“If you look at the brain chemically, it’s like a soup with a bunch of ingredients,” said Dr. Fan Lam.

Can we map the brain to show its behavior patterns when a patient is healthy and sick? This is what a recent study published in Nature Methods hopes to address as a team of researchers at the University of Illinois Urbana-Champaign used a $3 million grant obtained from the National Institute of Aging to develop a novel approach to mapping brain behavior when a patient is both healthy and sick. This study holds the potential to help researchers, medical professionals, and patients better understand how to treat diseases.

“If you look at the brain chemically, it’s like a soup with a bunch of ingredients,” said Dr. Fan Lam, who is an assistant professor of bioengineering at the University of Illinois Urbana-Champaign and a co-author on the study. “Understanding the biochemistry of the brain, how it organizes spatiotemporally, and how those chemical reactions support computing is critical to having a better idea of how the brain functions in health as well as during disease.”

Continue reading “3D Molecular Maps of the Brain: Unveiling Complexity with Spatial Omics” »

An endoscopic mapping device, developed over the course of a decade by scientists at the Auckland Bioengineering Institute, consists of an inflatable sphere covered in sensors, delivered down the esophagus and able to measure electrical activity in the gut.

In the same way, abnormal heart electrical signals can cause serious heart problems, research has found faulty bioelectric gut waves can lead to stomach pain, nausea, vomiting and bloating.

But often doctors can’t find out what the problem is. That’s because gut electrics aren’t nearly as strong or as easily measured as heart waves; without surgery it’s hard to know if someone has a so-called ‘dysrhythmic’ gut—and if so, where the problem is.

As a therapy for vision impairment resulting from inherited retinal degeneration, the mRNA would instruct cells in the retina, which are impaired because of a genetic mutation, to manufacture the proteins needed for sight. Inherited retinal degeneration, commonly abbreviated to IRD, encompasses a group of disorders of varying severity and prevalence that affect one out of every few thousand people worldwide.

An example of a genetic pulmonary condition is cystic fibrosis, a progressive disorder that results in persistent lung infection and affects 30,000 people in the U.S., with about 1,000 new cases identified every year. One faulty gene—the cystic fibrosis transmembrane conductance regulator, or CFTR—causes the disease, which is characterized by lung dehydration and mucus buildup that blocks the airway.

The thiophene-based LNP study, which involved mice and non-human primates, stems from a $3.2 million grant from the National Eye Institute. The grant’s purpose is addressing limitations associated with the current primary means of delivery for gene editing: adeno-associated virus, or AAV.

Plant genomics has come a long way since Cold Spring Harbor Laboratory (CSHL) helped sequence the first plant genome. But engineering the perfect crop is still, in many ways, a game of chance. Making the same DNA mutation in two different plants doesn’t always give us the crop traits we want. The question is why not? CSHL plant biologists just dug up a reason.

CSHL Professor and HHMI Investigator Zachary Lippman and his team discovered that tomato and Arabidopsis thaliana plants can use very different regulatory systems to control the same exact gene. Incredibly, they linked this behavior to extreme genetic makeovers that occurred over 125 million years of evolution.

The scientists used genome editing to create over 70 mutant strains of tomato and Arabidopsis thaliana plants. Each mutation deleted a piece of regulatory DNA around a gene known as CLV3. They then analyzed the effect each mutation had on plant growth and development. When the DNA keeping CLV3 in check was mutated too much, fruit growth exploded. They published their findings in PLoS Genetics.

Using lipid nanoparticles (LNPs), engineers have successfully delivered genetic material to the lung that suppresses lung tumors in mice.

Retrotransposons can insert new genes into a “safe harbor” in the genome, complementing CRISPR gene editing.

The recent greenlighting of a CRISPR-Cas9 treatment for sickle cell disease underscores the efficacy of gene editing technologies in deactivating genes to heal inherited illnesses. However, the capability to integrate entire genes into the human genome as replacements for faulty or harmful ones remains unachievable.

A new technique that employs a retrotransposon from birds to insert genes into the genome holds more promise for gene therapy, since it inserts genes into a “safe harbor” in the human genome where the insertion won’t disrupt essential genes or lead to cancer.

“Even primary school students and old farmers can master gene editing,” says [Southern University of Science and Technology, or SUSTech] scientist Zhu Jian-Kang, who has helped develop a new approach that could greatly simplify the difficult and time-consuming process of editing genes in plants.

While conventional methods of heritable gene editing in plants often take months, and in some cases up to a year, this innovative approach could reduce the process to about two weeks, according to [Cao Xuesong, a scientist at SUSTech and a member of Zhu’s team], who is also the first author of the study.