"Gene Therapy Will Cure You, or Your Money Back!"

For the first time ever, doctors in Italy are offering commercial gene therapy to people inflicted with rare diseases. The procedures come with a hefty price tag, but are guaranteed to work - or your money back!

The treatment is called Strimvelis, and it is the first outright cure for the autoimmune disease ADA-SCID, that leaves newborn babies with almost no defense against viruses, bacteria, or fungi, and is often fatal. This same disease was documented in the 1976 movie Bubble Boy about an American child who lived his life inside a plastic bubble.

Strimvelis uses a virus to deploy a missing gene into the bone marrow of afflicted children, allowing the stem cells within the marrow to naturally produce the proper immune defenses for life. It is considered a “repair and replace” strategy because doctors extract stem cells from bone marrow, then soak the cells with viruses to transfer a correct copy of the ADA gene.

Strimvelis was initially developed in Milan, Itlay at the San Raffaele Telethon Institute for Gene Therapy, but pharmaceutical giant GlaxoSmithKline purchased the rights to the procedure in 2010. The price tag for the one-time treatment will be $665,000. This makes it one of the most expensive single procedures in all of medicine, but is considered pennies compared to the cost of the established method of treating the disease.

GSK sells $30 billion is drug each year, so revenue from the treatment is not the bottom line. Rather, the British company hopes to master gene therapy technology, including virus manufacturing. Sven Kili, the executive of gene therapy development at GSK stated, "If we can first make products that change lives, then we can develop them into things that affect more people. We believe gene therapy is an area of important future growth; we don’t want to rush or cut corners."

Read more: https://www.technologyreview.com/s/602113/gene-therapy-cure-has-money-back-guarantee/

https://www.technologyreview.com/s/601390/gene-therapys-first-out-and-out-cure-is-here/

Furthering Stem Cells

As stem cells develope they turn into different cells. Scientist have begun the process of looking into what makes the stem cells turn into such a wide range of different cells in the body. A new study shows that a protein called OCT4 plays a vital role in determing what branch of cells the stem cell will ultimately become. This could help scientists to find a way to make specific cells out of stem cells and that could lead to the ability to fight new deseases and rehab new injuries that we couldn't rehab before. The sky is the limit for stem cells as most scientists belive they have a lot of potential in aiding the fight against new problems that we could not address before.























Picture from https://www.sciencedaily.com/releases/2016/08/160804140503.htm

New Bio-Ink Allows for 3D Printing of Living Tissue

A new stem cell-laden bio-ink created by scientists at the University of Bristol allows for the 3D printing of living tissue, or bioprinting. This development could lead to the printing of complex surgical bones and cartilage implants using a patient’s own stem cells in the future.

An artist’s impression of a 3D living tissue bioprinting. (Image courtesy of University of Bristol)

The bioink is composed of two different polymers: a natural extract from seaweed, and a synthetic polymer already in use in medical surgeries. The synthetic polymer converts from a liquid to a gel at exactly 37°C, while the seaweed polymer provides structural support once cell nutrients are absorbed. Amazingly, once cell nutrients are introduced, the synthetic polymer dissolves away, while the stem cells and seaweed polymer remain. The stem cells in the ink are osteoblasts (a cell that secretes the bone material) and chondrocytes (a cell that secretes the matrix of cartilage).

Dr. Adam Perriman, lead researcher from the School of Cellular and Molecular Medicine at Bristol, stated:

Designing the new bio-ink was extremely challenging as it required a material that is printable and durable enough to maintain its shape if immersed in nutrients and does no harm to the cells. There was a lot of trial and error tests before they were able to formulate a working method.

External Link: http://www.explainingthefuture.com/bioprinting.html 

Hepatocytes Engineered from Stem Cells

Scientists have discovered a cost efficient way to study hepatocytes in Jerusalem. They have figured out a way to create their own liver cells from human embryonic and genetic engineered stem cells. Before this, the only way to obtain accurately functioning hepatocytes was from donated organs. Although, hepatocytes have been created in a laboratory setting before, this is the first time the cells have shown functional ability for clinical analysis. Since the liver plays such a big role in breaking down drugs and other substances, it is usually the primary organ to be injured from drug abuse. The newly engineered hepatocytes can detect the effect of pharmaceutical toxins with a staggering 97% accuracy. Pharmaceutical companies also benefit from this discovery, as it is a cheaper way to study liver function. Also, it gives us access to an unlimited supply of hepatocytes and research. I find it interesting that scientists can improve research and possibly cure many diseases with the simple construction of cells. Just like the supply of hepatocytes, the possibility of further groundbreaking research in healthcare is endless. 

The human liver. 

Engineering Organs

 What if we could build organs from a single cell?  This form of stem cell research has been ongoing for years.  With the successful development of a new tissue “scaffold” technology, this may finally become a reality.  The Universities of Bristol and Liverpool led the research into the development of this scaffold, which is a combination of cells that produce living tissue that scientists hope can replace diseased parts of the body.  One roadblock the team faced was the oxygenation of cells in the center of the tissue, which decreased as the overall dimensions increased.  A new team of researchers found a possible solution to this oxygen deficiency problem by synthesizing myoglobin, an oxygen-carrying protein, and attaching it to the membrane of the stem cells.  These were used as a reservoir of oxygen to the cells.  Dr. Perriman from Bristol’s School of Cellular and Molecular Medicine compared the proteins to a scuba tank; the cells “use it to breathe from when there is not enough oxygen in the local environment.” 

The research opened new opportunities not only in creating new cartilage, but muscle and bone as well.  This new protein development opens up “a wide range of biotechnologies,” and is therefore a large step in the right direction for stem cell research.  The long-term goal, of course, is to be able to provide transplants of healthy tissue into patients with diseased or damaged tissue. 

