Feb. 6, 2013 Researchers have found a genetic variant that doubles the likelihood that people will have calcium deposits on their aortic valve. Such calcification, if it becomes severe, can cause narrowing or a blockage of the aortic valve, a condition called aortic stenosis. The study is the first large-scale, genome-wide association study to uncover a genetic link to aortic valve calcification.

An article detailing the findings is published in the February 7, 2013 issue of The New England Journal of Medicine.

The study's lead investigators -- from Johns Hopkins, Harvard University, McGill University, the University of Iceland and the National Institutes of Health -- found that having a genetic variant in the LPA gene, which codes for a type of cholesterol particle called lipoprotein (a), also increases the risk of developing aortic stenosis by more than 50 percent.

The researchers say their findings not only help explain why heart valve calcification may run in families, they could also lead to the development of targeted medications that might slow the progression of the disease. Statin medications, which reduce common forms of cholesterol that can clog blood vessels, have not been shown to reduce aortic valve calcification.

"This is an important step forward in understanding the biology of the development of aortic stenosis and how this common genetic variant, which is found in 7 percent of the general population, contributes to that risk," says Wendy Post, M.D., a cardiologist and associate professor of medicine and epidemiology at the Johns Hopkins University School of Medicine who is a senior author of the study.

Non-genetic risk factors for aortic valve calcification include advancing age, high blood pressure, obesity, high cholesterol levels and smoking. Men are at higher risk than women.

In the study, the researchers first looked at 2.5 million gene variants, called single nucleotide polymorphisms or SNPs, among more than 6,900 people of white European background, and found that this variant in the LPA gene was strongly associated with having aortic valve calcification on a heart CT scan, according to lead author Catherine Campbell, M.D., formerly the chief cardiology fellow at Johns Hopkins who now works for Kaiser Permanente.

Lipoprotein (a) is an unusual type of cholesterol particle that circulates in the blood and is associated with an increased risk for heart attacks.

"Increased levels of lipoprotein (a) have been previously associated with aortic valve disease. However, prior studies could not differentiate whether it was simply a marker or a causal factor," says Campbell. "Our results provide the first evidence for a causal relationship between lipoprotein (a) and calcific aortic valve disease," she adds.

Once the researchers identified the association between the LPA gene variant and those with evidence of aortic valve calcium in that first group, they confirmed their findings among three other groups -- more than 2,000 people of Hispanic origin, about 2,500 African-Americans and more than 700 Germans.

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4 February 2013 Last updated at 21:33 ET By James Gallagher Health and science reporter, BBC News

A tiny "genetic patch" can be used to prevent a form of deafness which runs in families, according to animal tests.

Patients with Usher syndrome have defective sections of their genetic code which cause problems with hearing, sight and balance.

A study, published in the journal Nature Medicine, showed the same defects could be corrected in mice to restore some hearing.

Experts said it was an "encouraging" start.

There are many types of Usher syndrome tied to different errors in a patient's DNA - the blueprint for building every component of the body.

One of those mutations runs in families descended from French settlers in North America.

When they try to build a protein called harmonin, which is needed to form the tiny hairs in the ear that detect sound, they do not finish the job.

It results in hearing loss at birth and has a similar effect in the eye where it causes a gradual loss of vision.

Scientists at the Rosalind Franklin University of Medicine and Science, in Chicago in the US, designed a small strip of genetic material which attaches to the mutation and keeps the body's factories building the protein.

