“We have entered the age where the human genome is a real drug target” – CRISPR stopped mice from going deaf

From This gene therapy stopped mice from going deaf — and could save some humans’ hearing too – The Verge

Although people can lose their hearing for a variety of reasons — old age, as well as exposure to loud noises — genetics are behind a little less than half of all deafness cases, says study co-author David Liu, a professor of chemistry and chemical biology at Harvard, who also has affiliations with the Broad Institute and the Howard Hughes Medical Institute. The hearing-loss disease tackled in this study is caused by mutations in a gene called TMC1. These mutations cause the death of so-called hair cells in the inner ear, which convert mechanical vibrations like sound waves into nerve signals that the brain interprets as hearing. As a result, people start losing their hearing in their childhood or in the 20s, and can go completely deaf by their 50s and 60s.

To snip those mutant copies of the gene, Liu and his colleagues mixed CRISPR-Cas9 with a lipid droplet that allows the gene-editing tool to enter the hair cells and get to work. When the concoction was injected into one ear of newborn mice with the disease, the molecular scissors were able to precisely cut the deafness-causing copy of the gene while leaving the healthy copy alone, even if the two copies differ by just one base pair. The treatment allowed the hair cells to stay healthier and prevented the mice from going deaf.

After four weeks, the untreated ears could only pick up noises that were 80 decibels or louder, roughly as loud as a garbage disposal, Liu says. Instead, the injected ears could typically hear sounds in the 60 to 65 decibel range, which is the same as a quiet conversation. “If one can translate that 15 decibel improvement in hearing sensitivity in humans, it would actually make a potential difference in the quality of their hearing capability,” Liu tells The Verge.

RNA targeting with CRISPR–Cas13

From RNA targeting with CRISPR–Cas13 : Nature

RNA has important and diverse roles in biology, but molecular tools to manipulate and measure it are limited. For example, RNA interference can efficiently knockdown RNAs, but it is prone to off-target effects, and visualizing RNAs typically relies on the introduction of exogenous tags. Here we demonstrate that the class 2 type VI RNA-guided RNA-targeting CRISPR–Cas effector Cas13a (previously known as C2c2) can be engineered for mammalian cell RNA knockdown and binding.

After initial screening of 15 orthologues, we identified Cas13a from Leptotrichia wadei (LwaCas13a) as the most effective in an interference assay in Escherichia coli. LwaCas13a can be heterologously expressed in mammalian and plant cells for targeted knockdown of either reporter or endogenous transcripts with comparable levels of knockdown as RNA interference and improved specificity. Catalytically inactive LwaCas13a maintains targeted RNA binding activity, which we leveraged for programmable tracking of transcripts in live cells.

Our results establish CRISPR–Cas13a as a flexible platform for studying RNA in mammalian cells and therapeutic development.

A Textile Dressing for Temporal and Dosage Controlled Drug Delivery

From A Textile Dressing for Temporal and Dosage Controlled Drug Delivery – Mostafalu – 2017 – Advanced Functional Materials – Wiley Online Library

Chronic wounds do not heal in an orderly fashion in part due to the lack of timely release of biological factors essential for healing. Topical administration of various therapeutic factors at different stages is shown to enhance the healing rate of chronic wounds. Developing a wound dressing that can deliver biomolecules with a predetermined spatial and temporal pattern would be beneficial for effective treatment of chronic wounds. Here, an actively controlled wound dressing is fabricated using composite fibers with a core electrical heater covered by a layer of hydrogel containing thermoresponsive drug carriers. The fibers are loaded with different drugs and biological factors and are then assembled using textile processes to create a flexible and wearable wound dressing. These fibers can be individually addressed to enable on-demand release of different drugs with a controlled temporal profile. Here, the effectiveness of the engineered dressing for on-demand release of antibiotics and vascular endothelial growth factor (VEGF) is demonstrated for eliminating bacterial infection and inducing angiogenesis in vitro. The effectiveness of the VEGF release on improving healing rate is also demonstrated in a murine model of diabetic wounds.

Universities testing a smart bandage that automatically dispenses medication

From This smart bandage releases meds on command for better healing | TechCrunch

Instead of plain sterile cotton or other fibers, this dressing is made of “composite fibers with a core electrical heater covered by a layer of hydrogel containing thermoresponsive drug carriers,” which really says it all.

It acts as a regular bandage, protecting the injury from exposure and so on, but attached to it is a stamp-sized microcontroller. When prompted by an app (or an onboard timer, or conceivably sensors woven into the bandage), the microcontroller sends a voltage through certain of the fibers, warming them and activating the medications lying dormant in the hydrogel.

Those medications could be anything from topical anesthetics to antibiotics to more sophisticated things like growth hormones that accelerate healing. More voltage, more medication — and each fiber can carry a different one.

Researchers Find A Way To Move Tiny, Foldable Robots Without Batteries

From Someday You Could Swallow This Wireless Robot Like A Pill

These little robots are powered by an electromagnetic field, similar to how you would wirelessly charge a cell phone. By changing the frequency of the magnetic field, the researchers are able to precisely control the exact movement of their prototype.

For instance, one triangular robot that’s no bigger than a quarter is composed of three triangular pieces of thin plastic, attached with hinges to a middle triangle that has a circuit. The hinges are controlled by coils of a metal called “shape-memory alloy,” which changes its form when it’s exposed to heat. When an electric current starts running through the central circuit, these coils heat up and contract, causing the three triangles to fold up toward the center of the robot. When the current stops, the hinges return to their flat state.