Lab Creates Cells Used by Brain to Control Muscle Cells

For the first time, the University of Central Florida researchers used stem cells to grow neuromuscular junctions between human muscle cells and human spinal cord cells. These are the key connectors used by the brain for communication and muscle control.
This discovery is a huge step in developing "human-on-a-chip" systems. These systems are models that can recreate how organs and series of organ functions in the body. This could possibly accelerate drug testing, medical research, and life-saving breakthroughs.
A UCF bioengineer, James Hickman, said that these types of systems need to be developed if you ever want to recreate human function. He is the one who led this breakthrough. Hickman is eager about this breakthrough because some federal agencies have granted at least $140 million to grant funding to jump start "human-on-a-chip" research. Right now, scientists rely on animal systems for medical research, but this allows for a pure human system.
http://www.biologynews.net/

Researchers complete first genome mapping of DNA modification

U.S. researchers have completed the first genome-wide mapping of a DNA modification code 5-hydroxymethylcytosine (5hmC) in embryonic stem cells.

The molecule is predominantly found in genes that are turned on, or active, according to the researchers from the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at University of California, Los Angeles.

The finding may prove to be important in controlling diseases like cancer, where the regulation of certain genes plays a role in disease development. Human embryonic stem cells were used because it had been shown previously that the molecule is abundant in those cells, as well as in brain cells.

In the study, the researchers found that 5hmC was associated with genes and tended to be found on genes that were active. The study also revealed that 5hmC was present on a type of DNA regulatory element, called enhancers, which help control gene expression. In particular, 5hmC was present on enhancers that are crucial for defining the nature of the human embryonic stem cells.The results suggest that 5hmC plays a role in the activation of genes. This is opposite of the role of the more well studied 5mC ( DNA methylation), which is involved in silencing genes. his relationship is in line with the view that 5hmC is created directly from 5mC.

If we can understand the function of 5hmC, that will lead to greater understanding of how genes are turned on and off and that could lead to the development of methods for controlling gene regulation, therefore helping control cancer and other human diseases.

5hmC is formed from the DNA base cytosine by adding a methyl group and then a hydroxy group. The molecule is important in epigenetics - the study of changes in gene expression caused by mechanisms other than changes in the DNA sequence - because the newly formed hydroxymethyl group on the cytosine can potentially switch a gene on and off. The molecule 5hmC was only recently discovered, and its function has not been clearly understood, Until now, researchers didn't know where 5hmC was located within the genome.

The study appears in the July issue of the journal Genome Biology.

Thymosin Beta 4 May Help Heal a "Broken Heart"

Studies show that a natural protein called thymosin ß4 (Tß4) can activate stem cells in mouse hearts to replace damaged tissue with new muscle cells. Damage to the heart muscle cells called cardiomyocytes, are usually irreparable. Scientists from University College London Institute of Child Health knew that stem cells expressing the gene Wt1 could become cardiomyocytes. So they injected mice with the protein tß4 every day for a week. They also stitched one of the animal’s arteries together, to mimic a heart attack. The mice do survive, so that it is possible to study the effect the protein has on the gene expression.

Just two days after the operation, the cells expressing the gene Wt1 where on the outer layer of the heart. By two weeks the cells moved inside of the heart, around the injury, and looked just like cardiomyocytes. The scientists believe the protein may have a chemical effect on the stem cell’s DNA that effect the gene expression. The protein in a sense “wakes up” the gene WT1 in stem cells.

Studies on thymosin ß4 injections on humans have already passed safety trials. I think this is excellent find; it will surely help us treat people who are prone to heart disease or are currently having heart problems. Further studies and clinical trials will truly show if this is an effective way to treat heart disease.

Stem cell trachea

According to an article on Cnn.com the first ever trachea made from stem cells has been implanted into a human being. There is a man in Stockholm who was diagnosed with late stage tracheal cancer who received the implanted windpipe on June 9th. The patient is doing well and two days after the surgery he already has a cough reflex. The same doctor who completed this surgery also did something similar three years ago. He implanted a windpipe in a woman that was engineered with her own stem cells after her lung had collapsed from tuberculosis.

I know that this subject is a major controversy in today's world. My opinion of it is that as long as we can help people that's what counts. If there is a way that we can heal someone or save someones life, why not give it a try. I understand how other people feel though because some see it as "playing God" but their view point may change if it was one of their family members in trouble.

Stem Cells Show Promising Results in Treatment of Parkinson's Disease

Research that is being conducted at Hayang University and Harvard Medical School have shown promising results in the reversal of the disease in a rat model.

Parkinson's disease is a degenerative disease which results in loss of nerve cells. The current treatments available to patients provide only relief from symptoms but cannot reverse the progression of the disease. It is believed these stem cells will have the ability to regenerate and repair diseased tissue.

This research shows so much promise in the treatment of Parkinson's disease and possibly many other diseases that can potentially be treated with stem cells.

Researchers Discover Human Lung Stem Cell

Researchers at Brigham and Women's Hospital have discovered a true human lung stem cell. The human lung stem cell is self-renewing and capable of forming multiple biological structures within the lung.

"These are critical first steps in developing clinical treatment for those with lung diseases for which no therapies exist. Further research is needed, but we are excited about the impact this discovery could have on our ability to regenerate or recreate new lung tissues to replace damaged areas of the lungs," said Joseph Loscalzo, M.D. Phd., chair of the Department of Medicine at BWH and co-author.

As someone who has lost two grandfathers and a father to lung cancer these findings seem promising for the treatment of lung cancer in the future. In the past a diagnosis of lung cancer was a death sentence.