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A Single Injection Reverses Blindness in Patient with Rare Genetic Disorder – Another RNA Success

A patient with a genetic form of childhood blindness gained vision, which lasted more than a year, after receiving a single injection of an experimental RNA therapy into the eye.

The gene editing research was conducted at the Perelman School of Medicine in the University of Pennsylvania. Results of the case, detailed in a paper published April 1 in Nature Medicine, show that the treatment led to marked changes at the fovea, the most important point of human central vision.

In the international clinical trial, participants received an intraocular injection of an antisense oligonucleotide called sepofarsen. This short RNA molecule works by increasing normal CEP290 protein levels in the eye’s photoreceptors and improving retinal function under day vision conditions.

Gut Bacteria Boost Host NAD Metabolism

Here’s my latest video (audio issues fixed!):


Papers referenced in the video:

Bacteria Boost Mammalian Host NAD Metabolism by Engaging the Deamidated Biosynthesis Pathway:
https://pubmed.ncbi.nlm.nih.gov/32130883/

CD38 Dictates Age-Related NAD Decline and Mitochondrial Dysfunction through an SIRT3-Dependent Mechanism:
https://pubmed.ncbi.nlm.nih.gov/27304511/

Aerobic and resistance exercise training reverses age-dependent decline in NAD + salvage capacity in human skeletal muscle:

Dr. María Blasco refers to Telomerase and Life Extension (in English with S/T en Español)

Extract from a conversation that María Blasco, Director of the Spanish National Cancer Research Center (CNIO for its acronym in Spanish) had with Mario Alonso Puig during the celebration of the South Summit 2020.

In this segment María Blasco refers to aging, cancer, telomerase, and life extension. The conversation is in English and I added subtitles in Spanish.

I find the message particularly important because Dr. María Blasco refers again (she already did it in a scientific paper) to the fact that, contrary to what she herself would have expected and was a concern within the scientific community, inducing the production of Telomerase in mice, besides from lengthening significantly their healthspan and lifespan, not only it did not cause Cancer but quite the opposite, reduced or even eliminated the occurrence of it.

Century-old problem solved with first-ever 3D atomic imaging of an amorphous solid

Glass, rubber and plastics all belong to a class of matter called amorphous solids. And in spite of how common they are in our everyday lives, amorphous solids have long posed a challenge to scientists.

Since the 1910s, scientists have been able to map in 3D the atomic structures of crystals, the other major class of solids, which has led to myriad advances in physics, chemistry, biology, , geology, nanoscience, drug discovery and more. But because aren’t assembled in rigid, repetitive atomic structures like crystals are, they have defied researchers’ ability to determine their with the same level of precision.

Until now, that is.

Faulty brain circuit helps explain obesity–depression link

The team found that feeding mice a high fat diet disrupted the circuit, which led not only to weight gain but also to signs of anxiety and depression on standard behavioral tests.

When the researchers used genetic techniques to restore the normal functioning of nerve receptors in the circuit, this resulted in weight loss and eliminated the animals’ signs of anxiety and depression.


A recent study in mice has found that eating a high fat diet may disrupt a newly discovered neural circuit that affects both mood and appetite.

‘Neutrobots’ smuggle drugs to the brain without alerting the immune system

A team of researchers from the Harbin Institute of Technology along with partners at the First Affiliated Hospital of Harbin Medical University, both in China, has developed a tiny robot that can ferry cancer drugs through the blood-brain barrier (BBB) without setting off an immune reaction. In their paper published in the journal Science Robotics, the group describes their robot and tests with mice. Junsun Hwang and Hongsoo Choi, with the Daegu Gyeongbuk Institute of Science and Technology in Korea, have published a Focus piece in the same journal issue on the work done by the team in China.

For many years, medical scientists have sought ways to deliver drugs to the brain to treat health conditions such as brain cancers. Because the brain is protected by the skull, it is extremely difficult to inject them directly. Researchers have also been stymied in their efforts by the BBB—a filtering mechanism in the capillaries that supply blood to the brain and that blocks foreign substances from entering. Thus, simply injecting drugs into the bloodstream is not an option. In this new effort, the researchers used a defense cell type that naturally passes through the BBB to carry drugs to the brain.

To build their tiny robots, the researchers exposed groups of white blood cells called neutrophils to tiny bits of magnetic nanogel particles coated with fragments of E. coli material. Upon exposure, the neutrophils naturally encased the tiny robots, believing them to be nothing but E. coli bacteria. The microrobots were then injected into the bloodstream of a test mouse with a cancerous tumor. The team then applied a to the robots to direct them through the BBB, where they were not attacked, as the identified them as normal neutrophils, and into the brain and the tumor. Once there, the robots released their cancer-fighting drugs.

Super-speedy Israeli COVID test gets European approval; airport rollout planned

An Israeli company said Wednesday that it received European approval for its rapid coronavirus test and it was poised to help kickstart international travel.

The handheld SpectraLIT machine eliminates the need for complex lab equipment by shining light through samples and giving immediate results using the spectral signature.

This means that staff in airport booths who are currently tasked with collecting test samples and dispatching them to labs will simply have a machine at hand and be able to give passengers results after just 20 seconds of analysis.

Houston startup with life-saving innovation receives $2M grant

A $2 million federal grant will enable Houston-based PolyVascular to launch human trials of what it hails as the first polymer-based heart valve for children.

In conjunction with the grant, Dr. Will Clifton has joined the medical device company as chief operating officer. He will oversee the grant as principal investigator, and will manage the company’s operations and R&D. Clifton is president and co-founder of Houston-based Enventure, a medical innovation incubator and education hub. He previously was senior director of medical affairs at Houston-based Procyrion, a clinical-stage medical device company.

PolyVascular’s Phase II grant came from the Small Business Innovation Research (SBIR) program, which promotes technological projects.

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