Lifeboat Foundation

Following the landmark approval of the first CRISPR-based cell therapy in December 2023, the CRISPR community is looking ahead to the next wave of commercial successes, fueled by continued innovation in the development of new gene editing and delivery tools and technologies. Equally exciting advances are occurring in livestock editing, xenotransplantation, and many other specialties.

In The State of CRISPR and Gene Editing virtual summit, GEN proudly gathers a tantalizing line-up of luminaries from academia and industry to discuss the latest research developments, innovations, and advanced technologies that are expanding the CRISPR toolbox, delivering new therapies to patients and safeguarding our food supply.

Cells in the human body chat with each other all the time. One major way they communicate is by releasing tiny spheres called exosomes. These carry fats, proteins, and genetic material that help regulate everything from pregnancy and immune responses to heart health and kidney function.

Now, a new Columbia University study in Nature Nanotechnology demonstrated that these “nanobubbles” can deliver potent immunotherapy directly to tough-to-treat lung cancer tumors via inhalation.

“Exosomes work like text messages between cells, sending and receiving information,” said lead researcher Ke Cheng, PhD, professor of biomedical engineering at Columbia. “The significance of this study is that exosomes can bring mRNA-based treatment to lung cancer cells locally, unlike systemic chemotherapy that can have side effects throughout the body. And inhalation is totally noninvasive. You don’t need a nurse to use an IV needle to pierce your skin.”

Editor’s note: This story is being highlighted in ASU Now’s year in review. Read more top stories from 2018 here.

In a major advancement in nanomedicine, Arizona State University scientists, in collaboration with researchers from the National Center for Nanoscience and Technology (NCNST) of the Chinese Academy of Sciences, have successfully programmed nanorobots to shrink tumors by cutting off their blood supply.

“We have developed the first fully autonomous, DNA robotic system for a very precise drug design and targeted cancer therapy,” said Hao Yan, director of the ASU Biodesign Institute’s Center for Molecular Design and Biomimetics and the Milton Glick Professor in the School of Molecular Sciences.

Using gene editing for human enhancement.

Shared with Dropbox.

The brain is one of the most complex entities in biology. For thousands of years, humans have wondered how the human brain works, but only in the past few years has technology evolved so that scientists can actually answer some of the many questions we have. What are the causes of brain disorders? How do our brains develop? How does the brain heal after a head injury? While we still have a long way to go before we can understand the many facets of the human brain, one technology – CRISPR – has allowed us to start answering these questions on a genetic level.

What is CRISPR?

Down syndrome (DS) is one of the most prevalent genetic disorders in humans. The use of new approaches in genetic engineering and nanotechnology methods in combination with natural cellular phenomenon can modify the disease in affected people. We consider two CRISPR/Cas9 systems to cut a specific region from short arm of the chromosome 21 (Chr21) and replace it with a novel designed DNA construct, containing the essential genes in chromatin remodeling for inactivating of an extra Chr21. This requires mimicking of the natural cellular pattern for inactivation of the extra X chromosome in females. By means of controlled dosage of an appropriate Nano-carrier (a surface engineered Poly D, L-lactide-co-glycolide (PLGA) for integrating the relevant construct in Trisomy21 brain cell culture media and then in DS mouse model, we would be able to evaluate the modification and the reduction of the active extra Chr21 and in turn reduce substantial adverse effects of the disease, like intellectual disabilities. The hypothesis and study seek new insights in Down syndrome modification.

Keywords: Down syndrome, CRISPR/Cas9, Designed DNA construct, Poly D L-lactide-co-glycolide (PLGA), Nano-carrier, Chromosome 21 inactivation.

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CRISPR gene editing leads to improvements in vision for people with inherited blindness, a recent clinical trial shows.

“Engineering the brain”. There was no intelligent design, and as a result, body organs do not resemble machines. Once we start building machines like body organs — with utility functions, self-organization and cells as building blocks, we can mesh engineering and evolutionary principles to arrive at better organisms.

Each CRISPR system has two parts: a strand of RNA that matches the target and a protein that makes the edit. The most commonly used protein for gene editing is called “Cas9,” but scientists have discovered CRISPRs with other proteins that give them unique capabilities — while CRISPR-Cas9 slices through DNA, for example, CRISPR-Cas13 targets RNA.

Our current CRISPR gene editors are far from perfect, though. They can make edits in the wrong places or edit too few cells to make a difference, so researchers are constantly on the hunt for new CRISPR systems.

AI-designed CRISPR: Up until now, that hunt has been limited to the CRISPRs that have been discovered in nature, but Profluent has used the same types of AI models that allow ChatGPT to generate language to develop an AI platform that can generate millions of CRISPR-like proteins.