Sight Restored Using Stem Cells

Stem cells have an amazing potential to heal, but a challenge is ensuring the transplanted cells survive long enough to work. Long-term studies of stem cell therapies have been performed in a variety of model organisms. Notably, researchers from the Buck Institute have provided one of the first demonstrations of long-term stem cell therapy efficacy. They transplanted photoreceptors (cells for receiving light signals), derived from human stem cells, into blind mice and observed long-term vision restoration. They found that even 9 months after injection, the mice were able to perceive light signals (something they were completely incapable of prior to the treatment).

This awesome example not only shows the great potential of stem cell therapies for healing, but can help scientists further develop stem cell therapies that circumvent immune rejection. Dr. Lamba, senior author on the paper, says "That finding gives us a lot of hope for patients, that we can create some sort of advantage for these stem cell therapies so it won't be just a transient response when these cells are put in, but sustained vision for a long time."

Link to article:

https://www.sciencedaily.com/releases/2017/01/170112141243.htm

Genome Editing Trials

For the first time, the exciting gene-editing tool CRISPR-Cas9 has been used in a human. This technology, which I wrote about several weeks ago (refer to post from Nov. 5 for more detail), allows scientists to alter DNA bases to introduce a change to any gene. The concept of altering human DNA is controversial, but the potential health applications are practically limitless. For example, if a disease is caused by a specific, known mutation, scientists could potentially alter that mutation, thus treating the disease. This technology is a hot topic among biologists, and is also currently one of the most-studied tools.

Cancer is one of the main diseases that scientists hope can be treated using CRISPR-Cas9. Because of this, the first use of CRISPR-Cas9 has been in a human patient with aggressive lung cancer. Scientists of the West China Hospital in Chengdu have altered a gene that normally prevents a cell from launching an immune response, so that the immune system is more likely to target and destroy cancerous cells. The researchers hope that after injection into the patient's blood stream, the modified cells will then start attacking the cancerous cells. 

While this Chinese trial, the first of its kind, began in October of 2016, the United States also plans to start a human trial of CRISPR-Cas9 early this year. The U.S. trial is also testing the safety and efficacy of using CRISPR technology to combat several forms of cancer. Since both China and the U.S. are launching CRISPR-Cas9 trials, some healthy competition is predicted to fuel the potentially life-saving research. Carl June, a scientific adviser to the U.S. trial, says "I think this is going to trigger 'Sputnik 2.0,' a biomedical duel on progress between China and the United States, which is important since competition usually improves the end product."

To watch a video and gain more information about this Chinese trial, follow this link:

http://www.usnews.com/news/national-news/articles/2016-11-17/gene-editing-tool-crispr-cas9-used-in-a-human-for-the-first-time

Synthetic Stem Cells

Stem cell therapies have the potential to treat many different diseases and help many people. One way these therapies work is by helping damaged tissue repair itself. Still, there are risks associated with these therapies including immune system rejection, and potentially cancerous growths. Additionally, natural stem cells are fragile, and must be treated with extreme care, which slows therapies and adds another element of risk. To circumvent some of these limitations, scientists are developing procedures that use synthetic stem cells, instead of natural cells.

Scientists from North Carolina State University, the University of North Carolina at Chapel Hill, and First Affiliated Hospital of Zhengzhou University, created synthetic cardiac stem cells, and implanted them into mouse hearts that had been damaged by heart attack. Usually, the damage that heart attacks inflict upon heart muscles are never repaired by the body. Yet, when the researchers injected the synthetic stem cells, they found that the previously damaged heart muscles were effectively repaired, and this repair was comparable to when natural cardiac stem cells are implanted.

These findings are very promising for the future of stem cell research and therapies. Compared to natural stem cells, the synthetic stem cells are more stable and can be modified for use in different parts of the body. Unlike natural cells, the synthetic versions are incapable of replicating, which reduces the risk of tumor formation. And since the synthetic stem cells are designed to bypass the patient's immune system, they are far less likely to be rejected by the body's immune system. Ultimately, using synthetic stem cells instead of natural stem cells may be more affordable and accessible, and could eliminate some of the dangerous side effects associated with stem cell therapies.

