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A gene therapy that could restore the fading sight of the elderly is being tested on humans for the first time after positive results in blind mice.

It could be used to treat age-related macular degeneration, a common condition that usually first affects people in their 50s and 60s, scientists said.

It involves a one-time injection of a modified virus into the eye. This viral vector is altered to contain a synthetic gene that produces a protein that plays a critical role in the perception of light.

For the first time, scientists have created pigs, goats and cattle that can serve as viable “surrogate sires,” male animals that produce sperm carrying only the genetic traits of donor animals.

The advance, published in the Proceedings of the National Academy of Sciences on Sept. 14, could speed the spread of desirable characteristics in livestock and improve food production for a growing global population. It also would enable breeders in remote regions better access to genetic material of elite animals from other parts of the world and allow more precision breeding in animals such as goats where using is difficult.

“With this technology, we can get better dissemination of desirable traits and improve the efficiency of food production. This can have a major impact on addressing food insecurity around the world,” said Jon Oatley, a reproductive biologist with WSU’s College of Veterinary Medicine. “If we can tackle this genetically, then that means less water, less feed and fewer antibiotics we have to put into the animals.”

Using the Crispr gene-editing technique that won a recent Nobel Prize, Crispr Therapeutics cleared blood cancers in patients with off-the-shelf immune cells. These so-called CAR-T therapies previously required a patient’s own cells.

In a Wednesday morning announcement, Crispr Therapeutics (ticker: CRSP) said that its gene-editing let doctors use cells from healthy donors—opening up prospects for broadly available, less-expensive use of CAR-T treatment.

In the Phase 1 trial, the lymphoma blood cancer in four of 11 patients responded completely to infusions of T cells whose genes were altered to target the cancer and prevent transplant rejection. Standard treatments had failed all participants. In patients that got higher doses, the complete responses have lasted for months.

An odd lump on Elizabeth Cowles Johnston’s breast prompted a Friday morning call to her primary care physician Rebecca Andrews at UConn Health.

Dr. Andrews quickly fit her in, and upon checking the lump sent her to Dr. Alex Merkulov, Section Head of Women’s Imaging at the Beekley Imaging Center at UConn Health for a mammogram and ultrasound. The following Monday she had a biopsy of her breast and by that Wednesday she had the diagnosis of breast cancer.

“It was all very quick,” says Johnston.

Current optical techniques can image neuron activity only near the brain’s surface, but integrated neurophotonics could unlock circuits buried deep in the brain. Credit: Roukes et. al.

But current optogenetic studies of the brain are constrained by a significant physical limitation, says Laurent Moreaux, Caltech senior research scientist and lead author on the paper. Brain tissue scatters light, which means that light shone in from outside the brain can travel only short distances within it. Because of this, only regions less than about two millimeters from the brain’s surface can be examined optically. This is why the best-studied brain circuits are usually simple ones that relay sensory information, such as the sensory cortex in a mouse—they are located near the surface. In short, at present, optogenetics methods cannot readily offer insight into circuits located deeper in the brain, including those involved in higher-order cognitive or learning processes.

Integrated neurophotonics, Roukes and colleagues say, circumvents the problem. In the technique, the microscale elements of a complete imaging system are implanted near complex neural circuits located deep within the brain, in regions such as the hippocampus (which is involved in memory formation), striatum (which controls cognition), and other fundamental structures in unprecedented resolution. Consider the similar technology of functional magnetic resonance imaging (fMRI), the scanning technique currently used to image entire brains. Each voxel, or three-dimension pixel, in an fMRI scan is typically about a cubic millimeter in volume and contains roughly 100,000 neurons. Each voxel, therefore, represents the average activity of all of these 100,000 cells.

Portable sequencing is making it possible for biologists to perform DNA analysis anywhere in the world. How is this technology reshaping the way they work?

Thanks to nanopore technology, scientists can now collect samples and sequence them anywhere. It is the concept of backpacking applied to scientific research.

French molecular biologist Anne-Lise Ducluzeau has experienced this first hand during her research in the freezing environment of Alaska. “I remember driving back home with my sequencing station on the passenger seat, it was −20ºF (−29ºC) but the car was warm and reads kept coming,” ‪relates Ducluzeau, who has been using a portable sequencer for her research for the past four years.

Knowing which proteins are key to protection from disease, and the deficiencies in expression or activity that are hallmarks of disease, can inform individualized medicine and the development of new therapies.


Twenty years after the release of the human genome, the genetic “blueprint” of human life, an international research team, including the University of British Columbia’s Chris Overall, has now mapped the first draft sequence of the human proteome.

Their work was published Oct. 16 in Nature Communications and announced today by the Human Proteome Organization (HUPO).

“Today marks a in our overall understanding of human life,” says Overall, a professor in the faculty of dentistry and a member of the Centre for Blood Research at UBC. “Whereas the provides a complete ‘blueprint’ of , the human proteome identifies the individual building blocks of life encoded by this blueprint: proteins. ” Proteins interact to shape everything from life-threatening diseases to cellular structure in our bodies.”

Article. The research/article indicates that childhood trauma can not only impact the current generation, but future generations. Biochemical signals are sent to the germ cells, modifying the expression of some genes and/or the DNA structure.


Traumatic experiences can have a lasting impact, so children that suffer through them can feel their effects for a lifetime. Work has also shown that trauma can change the way genes are expressed, through epigenetics. Epigenetic changes do not alter the sequence of genes but they alter the biochemistry of DNA, and these changes are sometimes passed down to future generations through germ cells. Scientists have been working to learn more about how traumatic events get embedded in the genetic code of germ cells.

Image credit: Pkist

New research reported in The EMBO Journal has used a mouse model to suggest that childhood trauma can influence the composition of blood, and this is the conduit for passing the impact down to offspring.

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The CRISPR-Cas9 system has revolutionized genetic manipulations and made gene editing simpler, faster and easily accessible to most laboratories.

To its recognition, this year, the French-American duo Emmanuelle Charpentier and Jennifer Doudna have been awarded the prestigious Nobel Prize for chemistry for CRISPR.