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Nobel laureate details new applications at Kuh Distinguished Lecture.

Jennifer Doudna, Nobel laureate and Li Ka Shing Chancellor’s Chair and Professor in the Departments of Chemistry and of Molecular and Cell Biology, presented this year’s Ernest S. Kuh Distinguished Lecture, “Delivering the Future of CRISPR-Based Genome Editing,” on February 2 at UC Berkeley. The sold-out event — produced by Berkeley Engineering in collaboration with the Society of Women Engineers — marks the 11th talk in the lecture series, which features scientists and engineers tackling the world’s most pressing problems.

Doudna is known for developing CRISPR-Cas9, a groundbreaking technology that some call “genetic scissors.” With it, scientists can snip and edit DNA — the genetic code of life — unlocking remarkable possibilities in biology, including treatments for thousands of intractable diseases. This work has changed the course of genomics research, allowing scientists to rewrite DNA with unprecedented precision, and won Doudna and collaborator Emmanuelle Charpentier the 2020 Nobel Prize in Chemistry.

A Chinese team of life scientists, microbiologists, plant researchers and seed designers has developed a way to grow engineered moss with partially synthetic genes. In their project, reported in the journal Nature Plants, the group engineered a moss that is one of the first living things to have multiple cells carrying a partially artificial chromosome.

Several research projects have been working toward the goal of creating plants with synthetic genes —such plants could be programmed to produce more food, for example, or more oxygen, or to pull more carbon dioxide from the air. Last year, one team of researchers developed a way to program up to half of the genome of yeast cells using synthetic genes.

In this new effort, the team in China upped the ante by replacing natural moss genes with genes created in a lab—moss is far more genetically complex than yeast. They call their project SynMoss.

In a new Nature Communications study, researchers have explored the construction of genetic circuits on single DNA molecules, demonstrating localized protein synthesis as a guiding principle for dissipative nanodevices, offering insights into artificial cell design and nanobiotechnology applications.

The term “genetic circuit” is a metaphorical description of the complex network of genetic elements (such as genes, promoters, and regulatory proteins) within a cell that interact to control gene expression and cellular functions.

In the realm of artificial cell design, scientists aim to replicate and engineer these genetic circuits to create functional, self-contained units. These circuits act as the molecular machinery responsible for orchestrating cellular processes by precisely regulating the production of proteins and other molecules.

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Scientists have been making nanoparticles out of DNA strands for two decades, manipulating the bonds that maintain DNA’s double-helical shape to sculpt self-assembling structures that could someday have jaw-dropping medical applications.

The study of DNA nanoparticles, however, has focused mostly on their architecture, turning the genetic code of life into components for fabricating minuscule robots. A pair of Iowa State University researchers in the genetics, development, and cell biology department—professor Eric Henderson and recent doctoral graduate Chang-Yong Oh—hope to change that by showing nanoscale materials made of DNA can convey their built-in genetic instructions.

“So far, most people have been exploring DNA nanoparticles from an engineering perspective. Little attention has been paid to the information held in those DNA strands,” Oh said.

Women are more likely than men to have conditions such as lupus, rheumatoid arthritis, and autoimmune hepatitis (depicted above in a cellular micrograph), in which their immune response attacks healthy, functioning parts of their body. Yet the reason behind this sex-based imbalance has long eluded scientists. Now, a study published last week in proposes that a molecule associated with the X chromosome may be partly to blame. Researchers noticed that many of the proteins commonly targeted by the immune system in people with autoimmune diseases had something in common: They help a molecule called Xist carry out its function. Xist molecules act a bit like quality control inspectors for women’s extra X chromosomes, preventing them from producing a toxic amount of proteins. The scientists suspect that when immune cells encounter large bunches of these Xist-related proteins—for instance, when a dead cell spills them into the bloodstream—they may react by making antibodies to attack them throughout the body. To test the idea, the team studied genetically engineered mice in which both males and females produced Xist. Like their female counterparts, these males were also at an increased risk of developing severe cases of lupus. The researchers also found that people with autoimmune disorders had more antibodies for Xist-related proteins in their blood. Still, Xist molecules may not be the only factor at play: Experts note that some people produce these Xist-related antibodies without developing autoimmune disorders, reports.

A team of researchers at the Carl Gustav Carus Faculty of Medicine, TUD Dresden University of Technology, led by Prof. Frank Buchholz, has achieved a major breakthrough in genome editing technology. They’ve developed a cutting-edge method that combines the power of designer-recombinases with programmable DNA-binding domains to create precise and adaptable genome editing tools.

Traditional genome editing faced limitations in achieving ultimate precision until now. Prof. Buchholz’s team has broken through this barrier by creating what many have sought after: a zinc-finger conditioned recombinase. This innovative approach involves integrating a zinc-finger DNA-binding domain into specially designed recombinases. These enzymes remain inactive until the DNA-binding domain engages with its target site, adjacent to the recombinase binding area.

The significance of this achievement lies in the fusion of two key strengths: the targeting ease of programmable nucleases and the precise DNA editing capabilities of recombinases. This breakthrough overcomes existing limitations in genome editing techniques and holds vast promise for therapeutic gene editing and various biomedical applications.

Most approved gene therapies today, including those involving CRISPR-Cas9, work their magic on cells removed from the body, after which the edited cells are returned to the patient.

This technique is ideal for targeting blood cells and is currently the method employed in newly approved CRISPR gene therapies for blood diseases like sickle cell anemia, in which edited blood cells are reinfused in patients after their bone marrow has been destroyed by chemotherapy.

A new, precision-targeted delivery method for CRISPR-Cas9, published in the journal Nature Biotechnology, enables gene editing on very specific subsets of cells while still in the body—a step toward a programmable delivery method that would eliminate the need to obliterate patients’ bone marrow and immune system before giving them edited blood cells.

Some hereditary genetic defects cause an exaggerated immune response that can be fatal. Using the CRISPR-Cas9 gene-editing tool, such defects can be corrected, thus normalizing the immune response, as researchers led by Klaus Rajewsky from the Max Delbrück Center now report in Science Immunology.

Familial hemophagocytic lymphohistiocytosis (FHL) is a rare disease of the immune system that usually occurs in infants and young children under the age of 18 months. The condition is severe and has a high mortality rate. It is caused by various gene mutations that prevent cytotoxic T cells from functioning normally. These are a group of immune cells that kill virus–infected cells or otherwise altered cells.

If a child with FHL contracts a virus—such as the Epstein-Barr virus (EBV), but also other viruses—the cytotoxic T cells cannot eliminate the infected cells. Instead, the immune response gets out of control. This leads to a cytokine storm and an excessive inflammatory reaction that affects the entire organism.