Wade Watcher

A Perryville, Missouri, family is organizing a bone-marrow registration drive in hopes of finding a match for their 6-year-old son, who needs a bone-marrow transplant.

Wade Watcher's mother Jenni said for the most part, he's a regular 6-year-old.

"Active and funny and adorable," she said. "He's smart and loves to draw. He likes playing basketball. He's a pretty awesome kid."

But for him to continue leading a normal childhood, Watcher likely would need a bone-marrow transplant.

"We knew that he had a rare disease when he was a baby, and so yearly we have to get a bone marrow biopsy to see if his bone marrow is failing," Watcher said. "It had been fairly normal until December. ... It showed his bone marrow was in the stages of failing and that it was kind of like a waiting game to see if he needs to be sent for a bone marrow transplant or not."

Wade, who suffers from Shwachman-Diamond syndrome, a rare congenital disorder, is stable, but his mother said they don't know for how long.

So they're organizing a registration drive for members of the community to sign up to have their cheeks swabbed and see whether they may be a match. The drive will be from 3 to 7 p.m. Friday at the AMVETS Post 94 in Perryville.

Watcher said she's not sure how many people are scheduled to participate, but she to register as many people ranging in age from 18 to 55 years old people as possible.

"Anybody that can would be amazing," she said. "It would provide a lot of help for our family as well as other families."

Registration involves filling out a form and having a cheek swabbed for about 30 seconds. Donor recruitment coordinator Olivia Haddox said people typically shy away from such drives because they are unsure of what it may mean if they are "matched" with a person in need.

"People are surprised to find how easy it is just to register, but then the next question is always, 'What's going to happen if I get that call?'" she said. "We definitely get that a lot."

There are two ways for the donation to happen if a match is found, she said. About 80 percent of the time, donations are done via peripheral blood stem-cell donation, a four- to eight-hour session in which blood is taken from one arm and filtered through an aphoresis machine to separate the blood from the stem cells. After taking the stem cells, the blood is returned to the donor's body.

"That can kind of be compared to a lengthier platelet or plasma donation," Haddox said. "You don't actually even lose any blood that day; you just lose some stem cells, and you regenerate those in about a week, so what you give you do get back," she said.

People usually watch Netflix while donating, she said, and minor side effects more often come from the series of injections donors receive before the procedure to boost the stem cells. Those injections can cause some fatigue or other side effects.

"Nothing so severe that it might keep anyone out of work," Haddox said. "It's just kind of your body preparing for the donation."

The other, less-common method is an outpatient procedure whereby liquid marrow from the lower back pelvic area is removed.

"And you're actually put under for this procedure, so you're not awake when it happens and you don't feel anything when it happens," Haddox said. "Afterwards, what most people tell me they feel is just a tenderness and a bruising around the site where they removed the marrow. A lot of people equate this to saying, 'I felt like I fell on some ice, and I had a bruise on my hip for a few days.'"

If people can't attend the drive, swab kits can be ordered at dkms.org.

tgraef@semissourian.com

(573) 388-3627

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Perryville family organizing bone-marrow drive Friday for ailing 6-year-old boy - Southeast Missourian

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GETTY STOCK IMAGE

The way to the Red Planet and other mysterious worlds is being inspired by the villainous Khan from the blockbuster films, according to new research.

The use of stem cell technology may mean the difference between life and death on any attempt to travel beyond Earth into the wilderness of space.

So the first person to walk on Mars is likely to be selected from the growing group of people whose parents took the step to store their child's stem cells at birth.

Stem cells are 'blank' cells that can be reprogrammed to turn into any other cell in the body, enabling the replacement of damaged cells.

More and more British parents, including TV presenter Natalie Pinkham and dancer Darcey Bussell, are paying more than 2,000 to freeze samples from their babies' umbilical cords at birth.

Stem cells are also found in bone marrow and some body tissue, but the procedure to harvest them from umbilical cords is less risky.

Adventurous Mars pioneers will have to be especially prepared for the dangerous trip, which could expose them to cancer and other diseases, through carefully researched gene therapy.

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We wince at the thought of genetically engineered humans

Mark Hall

Mark Hall, spokesperson for the UK's leading stem cell storage and diagnostics company StemProtect, said: "We wince at the thought of genetically engineered humans.

"And we are not going to create a Khan from Star Trek specifically to get to another planet. Getting humans to Mars and beyond will be both expensive and dangerous.

"But the scientific by-products - such as huge leaps in stem cell medicine - will benefit humanity for centuries to come."

Genetic engineering has featured in two Star Trek movies, and a number of TV episodes.

IG

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This still image strikes an uncanny resemblance to a figure of a woman

Khan, who appeared in Space Seed and Star Trek II: The Wrath of Khan, was modified to make him stronger and to give him greater stamina and intellectual capacity than a regular human.

Mr Hall said: "The first human to walk on Mars may not even be born yet - but that's an advantage."

StemProtect believes advanced medical techniques will be required to cope with the rigours of interplanetary space.

While a trip to Mars may appear "just around the corner" in galactic terms, it is highly possible exposure to radiation along the way could lead to the astronauts developing leukaemia and other cancers even before they arrived.

GETTY STOCK IMAGE

This means future travellers will have to be 'immunised' before they leave Earth.

Mr Hall said: "There was an article in The Times suggesting elephants would make ideal Martian travellers because they'd be largely immune to the radiation.

"But those laughing at the ridiculous sounding headline completely missed the point - the fact is scientists are already working on ways of getting humans there and back alive."

Recent research has shown radiation in deep space increase the risk of leukaemia while long term exposure to micro gravity may leave astronauts open to infection.

The three year round trip to Mars would affect humans at the stem cell level, leaving them with a drastically lowered immune system, NASA funded scientists say.

And NASA's own findings say stem cells may be crucial to the future of space travel, particularly how they respond in a low gravity environment.

One study showed stem cells flown in space and then cultured back on Earth had greater ability to self renew and generate any cell type, changing more easily into specialised heart muscle cells, for instance.

Mr Hall said an astronaut will have to be prepared for the journey "quite literally at the stem cell level."

He explained: "That means working with the best and most effective stem cells available to the patient - those harvested from the umbilical cord at birth."

GETTY STOCK IMAGE

The therapies required to 'immunise' humans to space travel are still being researched.

And with most space based science, it can only mean huge benefits to mankind back down on Earth when it comes to fighting otherwise deadly conditions and diseases.

Stem cells have the ability to treat a potentially infinite range of illnesses and diseases.

Stem cell therapy is already being used all over the world to treat some cancers and stroke victims - and there is fast progress being made in many other areas, including Parkinson's and Alzheimer's disease.

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SUPERHUMANS: Mars 'will be colonised by genetically engineered Star Trek-style beings' - Express.co.uk

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Using human skin cells, University of California, Irvine neurobiologists and their colleagues have created a method to generate one of the principle cell types of the brain called microglia, which play a key role in preserving the function of neural networks and responding to injury and disease.

The finding marks an important step in the use of induced pluripotent stem (iPS) cells for targeted approaches to better understand and potentially treat neurological diseases such as Alzheimer's. These iPS cells are derived from existing adult skin cells and show increasing utility as a promising approach for studying human disease and developing new therapies.

Skin cells were donated from patients at the UCI Alzheimer's Disease Research Center. The study, led by Edsel Abud, Wayne Poon and Mathew Blurton Jones of UCI, used a genetic process to reprogram these cells into a pluripotent state capable of developing into any type of cell or tissue of the body.

The researchers then guided these pluripotent cells to a new state by exposing the cells to a series of differentiation factors which mimicked the developmental origin of microglia. The resulting cells act very much like human microglial cells. Their study appears in the current issue of Neuron.

In the brain, microglia mediate inflammation and the removal of dead cells and debris. These cells make up 10- to 15-percent of brain cells and are needed for the development and maintenance of neural networks.

"Microglia play an important role in Alzheimer's and other diseases of the central nervous system. Recent research has revealed that newly discovered Alzheimer's-risk genes influence microglia behavior. Using these cells, we can understand the biology of these genes and test potential new therapies," said Blurton-Jones, an assistant professor of the Department of Neurobiology & Behavior and Director of the ADRC iPS Core.

"Scientists have had to rely on mouse microglia to study the immunology of AD. This discovery provides a powerful new approach to better model human disease and develop new therapies," added Poon, a UCI MIND associate researcher.

Along those lines, the researchers examined the genetic and physical interactions between Alzheimer's disease pathology and iPS-microglia. They are now using these cells in three-dimensional brain models to understand how microglia interact with other brain cells and influence AD and the development of other neurological diseases.

"Our findings provide a renewable and high-throughput method for understanding the role of inflammation in Alzheimer's disease using human cells," said Abud, an M.D./Ph.D. student. "These translational studies will better inform disease-modulating therapeutic strategies."

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Materials provided by University of California - Irvine. Note: Content may be edited for style and length.

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Skin stem cells used to generate new brain cells: Study to advance ... - Science Daily

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From Skin to Brain

The brain is one of the most vital organs in the human body, so damage to the brain from injury or aging can have major impacts on peoples quality of life.Neurological disorders representsome of todays most devastating medical conditions that are also difficult to treat.Among these is Alzheimers disease.