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Resposta de geneticista a Silas Malafaia
Homossexualidade é "comportamento e não genética"? E a Genética do Comportamento? Vídeo postado pelo Mestre Eli Vieira, parabéns! http://www.facebook.com ==Referências== Capítulo de Manual de Genética do Comportamento dedicado à Homossexualidade, citando mais de 50 referências científicas: Dawood, Khytam, J. Michael Bailey, and Nicholas G. Martin. "Genetic and environmental influences on sexual orientation." Handbook of behavior genetics (2009): 269-279. PDF: is.gd Artigos: Bailey, Nathan W., and Marlene Zuk. "Same-sex Sexual Behavior and Evolution." Trends in Ecology Evolution 24, no. 8 (August 1, 2009): 439--446. doi:10.1016/j.tree.2009.03.014. Savic, Ivanka, and Per Lindström. "PET and MRI show differences in cerebral asymmetry and functional connectivity between homo-and heterosexual subjects." Proceedings of the National Academy of Sciences 105, no. 27 (2008): 9403-9408. Kerr, Warwick Estevam, and Newton Freire-Maia. "Probable inbreeding effect on male homosexuality." Rev. Brasil. Genet 6 (1983): 177-180. PDF: web2.sbg.org.br Bocklandt, Sven, and Eric Vilain. "Sex Differences in Brain and Behavior: Hormones Versus Genes." Advances in Genetics 59 (2007): 245--266. doi:10.1016/S0065-2660(07)59009-7. Burri, Andrea, Lynn Cherkas, Timothy Spector, and Qazi Rahman. "Genetic and Environmental Influences on Female Sexual Orientation, Childhood Gender Typicality and Adult Gender Identity." PLoS ONE 6, no. 7 (July 7, 2011): e21982. doi:10.1371/journal.pone.0021982. Lübke, Katrin ...

By: Werner Mayer

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Genetic Mutation - Magnificent Modifier or Mega Meltdown? -- Creation Magazine LIVE! (2-03)
Special guest, former atheistic evolutionist Dr John Sanford provides remarkable insights into new discoveries in genetics that powerfully support a recent creation of humans and no evolution. The Creation Magazine LIVE! TV program is a ministry of Creation Ministries International. With offices in seven countries and more PhD scientists than any Christian organization this program features cutting edge science that supports the Bible delivered in a non-technical, visually-rich, discussion-based format. Related Articles: Can mutations create new information? (creation.com CCR5-delta32: a very beneficial mutation (creation.com Sickle-cell anemia does not prove evolution! (creation.com Beetle bloopers - Even a defect can be an advantage sometimes (creation.com Genetics Q A page: (creation.com Mutations Q A page: (creation.com 15 Questions for Evolutionists (Question 3) (creation.com Related Products: Genetic Entropy and the Mystery of the Human Genome (creation.com The Mystery of Our Declining Genes DVD (creation.com Dynamic Life DVD (creation.com Creation magazine (creation.com

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Adorable Black Duiker Born at the San Diego Zoo
An rare black duiker, Rashidi, was recently born at the San Diego Zoo. The young duiker, whose name means "rightly guided" in Afrikaans, is 7 weeks old and weighs 17 pounds. The species is near threatened in the wild, mainly due to natural predators and agricultural burn off, making this birth at the San Diego Zoo significant for the genetics of the species. sandiegozoo.org

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Why Failure hates Persistence
http://www.newellstrength.com Persistence will always allow you to reach your goals. Something so simple yet so few people follow through. This is how I, an guy of average genetics and intelligence, am living the life of my dreams.

By: Kyle Newell

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Attack of Mewtwo: The (mostly) unironic reading. Chapter 3
things are heating up! (A/N Because of confusing evil mewtwo will be called mewthree from now on and I know now the title does nt make sense but TOO BAD) Mewtwo and Mewthree faxed each other. Mewtwo shoot shadow balls at mewthree and mewthree dogded balls and laffed "you are infearier clone mewtwo now I kill you for trying to stop me from wiping out human race ha ha" "who are you" asked mewtwo "I am mewthree and humans made me from your genetics but I hat humans" "No!" shouted mewtwo "how dare they use my genetic its MY GENETIC NOT YORS" Suddenly a clon army came out from behind mewthree "it is the attack of the clones" said Sarah and mewto teleportled themt o lab. "where is this?" asked jake "I once tried to kill humans too" said mewto "but now I use my science for good I cannot fight clone army alone you must help me" Mewtwo used genetics on there pokemon and they evolved and becum stronger than normal pokemon except for Sarah #39;s eevee which could evolve into any evolushon when ever she wanted then turn back the eevee was also pink and blue in stead of brown and whit. "Now you are ready to fight mewthree" said mewtwo and then jake turned on television and saw that clone army was already killing humans and jake was mad "we have to stop the clones" said jake and mewtwo teleported them to the city where the clone pokemon were attacking but mewthree wasn #39;t there and it made them mad. "I will find mewthree" said mewtwo and mewtwo teleported and left them to fight clones all ...

