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Turning Piglets Into Personalized Avatars for Sick Kids

Written by: Ed Yong

A gene-edited pig with an NF-1 mutation. Photo credit: Jeff Miller, University of Wisconsin-Madison

A team of scientists wants to accelerate research into a genetic disorder by using CRISPR to copy unique mutations from affected children into pigs.

When Charles Konsitzke and Dhanu Shanmuganayagam first met, they were both just trying to get some peace and quiet. It was early 2014, and they were representing the University of Wisconsin-Madison at a fancy event to promote the university’s research to local politicians. After hours of talking to senators, Shanmuganayagam was fried, and went for a walk to clear his head. That’s when he bumped into Konsitzke, an administrator at the University of Wisconsin’s Biotechnology Center. They introduced themselves, and discussed their work. Shanmuganayagam said that he ran a facility that rears miniature pigs, which are genetically engineered to carry mutations found in human genetic disorders. Scientists can study the mini-pigs to better understand those diseases.

“And I said: I have a project for you,” Konsitzke recalls.

Konsitzke’s son Mason, now aged 7, was born with little brown birthmarks on his buttocks. Many kids have one or two of these café-au-lait spots and at first, Konsitzke thought they were cute. But after more appeared, he did some research and found that such spots are a common symptom of neurofibromatosis type 1 (NF-1)—an incurable inherited disease. Around Mason’s first birthday, a pediatrician confirmed the diagnosis. Read more.

Published by The Atlantic December 13, 2017

Texas Zika Researchers Studying Infected Primates To Help Prevent Birth Defects

Written by Joey Palacios

Photo credit: Texas Biomedical Research Institute

Scientists at Texas BioMedical Research Institute in San Antonio are using a type of primate to help prevent birth defects caused by the Zika virus. Texas BioMed is using four marmosets as its animal model for Zika infection. Virologist Dr. Jean Patterson said Zika infection in marmosets is similar to that in humans.

“Like humans, they develop almost immediate Viremia — meaning they have virus in their blood — and, for the males, after the virus declines in blood it then goes into semen, saliva and blood,” she said. Read more. 

Published December 11, 2017, by Texas Public Radio 

Hibernating ground squirrels provide clues to new stroke treatments

Multi-step screening process leads to molecule that may protect brain cells. 

In the fight against brain damage caused by stroke, researchers have turned to an unlikely source of inspiration: hibernating ground squirrels.

While the animals’ brains experience dramatically reduced blood flow during hibernation, just like human patients after a certain type of stroke, the squirrels emerge from their extended naps suffering no ill effects. Now, a team of NIH-funded scientists has identified a potential drug that could grant the same resilience to the brains of ischemic stroke patients by mimicking the cellular changes that protect the brains of those animals. The study was published in The FASEB Journal, the official journal of the Foundation of American Societies for Experimental Biology.

“For decades scientists have been searching for an effective brain-protecting stroke therapy to no avail. If the compound identified in this study successfully reduces tissue death and improves recovery in further experiments, it could lead to new approaches for preserving brain cells after an ischemic stroke,” said Francesca Bosetti, Ph.D., Pharm.D., program director at the NIH’s National Institute of Neurological Disorders and Stroke (NINDS).

An ischemic stroke occurs when a clot cuts off blood flow to part of the brain, depriving those cells of oxygen and nutrients like the blood sugar glucose that they need to survive. Nearly 800,000 Americans experience a stroke every year and 87 percent of those are ischemic strokes. Read more.

Published by NIH November 17, 2017

 

Memory-enhancing drug reverses effects of traumatic brain injury in mice

Written by Ryan Cross

The radial-arm water maze is a common test to assess working memory in rodents.
Photo credit: Sciencemag.org irin717/iStock

Whether caused by a car accident that slams your head into the dashboard or repeated blows to your cranium from high-contact sports, traumatic brain injury can be permanent. There are no drugs to reverse the cognitive decline and memory loss, and any surgical interventions must be carried out within hours to be effective, according to the current medical wisdom. But a compound previously used to enhance memory in mice may offer hope: Rodents who took it up to a month after a concussion had memory capabilities similar to those that had never been injured. Read more.