Usually, research involving Alzheimers rely on brain cells from mice. Now, neurobiologists from the University of California, Irvine (UCI) have developed a method that could allow the use of human cells instead of animal ones to help understand neurological diseases better.

In their study, which was published in the journal Neuron, the researchers found a way to transform human skin cells into stem cells and program them into microglial cells. The latter make up about 10 to 15 percent of the brain and are involved in the removing dead cells and debris, as well as managing inflammation. Micgrolia are instramentalin neural network development and maintenance, explained researcher Mathew Blurton Jones, fromUCIs Department of Neurobiology & Behavior.

Microglia play an important role in Alzheimers and other diseases of the central nervous system. Recent research has revealed that newly discovered Alzheimers-risk genes influence microglia behavior, Jones said in an interview for a UCI press release. Using these cells, we can understand the biology of these genes and test potential new therapies.

The skin cells had been donated by patients from UCIs Alzheimers Disease Research Center. These were firstsubjected toa genetic process to convert them into induced pluripotent stem (iPS) cells adult cells modified to behave as an embryonic stem cell, allowing them to become other kinds of cells. These iPS cells were then exposed to differentiation factors designed to imitate the environment of developing microglia, which transformed them into the brain cells.

This discovery provides a powerful new approach to better model human disease and develop new therapies, said UCI MIND associate researcher Wayne Poon in the press release. The researchers, in effect, have developed a renewable and high-throughput method for understanding the role of inflammation in Alzheimers disease using human cells, according to researcher Edsel Abud in the same source.

In other words, by using human microglia instead of those from mice, the researchers have developed a more accurate toolto study neurological diseases and to develop more targeted treatment approaches. In the case of Alzheimers, they studied the genetic and physical interactions between the diseases pathology and the induced microglia cells. These translational studies will better inform disease-modulating therapeutic strategies, Abud added in the press release.

Furthermore, they are now using these induced microglia cells in three-dimensional brain models. The goal is to understand the interaction between microglia and other brain cells, and how these influence the development of Alzheimers and other neurological diseases.

This is all made possible by reprogrammable stem cells. Indeed, this study is one more example of how stem cells arechanging medicine.

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A New Technique Transforms Human Skin Into Brain Cells - Futurism

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Women at the highest genetic risk for bone fractures benefit the most from hormone therapy, according to a University at Buffalo study of 10,000 participants in the national Women's Health Initiative.

As women age, their bone mineral density decreases, leaving them at greater risk of breaking bones from falls. Some women are more genetically prone to fractures. The researchers said their study is believed to be the first to investigate gene-hormone therapy interaction on fractures in postmenopausal white women.

We found that women who are genetically at the highest fracture risk can enjoy the greatest protection from fracture when they use hormone therapy, Heather Ochs-Balcom, a UB associate professor of epidemiology and environmental health who led the research team, said in a statement.

The findings were published online in the Journal of Clinical Endocrinology and Metabolism. The papers first author, Youjin Wang, conducted the research as a doctoral candidate in epidemiology and environmental health at UB.

Researchers looked at a subset of 9,922 women among the more than 27,000 who had participated in Women's Health Initiative hormone therapy clinical trials. The federally funded Initiative began in 1991 and consisted of a set of studies looking at health issues in more than 161,000 postmenopausal women.

Further studies on gene-therapy interaction are needed to evaluate the advantages of targeted treatments based on genetic profile, the researchers said.

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UB study suggests reduced fracture risk with hormone therapy in ... - Buffalo News

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By Jill U. Adams By Jill U. Adams April 24 at 8:38 AM

For many women this college graduation season, the primary reason to see a doctor soon after graduation may be to get birth control.

They may want to stick with whatever theyve been using, whether thats the pill or the patch or the vaginal ring. Or they may want to consider a broad menu of options that vary with regard to ease of use, side effects and duration of protection.

The most popular kind of birth control on campus is condoms, with 61percent of womenreporting that they used that method the last time they had sex. The pill came in a close second, with 58 percent, followed by withdrawal at 33 percent, according to a 2016 report by the American College Health Association from a survey of 80,129 undergraduate students. (Survey respondents often reported more than one method used.)

And yet, these commonly used contraceptive methods have failure rates that maygive one pause. For 100 women over the course of a year, there would be ninepregnancies with the pill, 18 with condom use and 22 with the withdrawal method.

The top two birth control options in terms of effectiveness are intrauterine devices (IUDs) and progestin implants. With these methods, the failure rate is less than one pregnancy per 100 women in a year.

These two methods have another advantage for users, which earns them the moniker long-acting reversible contraceptives, or LARCs. After a one-time procedure, women are protected from getting pregnant for at least three years or up to 10 years, depending on the product.

There are fewer or milder side effects with these long-acting methods, compared with birth control pills. Copper IUDs such as Paragard can increase menstrual pain and flow, especially in the first year of use. Hormone-releasing IUDs, such as Mirena and Skyla, can cause spotting or irregular bleeding, especially in the first six months of use.

The hormones released by IUDs stay locally in the uterus, says Kristyn Brandi, an OB/GYNat Boston University. So you dont get the same side effects as taking the pill, such as changes in mood and breast tenderness, she says.

A birth control implant can cause spotting throughout the monthly cycle. And its slowly released hormone distributes through the whole body, so hormonal side effects can occur, but less so than the pill,Brandi says.

With implants and hormonal IUDs, often menstrual periods become much lighter and in some women disappear altogether a side effect that many view as a benefit.

Why dont more young women use these long-acting, super-effective methods? In that survey of college students, IUDs were reported to be used by 9percent of females and implants by 6 percent.

One reason is lingering myths about their safety in young women. Its a myth that you cant have an IUD if you havent had a child, says Krishna Upadhya, a Johns Hopkins pediatrician who specializes in adolescent health.

Older versions of IUDs were thought to be too large for some young women, but thats no longer a concern, says Joanne Brown, a nurse practitioner at the University of Kentuckys health service. The newer IUDs are very small.

Another reason more young women dont use IUDs or implants is access, particularly on campus. Whereas 98percent of campus health services provide birth control pills, only 40percent provide the implant or IUDs. It can depend on the size of a college, how many providers or what level of services they have, says Brown, who works with the American College Health Association on sexual health issues.

Implants and IUDs require a procedure, not just a consultation and a prescription.

Cost can be a barrier, as well. The Affordable Care Actrequired health insurers to cover birth control, but that doesnt mean that every plan covers every birth control method. Getting an IUD can cost several hundred dollars and as high as $1,000, including a medical exam and insertion.

Even if youre paying some of the cost, IUDs are the most cost-effective birth control method, Brandi says. The non-hormonal IUD Paragard is good for 10years and cost-wise beats paying $20 per month for birth control pills.

A relatively new IUD called Liletta is made by a nonprofit company with the aim of making them cost-friendly. It costs $50 for a clinic to use, Brandi says.

Birth control implants, which last three years, are generally cheaper than IUDs, at a couple of hundred dollars, but can run as high as $800, including insertion.

Upadhya, who sees patients up to age 25, says she helps them explore all the options-- not just effectiveness and side effects, but how a particular option fits in with their lives. Comfort level can play a role, she says. The pill is the thing that everyone has heard of. People are very comfortable with the idea of it.

As Brandi puts it: The most effective form of birth control is the one people practice. Some people are good pill takers.

The bottom line is there are a lot of options: the LARCs, the pill, the patch, the ring and the shot. Even if youve had a problem with other kinds of birth control, Brandi advises, talk to you doctor; shell help you figure out how to find something that will work.

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Long-term birth control is the most reliable. So why do so few young women use it? - Washington Post

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San Francisco Business Times

At high-stakes table, growing Peninsula biotech antes up with 'fatty-liver' drug San Francisco Business Times This South San Francisco biotech has 150 employees, has raised $300 million and has an important partnership with a Big Pharma company. But it is the one drug that is not part of that deal that could have a big impact on a growing disease.

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At high-stakes table, growing Peninsula biotech antes up with 'fatty-liver' drug - San Francisco Business Times

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The objective of this study is to investigate the impact of metabolic status on associations of serum vitamin D with hypogonadism and lower urinary tract symptoms (LUTS)/benign prostatic hyperplasia (BPH).

A total of 612 men underwent physical examination, biochemical/hormonal blood testing, and transrectal prostate ultrasound. Moreover, the subjects filled out standard questionnaires for identification and grading of LUTS and hypogonadism symptoms. Parameters were statistically compared with independent t-tests and correlation analyses.

Vitamin D levels positively correlated with total testosterone (TT) but not with prostate volume or International Prostate Symptom Score (IPSS). Patients with metabolic syndrome had significantly lower vitamin D levels, which were not correlated with TT, prostate volume, or IPSS. However, vitamin D was positively correlated with TT, and negatively correlated with prostate volume and quality-of-life IPSS in subjects without metabolic syndrome.

The clinical usefulness of vitamin D for treatment of hypogonadism or LUTS/BPH varies according to metabolic status.