To read more about the research done here, check out the following links:

http://wallstreetpit.com/112614-worlds-first-synthetic-stem-cells-implanted/

https://news.ncsu.edu/2016/12/synthetic-stem-cells/

The Skin Gun

A whole field of research revolves around harnessing the power of stem cells to treat many different injuries and ailments. Recently, CNN released an article about a skin gun that sprays a patient's healthy skin stem cells onto severe burns. The goal is to completely heal the severe burns, much faster than typical healing, using the patient's own stem cells.

Developed by a New York biotech firm called RenovaCare, this "CellMist System" requires just about a square inch of unwounded skin. To summarize the process, the patients' healthy skin is removed, the stem cells are isolated from the other skin tissue, suspended in a water-based solution, and sprayed onto the wound where the new skin hopefully begins to grow. The stem cells are not modified in any way, simply isolated and sprayed onto burn wounds.

This skin gun seems to hold much potential for healing burn wounds. While still an experimental treatment, the President and CEO of RenovaCare, Thomas Bold, says "We've seen already a couple dozen patients, and we're very happy about the results." See some results of this treatment for yourself, by watching the video at the link below.

Link to watch the video and learn more:
http://www.cnn.com/2016/12/07/health/skingun-burn-care-technologies/

Myostatin Mutations

Many people strive to be stronger. There are even those who spend hours working out daily and eat high-protein diets to sustain their muscle growth. When we see someone that is completely ripped, we may think something along the lines of "Do they even have time for anything else?" While muscle growth does typically require much effort, if genes involved in muscle growth are mutated in a specific way, then muscles can be altered without a shred of intentional effort.

       Such is the case in Belgian Blue cattle (image below). These cattle have been selectively bred for many years since muscle mass in cattle often means more meat and money for breeders. For a long time, the more muscular bulls and cows were chosen for breeding, and this resulted in more muscular offspring. What breeders didn't necessarily understand, was that they were selecting mutations in the myostatin gene. This gene codes for the protein myostatin which inhibits muscle development. The mutated, shorter form of myostatin doesn't function properly, which results in more rapid muscle growth. This creates "double-muscled" offspring that, instead of having the normal amount of muscle fibers at birth, actually have twice as many muscle fibers. The myostatin mutation is considered a permanent muscle mutation within this cattle breed.

But myostatin has the same muscle-inhibiting function in other mammals. Mutations of myostatin have even been found in humans. Several cases of children born with these mutations have been documented, but have only recently been understood (image of boy with a myostatin mutation below). Additionally, myostatin mutations have been induced in mice to create "mighty mice" by Se-Jin Lee and colleagues at Johns Hopkins. Below, there is an image of a normal, wild-type mouse that lacks any myostatin mutation and a mouse with mutated myostatin. You can clearly see that the mutated mouse has much more muscle mass than the normal mouse. The discovery of myostatin mutations and their effect on increased muscle mass has practical applications. These mutations are currently being studied to potentially help people with muscular dystrophies (conditions with weakening muscles).

Link to primary article about Myostatin mutation:

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC24998/

Can you Ear me now?

Doctors in China are growing a new right ear for a man who lost his in a car accident. Not in a petri dish in a lab, but on the man's own arm! Dr. Guo Shuzhong, a reconstructive surgery specialist, is leading a team of doctors in a 3-part plan. In the first phase, the skin was expanded on the patient's arm using injections of water. In the second phase, some of the patient's own cartilage, shaped into the form of an ear, was placed into the arm to grow. In the third and final phase, the doctors plan to transplant the new ear onto the patient's head.

This isn't the first case of ears being grown on arms. An Australian artist by the name of Stelarc elected to grow a third ear on his arm for the sake of art. Although the ear isn't yet used for hearing, he plans to pursue further operations to install a Wi-Fi enabled microphone so that people can "tune in" to his station and listen to whatever his ear is hearing (image of Stelarc and his ear below).

Link to recent reconstructive ear story:
http://www.foxnews.com/health/2016/11/10/doctors-grow-man-new-ear-on-arm.html

Link to video of this reconstructive process:
http://www.wsj.com/video/chinese-doctors-grow-new-ear-on-man-arm/37480AB0-9361-4354-B75E-6C7CB273FED2.html

Link to 2015 Stelarc story:
http://www.cnn.com/2015/08/13/arts/stelarc-ear-arm-art/

Thanksgiving, Wine, and Bluebirds

Thanksgiving has come and gone, and we all likely feasted on turkey or ham, stuffing, potatoes, pumpkin pie...and maybe a glass or four of wine. Since wine is so often enjoyed, and wine-making can be very profitable, many researchers out there are studying what makes wine better. Still, before grapes are even collected, they are obviously grown in vineyards. A recent study looked into how vineyards may benefit from other animals in the environment: bluebirds.