Published by Science Magazine July 10, 2017

Back home with researchers, more science ahead for ‘mice-tronauts’

Written by: Sandy Mazza

The first 20 star-trekking mice to travel to the International Space Station, riding aboard a spacecraft built by Hawthorne-based Space X, have returned to their home lab at UCLA.

But the mission isn’t over for the mice, plucked last week from their capsule in San Pedro, according to a scientist participating in the project that aims to help humans battle bone loss. Read more.

Published by Daily Breeze July 8, 2017

Newly discovered brain network offers clues to social cognition

Photo credit: Kathy West – Speaking of Research

Scientists call our ability to understand another person’s thoughts—to intuit their desires, read their intentions, and predict their behavior—theory of mind. It’s an essential human trait, one that is crucial to effective social interaction. But where did it come from?

Working with rhesus macaque monkeys, researchers in Winrich Freiwald’s Laboratory of Neural Systems at The Rockefeller University have discovered tantalizing clues about the origins of our ability to understand what other people are thinking. As reported in Science on May 18, Freiwald and postdoc Julia Sliwa have identified areas in the brains of these primates that are exclusively dedicated to analyzing social interactions. And they may have evolved into the neural circuitry that supports theory of mind in the human brain. Read more.

Published by Rockefeller University May 18th, 2017

Scientists hail ‘promising cure’ for HIV after study in mice

Written by: Ian Johnston – Science Correspondent

A “promising cure” for HIV and Aids has been discovered, according to scientists who managed to almost entirely eliminate the devastating immune disease from infected mice.

The researchers said they had demonstrated the “feasibility and efficiency” of removing the HIV-1 provirus using a gene-editing technique called Crispr. Read more.

Published May 3, 2017 by The Independent

California National Primate Research Center Successfully Demonstrates Gene Editing in Nonhuman Primates

(U.C. DAVIS) – Imagine a world where maladies such as cystic fibrosis, Huntington’s Disease, or sickle cell anemia no longer exist. While the U.S. is far from achieving this lofty goal, it recently came a step closer at the California National Primate Research Center (CNPRC), where scientists have efficiently used CRISPR/Cas9 technology to modify the genes of rhesus macaque embryos.

The research, recently published in the latest edition of Human Molecular Genetics, paves the way for future studies where the possibility of birthing gene-edited monkeys that can serve as models for new therapies is greatly increased.

CRISPR, an acronym for Clustered Regularly-Interspaced Short Palindromic Repeats, is essentially a DNA segment that scientists can manipulate using a system known as CRISPR/Cas9 to edit the genes within organisms. CRISPR/Cas9 seeks and targets specific genes in organisms that are linked to diseases. It does this by utilizing a single strand of ribonucleic acid (RNA), a nucleic acid present in all living cells, as a guide to target specific genes for editing. Read more.

Published May 2, 2016 by California National Primate Research Center UC Davis

 

Children’s Hospital’s ‘artificial womb’ raises hope for extreme preemies

Written by: Tom Avril – Staff Writer

Photo credit: Children’s Hospital of Philadelphia
Premature lambs were kept alive in a bag filled with synthetic amniotic fluid, and their umbilical cords were connected to an external oxygenator.

In a major step aimed at improving the survival odds for extremely premature infants, Children’s Hospital of Philadelphia researchers have created an artificial womb — a fluid-filled “BioBag” that kept fetal lambs alive and healthy outside their mothers until they could survive on their own.
The animals received oxygen through their umbilical cords and continued to develop much as if they had remained in the uterus, leading the team to express hope that the procedure could be tried on the youngest human preemies within three to five years.

The authors of the research stressed that they were not trying to enable the delivery of babies earlier than the current limit of viability, generally 22 to 23 weeks of pregnancy. Read more.

Published April 25th, 2017 by philly.com