The aging male : the official journal of the International Society for the Study of the Aging Male. 2017 Apr 17 [Epub ahead of print]

Sun Gu Park, Jeong Kyun Yeo, Dae Yeon Cho, Min Gu Park

a Department of Preventive Medicine , Gachon University College of Medicine , Incheon , Korea., b Department of Urology , Inje University Seoul Paik Hospital , Seoul , Korea., c Department of Urology , Inje University, Sanggye Paik Hospital , Seoul , Korea.

PubMed http://www.ncbi.nlm.nih.gov/pubmed/28414251

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It was Thursday afternoon, and the little girl from near Cologne, Germany, and the 40-year-old Duluth woman had known each other for less than 24 hours. But it was obvious that Edwards already had bonded with Ina and her little sister Mila.

They were together because the girls' mother had given Edwards a much greater gift: the gift of life.

"By your donation, I still get to be a mom," Edwards told Daniela Halfkann, 30. "(You're) a mom, so you completely understand how important it is to be here with your children."

Edwards, the mother of 15-year-old twin boys and the wife of Duluth Fire Chief Dennis Edwards, is alive because of the stem cell transplant she received at the Mayo Clinic on Oct. 31, 2014. As a result, she said, she is in remission from the rare and aggressive form of leukemia with which she had been diagnosed that June.

All she was told at the time of the transplant was that the donor was a woman from Germany.

Halfkann had registered as a potential stem cell or bone marrow donor at the large insurance company where she works in Cologne, she said. One day she received a call, saying her donation was needed.

After the six-hour procedure, Halfkann was told nothing more than that the recipient was a woman in the United States.

After a two-year waiting period required in Germany, the two women learned each other's identities last October and connected via Facebook.

Their meeting in Duluth was arranged by Amanda Schamper, Midwest donor recruitment coordinator for DKMS, the international organization that facilitated the donation.

Halfkann made the trip along with husband Stefan and their daughters, leaving their home at 3 a.m. on Tuesday and arriving at the Duluth International Airport at 5 p.m. on Wednesday.

Like Edwards, DKMS wants to raise awareness of the need for people to enter the registry, said Schamper, who also traveled to Duluth for the occasion.

She said 14,000 patients are in need of a peripheral blood stem cell or bone marrow donation, but fewer than half will get one because there's no match on the registry.

"We're looking for a particular protein in our DNA," she explained.

Only in 30 percent of cases are siblings a match. Edwards' brother and sister both had been screened, she said, and neither was a match for her.

Finding a match "is equated to finding your genetic twin, or winning the genetic lottery," Schamper said.

If more people were on the registry a process that only requires taking a swab from your cheek there would be more potential matches. But only 2 percent of eligible Americans are registered, Schamper said.

When the Halfkanns arrived at the gate on Wednesday, Dennis and Merissa Edwards, along with sons Caden and Jaxon, were waiting at the gate.

It was an emotional moment.

"It was hard for me," Merissa Edwards said on Friday, speaking to Daniela Halfkann. "I was crying. I was so emotional, so happy to meet you and hug you."

She wiped away a tear. "I still am."

"It was amazing," Halfkann responded. "I cried at the gate, too."

The Halfkanns, who are staying at the Edgewater, initially focused on recovery from jet lag. But Edwards is making sure they'll get a full taste of Duluth and Minnesota before beginning their return trip to Germany next Saturday. That includes visits to the Mall of America, the Great Lakes Aquarium and a trip up the North Shore.

A "thank-you party," open to the public, is planned on Sunday afternoon. Halfkann also will be recognized on Monday during the Saints Sports Awards ceremony at the College of St. Scholastica, where Edwards is an administrative assistant in the athletics department.

Recovery from the ravages of leukemia has been a long process, Edwards said, but she remains in remission. She gets a PET scan every six months to make sure that's still the case; the next one takes place next week.

Edwards shares her story, she said, not to call attention to herself but to highlight the need for people to take the simple step of registering as a potential donor.

"It's so important for us to help other people keep their families together and save a mother or father or son or daughter," she said. "The more people we can encourage to cheek-swab and get on the registry, the more lives we can help save and help families stay together."

TO LEARN MORE

For more information and to learn how to get on the bone marrow and peripheral blood stem cell registry, visit dkms.org.

IF YOU GO

The thank-you party for Daniela Halfkann will be from 2 to 5 p.m. on Sunday at The Other Place Bar and Grill, 3930 E. Calvary Road.

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Duluth woman meets the German donor whose stem cells saved her ... - Duluth News Tribune

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Newswise Irvine, Calif., April 24, 2017 Using human skin cells, University of California, Irvine neurobiologists and their colleagues have created a method to generate one of the principle cell types of the brain called microglia, which play a key role in preserving the function of neural networks and responding to injury and disease.

The finding marks an important step in the use of induced pluripotent stem (iPS) cells for targeted approaches to better understand and potentially treat neurological diseases such as Alzheimers. These iPS cells are derived from existing adult skin cells and show increasing utility as a promising approach for studying human disease and developing new therapies.

Skin cells were donated from patients at the UCI Alzheimers Disease Research Center. The study, led by Edsel Abud, Wayne Poon and Mathew Blurton Jones of UCI, used a genetic process to reprogram these cells into a pluripotent state capable of developing into any type of cell or tissue of the body.

The researchers then guided these pluripotent cells to a new state by exposing the cells to a series of differentiation factors which mimicked the developmental origin of microglia. The resulting cells act very much like human microglial cells. Their study appears in the current issue of Neuron. Link: http://www.cell.com/neuron/fulltext/S0896-6273(17)30286-6.

In the brain, microglia mediate inflammation and the removal of dead cells and debris. These cells make up 10- to 15-percent of brain cells and are needed for the development and maintenance of neural networks.

Microglia play an important role in Alzheimers and other diseases of the central nervous system. Recent research has revealed that newly discovered Alzheimers-risk genes influence microglia behavior. Using these cells, we can understand the biology of these genes and test potential new therapies, said Blurton-Jones, an assistant professor of the Department of Neurobiology & Behavior and Director of the ADRC iPS Core.

Scientists have had to rely on mouse microglia to study the immunology of AD. This discovery provides a powerful new approach to better model human disease and develop new therapies, added Poon, a UCI MIND associate researcher.

Along those lines, the researchers examined the genetic and physical interactions between Alzheimers disease pathology and iPS-microglia. They are now using these cells in three-dimensional brain models to understand how microglia interact with other brain cells and influence AD and the development of other neurological diseases.

Our findings provide a renewable and high-throughput method for understanding the role of inflammation in Alzheimers disease using human cells, said Abud, an M.D./Ph.D. student. These translational studies will better inform disease-modulating therapeutic strategies.

Blurton Jones, Abud and Poon are with UCIs Institute for Memory Impairments and Neurological Disorders (UCI MIND). Ricardo Ramirez, Eric Martinez, Cecilia Nguyen, Sean Newman, Vanessa Scarfone, Samuel E. Marsh, Cristhian Fimbres, Chad A. Caraway, Ali Mortazavi, Michael Cahalan, Brian Cummings, Gianna Fote, Andriy Yeromin and Anshu Agrawal with UCI; Luke Healy and Jack Antel with McGill University, Montreal; Rakez Kayed with the University of Texas Medical Branch, Galveston, Texas; Karen Gylys with UCLA; and Abdullah Madany and Monica Carson with UC Riverside contributed to the study. The National Institutes of Health, the California Institute for Regenerative Medicine, and the Susan Scott Foundation provided support.

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Skin Stem Cells Used to Generate New Brain Cells - Newswise (press release)

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Stem cells help researchers identify neuronal defects causing ... Science Daily Researchers have used stem cells derived from patients with Angelman syndrome to identify the underlying neuronal defects that cause the rare neurogenetic ... and more »

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Stem cells help researchers identify neuronal defects causing ... - Science Daily

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"Sangamo once again has a very strong presence at ASGCT, with 19 oral and poster presentations," said Dr. Sandy Macrae, Sangamo's chief executive officer. "These data highlight the breadth of our clinical and early stage pipeline across genome editing, gene therapy, gene regulation and cell therapy. With our focus now on the translation of our groundbreaking science into new genomic therapies that transform patients' lives, our research and technology programs will continue to provide new assets for therapeutic development."

The following presentations are scheduled at the ASGCT Meeting sessions:

Invited Presentations at Scientific Symposia

Lysosomal Storage Disorders

Central Nervous System Disorders

Monogenic and Infectious Diseases

Technology and Delivery Developments

Applications of Gene Editing in Stem Cells

All abstracts for the ASGCT meeting are available online at 2017 ASGCT Annual Meeting Abstracts.

About Sangamo Therapeutics Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients' lives using the company's industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company is advancing Phase 1/2 clinical programs in hemophilia A and hemophilia B, and lysosomal storage disorders MPS I and MPS II. Sangamo has a strategic collaboration with Bioverativ Inc. for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington's disease. In addition, it has established strategic partnerships with companies in non-therapeutic applications of its technology, including Sigma-Aldrich Corporation and Dow AgroSciences. For more information about Sangamo, visit the Company's website at http://www.sangamo.com.