Unfortunately, trees that house bluebirds are frequently cut down to make room for expanding vineyards. This study examined whether putting up birdhouses (for the bluebirds to live in) is beneficial to vineyard owners. To do this, they collected fecal samples from many bluebirds in Napa Valley, and analyzed the poop for DNA fragments (a process they called "molecular scatology"). The researchers used readily available databases to match the DNA fragments they found in the bluebird poop to the DNA of known species. In other words, they figured out what the birds had been eating.

When they compiled their findings, the researchers saw that bluebirds generally eat a lot of herbivorous insects (mosquitoes and other species), and only about 3% of their diets are from predatory or useful insects. From the standpoint of winemakers, this is a really good thing! It means that the bluebirds are consuming the insects that could potentially eat or damage their crops. Also, we didn't know that bluebirds eat mosquitoes (insects that are not just annoying, but also can spread harmful viruses). These findings might encourage winemakers to install more birdhouses, which would benefit the birds, the winemakers, and all of us happy consumers of wine.

Check out this link to learn more:
http://www.popsci.com/bluebird-poop-proves-their-value-to-california-vineyards

We Are What We Eat

A very recent study by scientists at the University of Oxford reveals an interesting link between organisms and what they eat. To build DNA, an organism must have access to all the right components. If an organism has a restricted diet, and has limited DNA components, then it would make sense that their DNA would be different from an organism that did not have this limited diet. This was the scientists' hypothesis: the composition of food could alter their DNA.

To test their hypothesis, they examined similar groups of parasites that shared a common ancestor but have evolved to eat different food. The researchers developed mathematical models to make DNA comparisons and found that parasites with low-nitrogen diets had DNA sequences composed of less nitrogen than parasites that ate nitrogen-rich food. Interestingly, they also found that it's possible to predict diets of related organisms by comparing their DNA. The authors believe their findings shed light on the mystery of how two highly related organisms can have very different DNA, and also recognize that there are many other factors that influence the DNA of organisms.

Link to review article: https://www.sciencedaily.com/releases/2016/11/161115111720.htm

Primary Reference: 

1. Emily A. Seward, Steven Kelly. Dietary nitrogen alters codon bias and genome composition in parasitic microorganisms. Genome Biology, 2016; 17 (1) DOI: 10.1186/s13059-016-1087-9

Squirrels with Leprosy

Leprosy is a historical disease that has been around for thousands of years. In medieval England, leprosy was a fact of life, with hundreds of care facilities on the outskirts of towns. Although not life-threatening, leprosy is caused by a bacterial infection and visibly damages skin and nerves, making the disease one of deformity rather than death. While rates of leprosy in humans drastically decreased long ago, leprosy has recently been discovered in squirrels in the UK.

Bishop instructing clerics that have leprosy

A paper published on the 10th announced that two strains of leprosy-causing bacteria have been discovered in a red squirrel population in the UK. Previously, only humans and armadillos had been found susceptible to leprosy. One strain found in the squirrels is highly related to the strain that infected people in medieval Europe. Scientists aren't exactly sure how the squirrels became infected with leprosy, but they believe the disease may have been passed between squirrels and humans. This is similar to the few cases of leprosy in the southern U.S. that were transmitted to humans from armadillos. Yet, there is no serious need to fear becoming infected with leprosy, since the disease is now very treatable with antibiotics, and the bacterial strains carrying the disease are quite fragile and often die within a few hours after removal from their hosts.

Squirrel showing signs of leprosy on its ear and muzzle

Link to review article: http://www.popsci.com/medieval-strain-leprosy-found-in-red-squirrels-in-uk

Does This Tickle?

Aristotle was puzzled by an interesting fact: Why can't humans tickle themselves? We have all experienced being tickled at some point or other, but do we understand how tickling works? This is something that some scientists are striving to understand, but not only because of simple curiosity. One tell-tale sign of Schizophrenia is the ability to tickle oneself. Also, tickling is linked to our ability to laugh, play, and simply feel good, so researching how tickling works adds to our understanding of positive and negative emotions, even depression.

