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BIO 2020: Cell and Gene Therapies Will Dominate Medicine in 30 Years

One day, cell and gene therapies will be as common as small molecules and antibody-based therapies are today, according to panelists at BIO’s June 8 virtual session, “The Next Generation of Medicine: Cell Therapies, Gene Therapies and Beyond.”

Because cell and gene therapies have the potential to address complex biological issues such as dysregulation, translocation and mutations, they can use that power to change what the body is doing.

“So, while small molecules and antibody therapies will still be around 30 years from now, they will be less important. Cell and gene therapies will dominate,” James Sabry, global head of pharma partnering at Roche, said.

Very fast CRISPR on demand

Numerous efforts have been made to improve the temporal resolution of CRISPR-Cas9–mediated DNA cleavage to the hour time scale. Liu et al. developed a Cas9 system that achieved genome-editing manipulation at the second time scale (see the Perspective by Medhi and Jasin). Part of the guide RNA is chemically caged, allowing the Cas9-guide RNA complex to bind at a specific genomic locus without cleavage until activation by light. This fast CRISPR system achieves genome editing at high temporal resolution, enabling the study of early molecular events of DNA repair processes. This system also has high spatial resolution at short time scales, allowing editing of one genomic allele while leaving the other unperturbed.

Science, this issue p. 1265; see also p. 1180

CRISPR-Cas systems provide versatile tools for programmable genome editing. Here, we developed a caged RNA strategy that allows Cas9 to bind DNA but not cleave until light-induced activation. This approach, referred to as very fast CRISPR (vfCRISPR), creates double-strand breaks (DSBs) at the submicrometer and second scales. Synchronized cleavage improved kinetic analysis of DNA repair, revealing that cells respond to Cas9-induced DSBs within minutes and can retain MRE11 after DNA ligation. Phosphorylation of H2AX after DNA damage propagated more than 100 kilobases per minute, reaching up to 30 megabases. Using single-cell fluorescence imaging, we characterized multiple cycles of 53BP1 repair foci formation and dissolution, with the first cycle taking longer than subsequent cycles and its duration modulated by inhibition of repair. Imaging-guided subcellular Cas9 activation further facilitated genomic manipulation with single-allele resolution.

Smallest cavity for light realized by graphene plasmons

Miniaturization has enabled technology like smartphones, health watches, medical probes and nano-satellites, all unthinkable a couple decades ago. Just imagine that in the course of 60 years, the transistor has shrunk from the size of your palm to 14 nanometers in dimension, 1000 times smaller than the diameter of a hair.

Miniaturization has pushed technology to a new era of optical circuitry. But in parallel, it has also triggered new challenges and obstacles, for example, controlling and guiding at the nanometer scale. Researchers are looking for techniques to confine light into extremely tiny spaces, millions of times smaller than current ones. Studies had earlier found that metals can compress light below the wavelength-scale (diffraction limit).

In that aspect, , a material composed from a single layer of carbon atoms, which exhibits exceptional optical and electrical properties, is capable of guiding light in the form of plasmons, which are oscillations of electrons that strongly interact with light. These graphene plasmons have a natural ability to confine light to very small spaces. However, until now, it was only possible to confine these plasmons in one direction, while the actual ability of light to interact with small particles like atoms and molecules resides in the volume into which it can be compressed. This type of confinement in all three dimensions is commonly regarded as an optical cavity.

Sound waves transport droplets for rewritable lab-on-a-chip devices

Engineers at Duke University have demonstrated a versatile microfluidic lab-on-a-chip that uses sound waves to create tunnels in oil to touchlessly manipulate and transport droplets. The technology could form the basis of a small-scale, programmable, rewritable biomedical chip that is completely reusable to enable on-site diagnostics or laboratory research.

The results appear online on June 10 in the journal Science Advances.

“Our new system achieves rewritable routing, sorting and gating of droplets with minimal external control, which are essential functions for the digital logic control of droplets,” said Tony Jun Huang, the William Bevan Distinguished Professor of Mechanical Engineering and Materials Science at Duke. “And we achieve it with less energy and a simpler setup that can control more droplets simultaneously than previous systems.”

Squid and octopus can edit and direct their own brain genes

Circa 2017


Unlike other animals, cephalopods – the family that includes octopuses, squid and cuttlefish – do not obey the commands of their DNA to the letter.

Instead, they sometimes interfere with the code as it is being carried by a molecular “messenger”. This has the effect of diversifying the proteins their cells can produce, leading to some interesting variations.

The system may have produced a special kind of evolution based on RNA editing rather than DNA mutations and could be responsible for the complex behaviour and high intelligence seen in cephalopods, some scientists believe.

Cheese Triggers the Same Part of Brain as Hard Drugs

Independent.co.uk

Cheese contains a chemical found in addictive drugs, scientists have found.

The team behind the study set out to pin-point why certain foods are more addictive than others.

Using the Yale Food Addiction Scale, designed to measure a person’s dependence on, scientists found that cheese is particularly potent because it contains casein.

Scientists grow ‘model’ human embryos from stem cells

Paris (AFP) — Scientists have developed a human embryo “blueprint” using human stem cells, in a breakthrough that could provide vital insight into the early stages of infant development, new research showed Thursday.

Teams from the University of Cambridge and the Netherlands-based Hubrecht Institute said their model will allow them to observe never-before-seen processes underlying the formation of the human body.

The layout of humans — known as the body plan — happens through a process known as gastrulation, where three distinct layers of cells are formed in the embryo that will later give rise to the body’s three main systems: nervous, musculoskeletal and digestive.

Covid-19 Patient Gets Double Lung Transplant, Offering Hope for Others

“The operation is believed to be the first of its kind in the U.S. The patient, a woman in her 20s, had been healthy, but the coronavirus devastated her lungs.”


A young woman whose lungs were destroyed by the coronavirus received a double lung transplant last week at Northwestern Memorial Hospital in Chicago, the hospital reported on Thursday, the first known lung transplant in the United States for Covid-19.

The 10-hour surgery was more difficult and took several hours longer than most lung transplants because inflammation from the disease had left the woman’s lungs “completely plastered to tissue around them, the heart, the chest wall and diaphragm,” said Dr. Ankit Bharat, the chief of thoracic surgery and surgical director of the lung transplant program at Northwestern Medicine, which includes Northwestern Memorial Hospital, in an interview.

He said the patient, a woman in her 20s who had no serious underlying medical conditions, was recovering well: “She’s awake, she’s smiling, she FaceTimed with her family.”

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