Forward Looking StatementsThis press release contains forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, references relating to presentation of data from various therapeutic and research programs and the potential of these programs to transform the lives of patients. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the dependence on the success of clinical trials of lead programs, the lengthy and uncertain regulatory approval process, uncertainties related to the timing of initiation and completion of clinical trials, whether clinical trial results will validate and support the safety and efficacy of ZFP Therapeutics, and the ability to establish strategic partnerships. Further, there can be no assurance that the necessary regulatory approvals will be obtained or that Sangamo and its partners will be able to develop commercially viable gene-based therapeutics. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business environments. These risks and uncertainties are described more fully in Sangamo's Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q as filed with the Securities and Exchange Commission. Forward-looking statements contained in this announcement are made as of this date, and Sangamo undertakes no duty to update such information except as required under applicable law.

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/sangamo-therapeutics-announces-presentations-at-2017-annual-meeting-of-the-american-society-of-gene--cell-therapy-300444609.html

SOURCE Sangamo Therapeutics, Inc.

http://www.sangamo.com

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Sangamo Therapeutics Announces Presentations at 2017 Annual meeting of the American Society of Gene & Cell ... - PR Newswire (press release)

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BERKELEY, Calif., April 24, 2017 (GLOBE NEWSWIRE) -- XOMA Corporation (XOMA), a pioneer in the discovery and development of therapeutic antibodies, today announced that it has achieved positive Phase 2 proof-of-concept results for X213 in physiological hyperprolactinemia (HPRL). X213 is a monoclonal antibody that neutralizes prolactin action.

This proof-of-concept study was an important milestone in demonstrating the potential efficacy of this novel antibody. We believe that X213 could be a treatment option for a wide range of patients with hyperprolactinemia including prolactinoma and anti-psychotic induced HPRL as the signs and symptoms are similar irrespective of the etiology, said Jim Neal, Chief Executive Officer of XOMA. Consistent with our business strategy, we intend to maximize the value of X213 for shareholders by seeking a license partner for the program.

The Phase 2 study was a multi-center, open-labelled, randomized, single-dose, controlled trial of intravenously administered X213 in women who wished to suppress lactation immediately post-partum. The results of the study indicate that X213, when given as a single 700mg intravenous infusion during the first day post-partum, was effective in suppressing milk secretion, as well as breast engorgement and pain in 100 percent of the treated women. In addition, none of the treated women experienced rebound breast symptomatology during the 21-day study period. While the study was not intended, or powered to show statistical significance, it demonstrated that X213 was: safe and well tolerated; caused no significant adverse events (SAEs); showed favorable pharmacokinetics with a terminal half-life of two weeks and; demonstrated target (prolactin receptor) engagement and mechanism of action confirmation by serum prolactin profiling.

The findings from this proof-of-concept study are encouraging and confirm that X213 inhibits prolactin signaling and thus, may be effective in blocking effects of symptomatic hyperprolactinemia, said Dr. Shlomo Melmed, endocrinologist, Dean of the Medical Faculty and Professor of Medicine, Cedars-Sinai Medical Center, Los Angeles. New classes of drugs such as X213 may offer benefit to the up to 20 percent of patients who do not respond to, or are intolerant of, current standard of care involving dopamine agonist medications.

Prolactin is a multifunctional hormone that is primarily secreted by the pituitary and whose best-known functions are related to lactation and reproduction. In pregnant women, excess prolactin secretion (hyperprolactinemia) occurs to enhance breast development and to induce lactation postpartum. Commonly encountered etiologies of hyperprolactinemia include prolactinoma, medication effect, kidney failure, cystic or granulomatous pituitary lesions, and disorders which interfere with hypothalamic inhibition of prolactin release. Prolactinomas, benign tumors of the pituitary gland, hypersecrete prolactin with significant medical consequences, particularly hypogonadism, infertility and osteoporosis.

About X213 X213 (formerly LFA 102) is a first-in-class allosteric inhibitor of prolactin action. It is a humanized IgG1-Kappa monoclonal antibody that binds to the extracellular domain of the human prolactin receptor with high affinity at an allosteric site. The antibody has been shown to inhibit prolactin-mediated signaling, and it is potent and similarly active against animal and human prolactin receptors.

The Phase 2 study evaluated the safety, tolerability, pharmacokinetics and pharmacodynamics of X213 in post-partum women. The study was conducted at multiple medical centers in Spain.

About XOMA Corporation XOMA has an extensive portfolio of products, programs, and technologies that are the subject of licenses the Company has in place with other biotech and pharmaceutical companies. Many of these licenses are the result of the Company's pioneering efforts in the discovery and development of antibody therapeutics. There are more than 20 such programs that are fully funded by partners and could produce milestone payments and royalty payments in the future. In order to maximize its value in a licensing transaction, XOMA continues to invest in X358, an allosteric monoclonal antibody that reduces insulin receptor activity, as the antibody could have a major impact on the treatment of hyperinsulinism. For more information, visit http://www.xoma.com.

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Forward-Looking Statements Certain statements contained in this press release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, including statements regarding: X213 as a potential treatment option for patients with hyperprolactinemia including prolactinoma and anti-psychotic induced HPRL; XOMA's portfolio of partnered programs and licensed technologies; XOMA's intent to license X213 and X358; and statements that otherwise relate to future periods. These statements are based on assumptions that may not prove accurate, and actual results could differ materially from those anticipated due to certain risks inherent in the biotechnology industry and for companies engaged in the development of new products in a regulated market. Potential risks to XOMA meeting these expectations are described in more detail in XOMA's most recent filing on Form 10-K and in other SEC filings. Consider such risks carefully when considering XOMA's prospects. Any forward-looking statement in this press release represents XOMA's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. XOMA disclaims any obligation to update any forward-looking statement, except as required by applicable law.

Continued here:
XOMA Announces Positive Results from its Phase 2 Proof-Of ... - Yahoo Finance

Recommendation and review posted by sam

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A Lincoln student is set to save someone's life after finding out he was a match for someone who needed a bone marrow transplant.

John-Paul Dickie, the vice-president of academic affairs at the University of Lincoln, said he joined the register after his flatmate told him about it.

He doesn't know the identity of the person whose life he saved, but said he was delighted that he's been able to help someone who was desperate for a transplant.

He said: "My flatmate was involved with Lincoln Marrow, a student-led group trying to sign people up to the British Bone Marrow Register. He was telling me the benefits of it, including the fact it could potentially save someone's life.

READ MORE: Selfless mum marks 50th blood donation with daughter's first

"I signed up in February 2014, so it was a surprise when I heard back earlier this year that I was a potential match. I had some samples taken and eventually I had a date set for the operation in May.

"I'm looking forward to it, as it's an amazing way to help somebody.

"However I'm also a bit hesitant as it will require me to be strapped to a machine for four or five hours. My partner will be there to keep me company and I'll have books and TV to stop me from getting too bored."

When bone marrow is damaged it prevents a person from creating healthy blood cells and transplants like this help to treat the condition.

The transplant requires taking stem cells from the blood or bone marrow of one person and giving them to another.

John-Paul added: "There are two ways to take stem cells. One is taking them out of your back using a needle, which is painful but only 10 per cent of people have. Fortunately, I'm having the more common method in which blood is taken out of one arm, the stem cells are removed and then it is returned in the other arm."

READ MORE: 'Gordon was denied stem cell treatment, but I'll hold him in my heart forever'

Most people who need stem cells will be a match with a close family member. However, if this doesn't work then they will have to wait on the British Bone Marrow Registry.

"It's a great way to contribute and help save someone's life. All you have to do is give a sample of spit to get on the register, the process is so simple and easy. If you're able to do it, I would definitely encourage you to give it a try.

"The procedure is anonymous in case something goes wrong. You can find out their age and sex, but at the moment I don't know anything. After two years, you can apply to find out who they are."

Bone marrow donors need to be aged between 17 and 40 and already registered as a blood donor.

If you meet these criteria interested in signing up to the British Bone Marrow Register, visit their website for more information at: http://www.nhsbt.nhs.uk/bonemarrow/

The rest is here:
'It's amazing!' Student discovers he's a potential life-saving bone marrow match - Lincolnshire Echo

Recommendation and review posted by simmons

The biggest national project to treat patients with cerebral palsy (CP) through injection of stems cells from umbilical cord blood (UCB) into the brain began its trial run in March. The project is jointly undertaken by Royan Institute, Childrens Medical Center (affiliated to Tehran University of Medical Sciences), and the Iran Blood Transfusion Organization (IBTO). In the first phase, it will provide treatment to 130 children with CP between the ages 5-13. The treatment was tested in September 2016 on children with CP in some hospitals, and the results were highly satisfactory. Approximately four in every 1,000 children in Iran have CP while in the developed countries the rate is 2 to 2.5 per 1000 live births. Cerebral palsy is an umbrella term for the effects of damage to a developing brain by various causes. It is connected with a range of symptoms, including muscle weakness and movement problems. The damage to the brain usually occurs early on in its development, either in the baby during pregnancy or during the period soon after birth. Symptoms may include difficulties in walking, balance and motor control, eating, swallowing, speech or coordination of eye movements. Some people affected by CP also have some level of intellectual disability. No two people with cerebral palsy are affected in exactly the same way. The IBTO plans to expand the storage of stems cells from umbilical cord blood to 100,000 samples from the current 80,000, said Ali Akbar Pourfathollah, head of the organization, ILNA reported. Around 75,000 samples have been stored in private banks and 5,000 in public banks, but the number will surpass 100,000 soon, he added.