Neuroscientists performed an experiment in which they tickled rats and studied their physical and neural responses. They found that the rats learned to enjoy the tickling and even came to think of the hand doing the tickling as their "playmate." The rats displayed a universal expression of positive emotion called "joy jumps" that are also seen in human children, dogs, foxes, and guinea pigs (to name just a few). To record neural activity, the scientists inserted electrodes into the somatosensory cortex of the rats. Cells in this area of the brain increased firing while the rats were being tickled, but also after the tickling as the rats chased the hand and "giggled." Interestingly, when the researchers applied an electrical current to these same cells, this stimulated the rats to behave as if they were being tickled (giggling and jumping playfully). This is important evidence that shows these cells are responsible for ticklishness.

As you might already know from experience, ticklishness also depends on mood. If we are in good moods we are more likely to laugh when being tickled and when we are in bad moods we are likely to complain when we are tickled. They also tested this on the rats by exposing nocturnal rats to a bright light (making them unhappy or anxious) and observing their responses to being tickled. They found the rats were much less ticklish and did not display the signs of playfulness that they did in the previous experiment. Also, the cells in that somatosensory cortex were suppressed, adding evidence to the finding that these cells are required for the typical tickle response. The researchers also came to the conclusion that our brains must form hard-wired connections early in life for the tickling sensation to be learned, and even potentially enjoyed. If we don't experience tickling when we are young, then we are much less likely to enjoy tickling later in life.

Link to video and review of this research:
http://www.sciencemag.org/news/2016/11/watch-these-ticklish-rats-laugh-and-jump-joy

Brain-Spinal Interface: Bypassing Injuries

An international group of scientists collaborated to develop a brain-spinal interface that is able to bypass spinal cord injuries to restore intentional walking in a paralyzed leg. Typically, neurons in the brain work with the spinal cord in order to make walking possible. Electrical signals originating in the motor cortex of the brain travel down to the lower spinal cord where they activate motor neurons that signal muscles to extend and flex the leg. An injury in the spine can cut off this communication. To help regain this communication, a pill-sized brain chip was made and put in the brains of paralyzed rhesus monkeys where it recorded signals from the motor cortex. These signals were then sent to a computer for decoding. The decoded neural messages were wirelessly sent to an electrical spinal stimulator implanted below the area of injury. This signaled the spinal nerves to perform locomotion and the paralyzed monkeys were able to walk on a treadmill. The many scientists who played a part in developing this brain-spinal interface are very excited by this progress but stress the importance of further research before this type of interface can be used in humans.

Review: https://www.sciencedaily.com/releases/2016/11/161109133133.htm

Primary study: http://www.nature.com/nature/journal/v539/n7628/full/nature20118.html

Growing Hearts for Transplants

Many people are on waiting lists for organ transplants. Unfortunately, many people will not receive the organ they desperately need. For example, there are about 4,000 people in the U.S. awaiting heart transplants and only 2,500 of these will receive a new heart within a year. Additionally, a serious problem with transplants is the possibility of organ rejection (when the body's immune system doesn't recognize the new organ and instead fights the foreign cells).

Now imagine if human hearts could actually be grown, not taken from an organ donor, but actually grown by scientists in a lab. This would potentially provide many sick patients with the heart they need to survive and wouldn't otherwise receive. This concept is not science fiction. Scientists have been working on growing organs (ears, lungs, bladders, windpipes etc.) that are specifically designed for their recipient. The process works by using a scaffold of the particular organ (like a template) and seeding the scaffold with stem cells derived from the patient (building upon the template). This means that the organ will not be rejected by the patient's immune system since it was formed from his or her own cells. 

Recently, scientists from Massachusetts General Hospital and Harvard Medical School have grown full-sized, beating human hearts using stem cells. First, they take skin cells from the adult patient and genetically reprogram them to make them into stem cells (called Induced Pluripotent Stem Cells or IPS cells). Then, a donor heart that is deemed unfit for transplantation is washed with a detergent and many of its cells are stripped away, leaving the heart scaffold. At this point, the patient's reprogrammed stem cells are applied to the scaffold where they take hold, grow, and divide. After about 2 weeks in a nutrient-rich solution, the heart looks like a developing human heart and even beats when given an electrical signal! 

While not quite ready for transplantation into patients, this research provides much hope to those that need a heart transplant. Researchers are now trying to increase the number of IPS cells they can create, as well as speed-up the cell maturation process. 

http://www.popsci.com/scientists-grow-transplantable-hearts-with-stem-cells

Images: Stripped human heart (left) and heart grown using a scaffold and IPS cells (right)