Valuable Source for Treatment Umbilical blood is a valuable source of hematopoietic stem cells which can be used for treatment of many malignant diseases such as leukemia. Hundreds of transplants have been performed using stem cells from such blood, which is easy and risk free. The use of stem cells reduces the risk of viral diseases transmission and incidence of Graft Versus Host Disease (GVHD). The ability to perform organ transplants is among the benefits of umbilical cord blood transfusion. Using stems cells is also one of the best ways to treat blood diseases since the method has a success rate of 70% worldwide. Storage of stem cells is a valuable investment. So far, 27 cord blood banks have been launched across the country. There are two types: public and private banks for stem cell storage. The former does not charge a fee for storage. But in the latter, the cost of collection and genetic testing is about $645 and the annual charge for storage is $33, according to ISNA. Pourfathollah said the IBTO is looking to store stem cells in medical cases when a patient needs to receive treatment from matched unrelated donors. In the past Iranian year (ended March 20), out of the 8,000 stem cell transplantations in the country, only 100 were from matched unrelated donors and the rest came from sibling (or related) donors. IBTO is also looking to set up coagulation/transfusion and HLA/immunogenetics laboratories in the country on par with international standards. The Immunogenetics and HLA Laboratory provides human leukocyte antigen (HLA) typing, HLA antibody identification and post-transplant engraftment monitoring services. These tests are required for patients undergoing evaluation for organ transplantation, recipients of bone marrow/stem cell transplants, patients requiring platelet transfusions from HLA-matched donors, and patients undergoing evaluation of particular health conditions.

Read more:
Treating Cerebral Palsy With UCB Stem Cells - Financial Tribune

Recommendation and review posted by Bethany Smith

The end of 2016 brought with it a spike in classic dystopian book sales. George Orwells Nineteen Eighty-Four and Margaret Atwoods The Handmaids Tale which will be released as a Hulu show this month eachpiqued the interest of book buyers, who mightve drawn uncomfortable parallels between the stories and the world around them.

These books, of course, are not the only dystopian titles resonating with readers. The science fiction subgenre has enjoyed a long period of popularity thanks to YA installments like The Hunger Games, Divergent, The Maze Runner and The 100, each with its own onscreen offshoot.

There are those in the sci-fi genre who are tired of dystopias proliferation; there are, after all, many ways to speculate about the future, and not all of them need be pessimistic. Still, as the subgenre grows, its capacity for holding a mirror to todays problems climate change, stringent definitions of gender, and discrimination based on race or gender or nationality persists.

If you still see the worth in dystopian stories for social change or for entertainment value there are, luckily, loads to choose from. Climate-fiction, or cli-fi, has emerged as a sub-subgenre of dystopian fiction, with authors like Lidia Yuknavitch and Jeff VanderMeer both of whom have upcoming film adaptations leading the charge. Other titles explore cryonics, religion, gender and more.

Weve included a few were excited about below. Just note that our definition of dystopia is a broad one; any vision of the future that could go awry qualifies.

Knopf

1. American War by Omar El Akkad

Fought amid a changing climate, Americas second Civil War lasting nearly 20 years was fought with homicide bombings and drones. An academic born during this period remembers the story of a girl who lived through it.

47north

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In a town outside of Estiel what was once St. Louis a girl named Etta fulfills her duties as a forager, but must venture to face a tyrant called Lion when women from her community are kidnapped.

HarperCollins

Lidia Yukanavitch is skilled at writing poetically about the human body, and about nature, so this book her first foray into science fiction makes sense. Its a retelling of the story of Joan of Arc, but in a world ravaged by radiation, and with few land-based survivors.

FSG

Rachel and Wick live in a city destroyed by drought and terrorized by a giant bear, doing what they can to prioritize their survival until Rachel finds Borne, a plant-animal shes immediately attached to.

Orbit

When two coders go missing, an entire future society is at risk. Robinsons work may not be squarely dystopian, but he has a knack for drawing imagined worlds and their societal problems. In his latest,rising tides leave New York partly submerged.

FSG

If the future of the ever-growing tech industry has a physical home, its San Fransisco, where Masons novel is set. Life extension, artificial memory and rising waters converge in a sprawling future epic.

Torcom

Kir has been asked to join a project working towards the possibility of humans inhabiting another planet a project designed to give Earthlings, living on a planet thats overcrowded and climate change-wrecked, a chance at survival. Will her brain wired for optimism be able to heed the warnings of the artificial intelligence she hosts?

Small Beer Press

Sofia Samatars stories are more fantasy than sci-fi, and shes more likely to chronicle an alternate or parallel reality than a possible future. Her story How to Get Back to the Forest earned a spot among the Best American Science Fiction and Fantasy Stories 2015.

Weidenfeld Nicolson

Lallas father plans to escape the increasingly dangerous world of future-Britain via ship, but the boat turns out to be eerily different than expected.

Dutton Books

What if the world were living in now was the dystopian version of some happier, more progressive alternate reality?Thats the premise of Elan Mastais debut, which is centered around protagonist Tom, who has to make a tough choice between a thrumming, messy world or a neat and perfect one.

Harper Voyager

On the surface, Elianas life is a pleasant one. She lives on an idyllic island where she works as a weaver, but she is forced to hide the fact that shes capable of dreaming, lest she be cast out. The cracks in her perfect world begin to show when a young girl washes up on the shore, bearing a tattoo of Elianas name.

Scribner

Jeffs father, Ross, has always been somewhat absent from his life; hes a billionaire and hes happily remarried. But when Rosss second wife Artis gets sick, he invites his son to visit him at a mysterious cryogenics facility, where pseudo-science meets spiritual practice.

Tor Books

Patricias a witch. Laurence is a tech wunderkind. Their star-crossed relationship is a love story for the 21st century, where spirituality and intuition are at odds with scientific advancements.

Bloomsbury

Eddie and Lauras suburban life devolves amid an ecological disaster, one that forces them each to reconsider what it is that they cherish most.

Amethyst Editions

The world, it turns out, is ending. That doesnt stop Michelle from dating, from writing, from relocating to a new city to distance herself from her drug-addled past, or from proceeding more or less as normal, except that now, the apocalypse looms.

Two Dollar Radio

For Ellingsen, the personal is political. His storys hero, Brandon, retreats to the wilderness after his professor and lover makes him commit an act of violence. From there, he fosters hope for a future threatened by rising temperatures and the attendant damage done to the environment.

Tor Books

Near-future sci-fi may be all the rage; it would seem that its more capable of shedding new light on present dangers, anyway. But Palmers novel set in the 25th century, when societys perceptions of gender and religion have morphed considerably gives those stories a run for their money.

Originally posted here:
17 Spine-Tingling New Books For Fans Of Dystopia - Huffington Post

Recommendation and review posted by sam

Confocal micrograph of mouse retina depicting optic fiber layer. Image courtesy of National Center for Microscopy and Imaging Research, UC San Diego.

Using the gene-editing tool CRISPR/Cas9, researchers at University of California San Diego School of Medicine and Shiley Eye Institute at UC San Diego Health, with colleagues in China, have reprogrammed mutated rod photoreceptors to become functioning cone photoreceptors, reversing cellular degeneration and restoring visual function in two mouse models of retinitis pigmentosa.

The findings are published in the April 21 advance online issue of Cell Research.

Retinitis pigmentosa (RP) is a group of inherited vision disorders caused by numerous mutations in more than 60 genes. The mutations affect the eyes photoreceptors, specialized cells in the retina that sense and convert light images into electrical signals sent to the brain. There are two types: rod cells that function for night vision and peripheral vision, and cone cells that provide central vision (visual acuity) and discern color. The human retina typically contains 120 million rod cells and 6 million cone cells.

In RP, which affects approximately 100,000 Americans and 1 in 4,000 persons worldwide, rod-specific genetic mutations cause rod photoreceptor cells to dysfunction and degenerate over time. Initial symptoms are loss of peripheral and night vision, followed by diminished visual acuity and color perception as cone cells also begin to fail and die. There is no treatment for RP. The eventual result may be legal blindness.

In their published research, a team led by senior author Kang Zhang, MD, PhD, chief of ophthalmic genetics, founding director of the Institute for Genomic Medicine and co-director of biomaterials and tissue engineering at the Institute of Engineering in Medicine, both at UC San Diego School of Medicine, used CRISPR/Cas9 to deactivate a master switch gene called Nrl and a downstream transcription factor called Nr2e3.

CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, allows researchers to target specific stretches of genetic code and edit DNA at precise locations, modifying select gene functions. Deactivating either Nrl or Nr2e3 reprogrammed rod cells to become cone cells.

Cone cells are less vulnerable to the genetic mutations that cause RP, said Zhang. Our strategy was to use gene therapy to make the underlying mutations irrelevant, resulting in the preservation of tissue and vision.

The scientists tested their approach in two different mouse models of RP. In both cases, they found an abundance of reprogrammed cone cells and preserved cellular architecture in the retinas. Electroretinography testing of rod and cone receptors in live mice show improved function.

Zhang said a recent independent study led by Zhijian Wu, PhD, at National Eye Institute, part of the National Institutes of Health, also reached similar conclusions.

The researchers used adeno-associated virus (AAV) to perform the gene therapy, which they said should help advance their work to human clinical trials quicker. AAV is a common cold virus and has been used in many successful gene therapy treatments with a relatively good safely profile, said Zhang. Human clinical trials could be planned soon after completion of preclinical study. There is no treatment for RP so the need is great and pressing. In addition, our approach of reprogramming mutation-sensitive cells to mutation-resistant cells may have broader application to other human diseases, including cancer.

This article has been republished frommaterialsprovided by University of California - San Diego. Note: material may have been edited for length and content. For further information, please contact the cited source.

Reference

Zhu, J., Ming, C., Fu, X., Duan, Y., Hoang, D. A., Rutgard, J., . . . Zhang, K. (2017). Gene and mutation independent therapy via CRISPR-Cas9 mediated cellular reprogramming in rod photoreceptors. Cell Research. doi:10.1038/cr.2017.57

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Reversing Gene Damage to Treat Blindness | Technology Networks - Technology Networks

Recommendation and review posted by simmons

BUFFALO, N.Y. Women at the highest genetic risk for fracture benefit the most from hormone therapy, according to a first-of-its-kind study led by researchers at the University at Buffalo.

The study included nearly 10,000 participants from the Womens Health Initiative (WHI), a national, long-term study of more than 150,000 women.

We found that women who are genetically at the highest fracture risk can enjoy the greatest protection from fracture when they use hormone therapy, said Heather Ochs-Balcom, associate professor of epidemiology and environmental health in UBs School of Public Health and Health Professions, who led the research team.

The findings were published online ahead of print in the Journal of Clinical Endocrinology and Metabolism. The papers first author, Youjin Wang, conducted the research as a doctoral candidate in epidemiology and environmental health at UB.

This study provides a better understanding of who can benefit the most in terms of bone health from hormone therapy use, Ochs-Balcom said, adding that the results have implications for personalized medicine. Its important information as women and their doctors make decisions about hormone therapy use.

The study, believed to be the first to investigate gene-hormone therapy interaction on fracture in postmenopausal white women, utilizes the largest set of known genes linked to fracture risk from a meta-analysis of genome-wide association studies.

Researchers looked at a subset of 9,922 women from among the more than 27,000 who had participated in WHI hormone therapy clinical trials. They wondered whether women who are more genetically susceptible to fractures could benefit from hormone therapy.

This is important because, as previous WHI studies have identified, there are risks and benefits with hormone therapy, Ochs-Balcom said. This is where precision or personalized medicine comes in the attempt to get the right drugs to the right person to ensure the most benefit and least harm.

As women age, their bone mineral density (BMD) decreases, leaving them at greater risk of breaking bones from falling, which also increases as they age. But some women also are more genetically prone to fractures. Our study represents a first look at how inherited predisposition to fracture is related to hormone therapy use, said Ochs-Balcom, who also holds a faculty appointment in the program in Genetics, Genomics and Bioinformatics in UBs Jacobs School of Medicine and Biomedical Sciences.

Wang notes that further studies on gene-therapy interaction are warranted to evaluate the advantages of targeted interventions based on genetic profile. The research team is currently analyzing other gene-environment interactions and recently published another paper on the association of calcium plus vitamin D supplementation and genetic risk of fracture.

In addition to Wang and Ochs-Balcom, other UB co-authors include Jean Wactawski-Wende, dean of the School of Public Health and Health Professions and SUNY Distinguished Professor and professor of epidemiology and environmental health; and Leah Preus, Kathleen Hovey and Jing Nie from the School of Public Health and Health Professions.

Additional co-authors include Lara Sucheston-Campbell, Rebecca Jackson and Samuel Handelman, The Ohio State University; Rami Nassir, University of California, Davis; and Carolyn Crandall, University of California, Los Angeles.

The Womens Health Initiative began in 1991 and consisted of a set of clinical trials and an observational study. Combined, they included more than 161,000 generally healthy postmenopausal women aged 50 to 79.

One of 40 WHI centers nationwide, the University at Buffalo serves as the Northeast Regional Center, managing data collection and scientific coordination among nine WHI-affiliated institutions in the mid-Atlantic and Northeast regions. UB has received WHI extension funding to conduct follow-up studies on the original participants, many of whom are now between ages 67 and 100.

The WHI is funded by the National Heart, Lung, and Blood Institute of the National Institutes of Health.

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Genetics are key to hormone therapy lowering risk of broken bones in older women - UB News Center

Recommendation and review posted by simmons

Science Daily

Using CRISPR to reverse retinitis pigmentosa and restore visual ... Science Daily Using the gene-editing tool CRISPR/Cas9, researchers have reprogrammed mutated rod photoreceptors to become functioning cone photoreceptors, reversing ... and more »

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Using CRISPR to reverse retinitis pigmentosa and restore visual ... - Science Daily

Recommendation and review posted by Bethany Smith

It was Thursday afternoon, and the little girl from near Cologne, Germany, and the 40-year-old Duluth woman had known each other for less than 24 hours. But it was obvious that Edwards already had bonded with Ina and her little sister Mila.

They were together because the girls' mother had given Edwards a much greater gift: the gift of life.

"By your donation, I still get to be a mom," Edwards told Daniela Halfkann, 30. "(You're) a mom, so you completely understand how important it is to be here with your children."

Edwards, the mother of 15-year-old twin boys and the wife of Duluth Fire Chief Dennis Edwards, is alive because of the stem cell transplant she received at the Mayo Clinic on Oct. 31, 2014. As a result, she said, she is in remission from the rare and aggressive form of leukemia with which she had been diagnosed that June.

All she was told at the time of the transplant was that the donor was a woman from Germany.

Halfkann had registered as a potential stem cell or bone marrow donor at the large insurance company where she works in Cologne, she said. One day she received a call, saying her donation was needed.

After the six-hour procedure, Halfkann was told nothing more than that the recipient was a woman in the United States.

After a two-year waiting period required in Germany, the two women learned each other's identities last October and connected via Facebook.

Their meeting in Duluth was arranged by Amanda Schamper, Midwest donor recruitment coordinator for DKMS, the Germany-based organization that facilitated the donation.

Halfkann made the trip along with husband Stefan and their daughters, leaving their home at 3 a.m. on Tuesday and arriving at the Duluth International Airport at 5 p.m. on Wednesday.

Like Edwards, DKMS wants to raise awareness of the need for people to enter the registry, said Schamper, who also traveled to Duluth for the occasion.

She said 14,000 patients are in need of a peripheral blood stem cell or bone marrow donation, but fewer than half will get one because there's no match on the registry.

"We're looking for a particular protein in our DNA," she explained.

Only in 30 percent of cases are siblings a match. Edwards' brother and sister both had been screened, she said, and neither was a match for her.

Finding a match "is equated to finding your genetic twin, or winning the genetic lottery," Schamper said.

If more people were on the registry a process that only requires taking a swab from your cheek there would be more potential matches. But only 2 percent of eligible Americans are registered, Schamper said.

When the Halfkanns arrived at the gate on Wednesday, Dennis and Merissa Edwards, along with sons Caden and Jaxon, were waiting at the gate.

It was an emotional moment.

"It was hard for me," Merissa Edwards said on Friday, speaking to Daniela Halfkann. "I was crying. I was so emotional, so happy to meet you and hug you."

She wiped away a tear. "I still am."

"It was amazing," Halfkann responded. "I cried at the gate, too."

The Halfkanns, who are staying at the Edgewater, initially focused on recovery from jet lag. But Edwards is making sure they'll get a full taste of Duluth and Minnesota before beginning their return trip to Germany next Saturday. That includes visits to the Mall of America, the Great Lakes Aquarium and a trip up the North Shore.

A "thank-you party," open to the public, is planned on Sunday afternoon. Halfkann also will be recognized on Monday during the Saints Sports Awards ceremony at the College of St. Scholastica, where Edwards is an administrative assistant in the athletics department.

Recovery from the ravages of leukemia has been a long process, Edwards said, but she remains in remission. She gets a PET scan every six months to make sure that's still the case; the next one takes place next week.

Edwards shares her story, she said, not to call attention to herself but to highlight the need for people to take the simple step of registering as a potential donor.

"It's so important for us to help other people keep their families together and save a mother or father or son or daughter," she said. "The more people we can encourage to cheek-swab and get on the registry, the more lives we can help save and help families stay together."

TO LEARN MORE

For more information and to learn how to get on the bone marrow and peripheral blood stem cell registry, visit dkms.org.

IF YOU GO

The thank-you party for Daniela Halfkann will be from 2 to 5 p.m. on Sunday at The Other Place Bar and Grill, 3930 E. Calvary Road.

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Duluth woman meets the German donor whose stem cells saved her life - WDAY

Recommendation and review posted by Bethany Smith

Organ transplant rejection might eventually be preventable by giving recipients an immune-suppressing vaccine derived from induced pluripotent stem cells, according to a study led by Japanese researchers.

In mice, the treatment allowed permanent acceptance of heart grafts by selectively inhibiting the immune response to the donor graft, said the study, published April 20 in Stem Cell Reports. The work might also be applicable to autoimmune diseases, the study said.

The study can be found at j.mp/ipscden. The co-first authors were Songjie Cai, Jiangang Hou, and Masayuki Fujino. The senior author was Xiao-Kang Li. All are of the National Research Institute for Child Health and Development in Tokyo.

The IPS cells were matured into donor-type regulatory dendritic cells (DCregs) which in turn caused production of tolerance-inducing regulatory T cells, or Tregs, that allow the graft to be treated as self.

While the technology looks good, a UC San Diego stem cell researcher said it faces a number of hurdles that make practical use of it difficult, especially the difficulty in producing the donor-derived regulatory cells in time to be of use in a transplant.

Use of these Tregs and immature DCregs for transplant has been investigated for several years now. In theory, they would provide a better method of preventing rejection than immunosuppressive drugs that knock down immune functioning across the board.

However, activating Tregs must be done precisely, or other T cell types will be activated, increasing the risk of rejection.

The study found that donor-type dendritic cells reliably activated Tregs and not the other types. Peptide antigens from the graft directed naive CD4+ T cells to mature into donor-specific Tregs, providing a selective immune signal to tolerate the graft.

Use of IPS cells for producing these immune regulatory cells is quite novel, said Dan Kaufman, director of cell therapy at UC San Diego, and affiliated with the universitys Sanford Stem Cell Clinical Center.

Obviously, it fits my interest in making immune cells from ES and IPS cells, Kaufman said. The ability to use these cells to suppress transplant rejection seems quite strong. I think the data is all good.

That said, the findings could be strengthened by extending the work from animals to human xenografts, he said. That would demonstrate that human IPS cells can similarly function, although it would be challenging.

Another limitation is the need to use donor-derived cells to induce immune tolerance.

How you would translate that would be unclear to me, Kaufman said.

Are you going to get a heart and then make IPS cells from that donor, which obviously you couldnt do in a reasonable time frame? Could you create a bank of these types of cells that might be suitable for certain patient populations with certain HLA types? Im not sure. I think that gets a little more speculative.

Another speculative possibility is to make the donor-derived IPS cells grow into an organ, and then also create the immune-regulating cells from these IPS cells to selectively induce tolerance.

But were still, I think, a long ways off from having IPS-derived organs, he said.

Autoimmune disease treatment with this technology is worth exploring, Kaufman said. In that case, the IPSCs would be made from the patients themselves.

More than 118,000 Americans are on the waiting list for an organ transplant, according to the Organ Procurement and Transplantation Network.

bradley.fikes@sduniontribune.com

(619) 293-1020

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Stem cell-based treatment prevents transplant rejection, in animal study - The San Diego Union-Tribune

Recommendation and review posted by simmons

It was Thursday afternoon, and the little girl from near Cologne, Germany, and the 40-year-old Duluth woman had known each other for less than 24 hours. But it was obvious that Edwards already had bonded with Ina and her little sister Mila.

They were together because the girls' mother had given Edwards a much greater gift: the gift of life.

"By your donation, I still get to be a mom," Edwards told Daniela Halfkann, 30. "(You're) a mom, so you completely understand how important it is to be here with your children."

Edwards, the mother of 15-year-old twin boys and the wife of Duluth Fire Chief Dennis Edwards, is alive because of the stem cell transplant she received at the Mayo Clinic on Oct. 31, 2014. As a result, she said, she is in remission from the rare and aggressive form of leukemia with which she had been diagnosed that June.

All she was told at the time of the transplant was that the donor was a woman from Germany.

Halfkann had registered as a potential stem cell or bone marrow donor at the large insurance company where she works in Cologne, she said. One day she received a call, saying her donation was needed.

After the six-hour procedure, Halfkann was told nothing more than that the recipient was a woman in the United States.

After a two-year waiting period required in Germany, the two women learned each other's identities last October and connected via Facebook.

Their meeting in Duluth was arranged by Amanda Schamper, Midwest donor recruitment coordinator for DKMS, the Germany-based organization that facilitated the donation.

Halfkann made the trip along with husband Stefan and their daughters, leaving their home at 3 a.m. on Tuesday and arriving at the Duluth International Airport at 5 p.m. on Wednesday.

Like Edwards, DKMS wants to raise awareness of the need for people to enter the registry, said Schamper, who also traveled to Duluth for the occasion.

She said 14,000 patients are in need of a peripheral blood stem cell or bone marrow donation, but fewer than half will get one because there's no match on the registry.

"We're looking for a particular protein in our DNA," she explained.

Only in 30 percent of cases are siblings a match. Edwards' brother and sister both had been screened, she said, and neither was a match for her.

Finding a match "is equated to finding your genetic twin, or winning the genetic lottery," Schamper said.

If more people were on the registry a process that only requires taking a swab from your cheek there would be more potential matches. But only 2 percent of eligible Americans are registered, Schamper said.

When the Halfkanns arrived at the gate on Wednesday, Dennis and Merissa Edwards, along with sons Caden and Jaxon, were waiting at the gate.

It was an emotional moment.

"It was hard for me," Merissa Edwards said on Friday, speaking to Daniela Halfkann. "I was crying. I was so emotional, so happy to meet you and hug you."

She wiped away a tear. "I still am."

"It was amazing," Halfkann responded. "I cried at the gate, too."

The Halfkanns, who are staying at the Edgewater, initially focused on recovery from jet lag. But Edwards is making sure they'll get a full taste of Duluth and Minnesota before beginning their return trip to Germany next Saturday. That includes visits to the Mall of America, the Great Lakes Aquarium and a trip up the North Shore.

A "thank-you party," open to the public, is planned on Sunday afternoon. Halfkann also will be recognized on Monday during the Saints Sports Awards ceremony at the College of St. Scholastica, where Edwards is an administrative assistant in the athletics department.

Recovery from the ravages of leukemia has been a long process, Edwards said, but she remains in remission. She gets a PET scan every six months to make sure that's still the case; the next one takes place next week.

Edwards shares her story, she said, not to call attention to herself but to highlight the need for people to take the simple step of registering as a potential donor.

"It's so important for us to help other people keep their families together and save a mother or father or son or daughter," she said. "The more people we can encourage to cheek-swab and get on the registry, the more lives we can help save and help families stay together."

TO LEARN MORE

For more information and to learn how to get on the bone marrow and peripheral blood stem cell registry, visit dkms.org.

IF YOU GO

The thank-you party for Daniela Halfkann will be from 2 to 5 p.m. on Sunday at The Other Place Bar and Grill, 3930 E. Calvary Road.

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Duluth woman meets the German donor whose stem cells saved her life - WDAZ

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Madhu Srivastava/IANS

Our health scenario has undergone drastic changes over the past few decades. Is it not fair to say that given today`s lifestyles, finding someone who is completely fit and healthy is quite rare?

In an attempt to take precautions, people are doing their best such as exercising, controlling their diet, taking health supplements, undergoing periodic health check-ups and what not. However, these are only precautionary or preventive measures. How can you protect yourself or your family in the worst case of being diagnosed by a disorder that could take you by surprise?

The good news is that while more new diseases are being discovered, medical science is also growing at a matching pace with treatment solutions for such conditions. Here comes the role of stem cells. Stem cell treatment has been found to be a solution for many ailments that are not treatable by conventional methods of surgery or medication. Diseases such as leukemia ans thalassemia can now be treated by stem cells with a hope of survival, which was not possible even a decade or so ago.

As stem cell medicine keeps advancing with more conditions being researched, the future sounds quite promising as more and more disorders that were once considered as permanent or terminal will soon become treatable.

But, the question is, where does one find these stem cells? Well, the answer is, within you; in your bone marrow.

Unfortunately in more than 80 per cent of these disorders, your own stem cells cannot be used and you would need to seek them from someone else. Here comes the challenge of finding a matching donor and someone who is willing to donate his stem cells. Alternatively, you can source stem cells from a public bank which preserves umbilical cord stem cells of donors. Here, in addition to the rarity of finding a matching stem cell of Indian ethnicity, the problem gets compounded with the need of Rs 15 lakh to Rs 20 lakh for stem cell treatment. Seven out of 10 patients who require a matching stem cell do not find a match in their family.

Can this scenario change? Is there a hope for treating such dreaded conditions? The answer is yes. If you are expecting a baby in your family, then you are blessed.

We say a child is a blessing from God, which is true in real terms. While as parents we bless our children, now the baby can bless us to stay protected against such medical conditions.

The baby`s umbilical cord is a rich source of stem cells. These can be collected and preserved at the time of birth for future use so that it can come to the rescue when required. With the new concept of community stem cell banking by one of the leading stem cell banks in the country which preserves a baby`s stem cells by making him/her a member of the community of parents who have also preserved their baby`s stem cells. The stem cell of all the babies within the community forms a collective pool to be accessed by all members.

Hence, at any given time, one can access donor stem cells from this collective pool for treatment, protecting not just your baby but your family too from such ailments. There is only the initial cost of preserving your baby`s stem cells which is much lower than sourcing stem cells from a public stem cell bank. More importantly, the probability of finding a match of Indian ethnicity is higher and stem cells are readily available for treatment when required.

If you are worried about the uncertainties over the health of your family, you can now rest assured that your baby`s birth will now bless your family with the protection of good health.

Link:
Stem cells and the art of giving - Zee News (blog)

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Underlying cause of a form of macular degeneration characterized Science Daily The new information sets the team up for testing a gene therapy to treat the disease, as the researchers will be able to observe whether or not these structural and biochemical abnormalities have been corrected. "Now that we understand what we're ...

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Underlying cause of a form of macular degeneration characterized - Science Daily

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Do You Have A Hormone Deficiency?

Thierry Hertoghe M.D. developed this symptom questionnaire and self-test to help you determine if your levels of hormones are below normal (read his book). Your doctor will help you understand these hormones better your first visit. We believe in the principle of doctor as teacher and we want you to understand how your body works and how the treatments will help you.

Your physician at Femme Clinique can help you simultaneously balance your hormones and help you determine the most beneficial supplements to support your therapy using a combination of prescription medications and nutritional and lifestyle coaching. We strongly believe in prevention and will help you determine the most effective and safest approach to enhancing your life and delaying the effects of aging.

The more symptoms you have in a category the more likely you have a deficiency of that hormone. Keep in mind that hormone excess can inhibit other hormones so you need an experienced physician to provide your with the correct lab testing and treatment protocols. We feel the safest approach to hormone replacement is balancing multiple hormones instead of just trying to balance one hormone as is done in traditional hormone replacement with horse hormones (estrogen only).

Using 24 hour urine hormone testing we can diagnose deficiencies of these hormones in a very holistic way (see hormone chart). In regular blood and saliva tests we only see snapshots. With a 24 hour urine collection we can check over 30 different hormones and help optimize hormone related cancer and bone health markers.

Over 20 estrogens are known and three are used clinically. These three estrogens are estrone (E1), estradiol (E2), and estriol (E3). Estradiol is the most potent estrogen produced by the ovaries.

Estrogens are also made from testosterone and other male hormones produced in the adrenal glands. Obese women tend to convert more of their testosterone to estrogen. This can lead to a poor testosterone to estrogen ratio in women and related symptoms of decreased libido and increased risk of osteoporosis.

Most of your estrogens are bound to proteins, preventing them from being active in your body. Using 24 hour hormone testing, we can measure the active hormones which are the most important because they tell us about the hormones activity in your body.

It is important to balance the different estrogen levels because this has been shown to reduce cancer risk. One of the products of estrogen breakdown (metabolism) is 2-methoxy-estradiol, a methylated estrogen. 2-methoxy-estradiol has been shown to have anti-cancer benefits.

In addition, it is important to balance estrogen with other hormones like testosterone. In a retrospective, observational study that included 508 women in 2005, researchers found that the addition of testosterone to conventional hormone therapy for postmenopausal women does not increase and may indeed reduce the hormone therapy-associated breast cancer risk-thereby returning the incidence to the normal rates observed in the general, untreated population.

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Growth hormone-releasing hormone (GHRH), growth hormone, and insulinlike growth factor 1 have potent effects on brain function, their levels decrease with advancing age, and they likely play a role in the pathogenesis of Alzheimer disease.

Human Growth Hormone (HGH) is produced in your anterior pituitary gland in pulses throughout the day. The largest of these GH pulses is about an hour after you fall asleep.

HGH production can be stimulated by other hormones. The main signal for HGH production in your body is growth hormone releasing hormone (GHRH).

The effects of GHRH on cognitive (brain) function in adults with mild brain impairment and healthy older adults were studied in 2012. The results of this controlled trial were published in the journal Neurology and showed that brain function could be improved even in normal healthy adults with benefits including increased acuity and memory. We use this similar approach with our patients using secretagogues. Secretagogues help balance hormone levels by stimulating the production of other hormones. HCG has a similar effect on progesterone and estrogen levels in women. Sermorelin in a peptide that stimulates growth hormone production similar to GHRH.

Growth hormone has many effects on your body:

There are some concerns about HGH and cancer risk.Our growth hormone levels are highest when we are younger and then decline with age. Our cancer risks increase as we age. Since growth hormone levels decline as we age and cancer risks increase it would make sense that growth hormone deficiency may increase cancer risk. Despite these theoretical risks and benefits, we feel that it is all about balance.

For example, in brain function, balance is very important:

Data suggest that not only absolute levels of sex hormones but also the balance between estrogen and testosterone and their metabolites may be important for cognitive function in women.

Despite the potential risks and benefits of growth hormone therapies, there is evidence that maintaining optimal growth hormone levels (IGF-1) can decrease your overall healthcare costs (and overall mortality risks):

Subjects with low in contrast to intermediate IGF-I exhibited 30.6% higher annual total costs 5 years after baseline examination, corresponding to a difference in adjusted costs of EUR436.61.

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Progesterone had no effect on undisturbed sleep but restored normal sleep when sleep was disturbed (while currently available hypnotics tend to inhibit deep sleep), acting as a physiologic regulator rather than as a hypnotic drug.

A 2011 study on the use of progesterone in post-menopausal women found that progesterone can help improve sleep only if your body needs it. Progesterone deficiency can cause anxiety and anger, which can be exacerbated by anemia from extended periods in younger women.

During ovulation, progesterone is secreted from the ovary and during the second 2 weeks of the cycle. During menopause women make less progesterone than ovulating women. It is a myth that progesterone is not needed for women who have had a hysterectomy because progesterone effects the entire body. The bone, nerves, brain, white blood cells, lungs, uterus, breast and colon all have progesterone receptors.

Progesterone can improve sleep, mood, and many other aspects of your life. It is all about balance.

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Over 50% of women are believed to be affected by female sexual dysfunction (FSD).

Testosterone therapy for women has been in use for over 80 years. Uses was have included treatments sexual dysfunction, abnormal uterine bleeding, dysmenorrhea, menopausal symptoms, chronic mastitis and lactation, and breast, uterine, and ovarian tumors.

Testosterone replacement for menopausal women, whether from surgery or aging, can improve sexual desire and libido. Most research shows that testosterone therapy is safe, when done correctly.

The North American Menopause Society position on testosterone therapy in postmenopausal women with decreased sexual desire may benefit for testosterone therapy. They recommend testosterone therapy with estrogen therapy at the same time.

Testosterone therapy improves arousability, sexual desire and fantasy, frequency of sexual activity and orgasm, and satisfaction and pleasure from the sexual act.

Over 16 million women aged 50 and older have low sexual desire. Hypoactive sexual desire disorder (HSDD) is common and symptoms include decreased sexual desire that causes relationship or personal problems. HSDD is common in women who have had their ovaries removed. Women receiving testosterone therapy typically notice great improvement in sexual satisfaction.

A 2009 study in the Journal of Sexual Medicine, with 637 women using testosterone over 18 years, found that testosterone therapy had no impact on breast cancer risk.

Testosterone (T) improves the ability of estrogen to improve bone health. A 2008 study on post-menopausal women receiving hormone replacement therapy found that women receiving testosterone saw greater improvement in bone density than with estrogen (E) replacement alone. The favourable estrogenic effects on lipids were preserved in women treated with T, in association with beneficial changes in body composition. They also noted that the, addition of testosterone resulted in a significantly greater improvement compared to E for sexual activity (P < 0.03), satisfaction (P < 0.03), pleasure (P < 0.01), orgasm (P < 0.035) and relevancy (P < 0.05). In the statistical world, those are some nice looking P values. The smaller the P value, in this case, the more likely it is you will see the same benefit.

Testosterone therapy improves heart function, blood sugar regulation, and muscle strength in women with advanced chronic heart failure. The heart is a muscle and all the blood vessels are lined with smooth muscle. Testosterone has been shown to be a effective and safe therapy for elderly women with chronic heart failure.

Inadequate response to antidepressant monotherapy in women with major depressive disorder is common.

Low doses of testosterone in women have been used with good success in the treatment of depression. Remember, depression is not a symptom of a Prozac deficiency but rather the symptom of an imbalance.

With our approach to balancing hormone levels, side effects of testosterone therapy are uncommon. With excessive testosterone levels you may notice acne or increased body hair. This is why we always start with a very low dose and increase as needed.

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DHEA, also known dyhdroepiandrosterone, is very abundant in our bodies at younger ages but as you age your levels decline. DHEA is converted into pheromones by our bodies so one symptom of deficiency is lack of special scent during sexual arousal. The best way to take DHEA is transdermally in order to bypass the liver. DHEA is one of the most plentiful hormones in our bodies when we are younger. Its decline in your body can have a significant impact on libido and youthful appearance.

Low thyroid is more common in women than men. The test most often ordered to screen for thyroid problems is called TSH, thyroid stimulating hormone, and is practically useless in evaluating function. This is because TSH is just the signal from the brain to the thyroid. We want to know how much thyroid hormone is actually being made. To do this we measure T3 and T4 and other blood levels and often find that the levels are out of balance despite being treated with synthetic thyroid hormones.

Thyroid hormones are meant to be released slowly over several hours but more thyroid medications just give a big spike. With combination, T4 and T3, natural thyroid hormones can potentially provide more sustained levels. In addition, autoimmune thyroid is very common in women and often leads to sudden surges of thyroid hormones being released during the attack phase. Patients may notice this as sudden increases in heart rate and a feeling of anxiety.

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Longevity Clinic BioIdentical Hormone Therapies - Bellevue ...

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