By: dorfinators

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Attack of Mewtwo: The (mostly) unironic reading. Chapter 4
My face, the whole time. (AN: I know Im not very gud at spelling but I don #39;t think matters you can still understan me rite? Besides English not first langauge. Also I have not played pokemon games so I don #39;t no how to spell certain moves ok but like I sad it dont matter and Sarah and Jake find themselves in Safron city and saw clones everywere. "Their everywere" said Jake. "Dont worry!" said Sarah and then Eevee turned into an umbrion but it was still pink. mewthree odered the clones to attack, and they started to blow buildings. "No there were people in there!" said Jake. "Gallad go!" then Gallaid came out. It was blue and white and shiny. "Syco cut" ordered Jake and then the gallaid used Syco cut which hurt a weezing clone. Then a chairzard started to breath fire at them but Eevee turned to vaporion to stop it then it used hydro pimp. Then more clones came and Eevee changed form to stop them but there were too many of them. "There are too many of them" sad Jake. And then they sent out a bunch of other pokemon and they started to do well against clones but there were still too many. Then Saraj looked up in the sky and saw somone with purple hair riding on garchomp it was Paul. "LEAVE HER ALONE YOU DAMN CLONS" said Paul and he sent out a bunch of pokemon and beat up the rest of the clones. "Thank you Paul" said Sarah "No prolbem" said Paul. "Jus here to help". Jake was jealus because Paul was look amazing but Sara dint notice. Suddenly Mewthree in the sky. "Attenton every ...

By: dorfinators

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Focus Genetics - Proven in the Paddock (One)

By: FocusGenetics

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Focus Genetics - Proven in the Paddock (Two)

By: FocusGenetics

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Focus Genetics - Proven in the Paddock (Three)

By: FocusGenetics

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West Texas Genetics Concrete

By: Clint Halfmann

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Living with Gynaecological Cancer Part 2.1 -- Physical Aspects - Genetics
Living with Gynaecological Cancer A resource for Women and their families living with the impact of gynaecological cancer. Part 2.1 -- Physical Aspects - Genetics While the majority of gynecological cancers are sporadic, hereditary factors may play a role in diagnosis, particularly in the presence of ovarian and to a degree endometrial cancer. Genetic testing is a process in which a blood test may help to determine if you or your family members are at very high risk of gynecological cancers. Decisions about when these tests are undertaken cannot be taken lightly, both for practical and financial implications, but also because the results can impact significantly on future plans for things like childbearing and careers.

By: LifehouseatRPA

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DNA - Little Mix
DNA - Little Mix Does he tell you he loves you when you least expect it? Does he flutter your heart when he kisses your neck? No scientist or biology It #39;s obvious when he #39;s holding me It #39;s only natural that I #39;m so affected And my heart won #39;t beat again If I can #39;t feel him in my veins No need to question, I already know It #39;s in his DNA DDD-DNA It #39;s in his DNA And he just takes my breath away Bbb-breath away I feel it every day, And that #39;s what makes a man Not hard to understand Perfect in every way I see it in his face Nothing more to say It #39;s in his DDD-DNA It #39;s the blue in his eyes that helps me see the future Fingerprints that leave me covered for days, yeah, hey, yeah Now I don #39;t have any first degree But I know, what he does to me No need to work it out, it #39;s so familiar, ooh, ooh, ooh And my heart won #39;t beat again If I can #39;t feel him in my veins No need to question, I already know It #39;s in his DNA DDD-DNA It #39;s in his DNA And he just takes my breath away Bbb-breath away I feel it every day, And that #39;s what makes a man Not hard to understand Perfect in every way I see it in his face Nothing more to say It #39;s in his DDD-DNA It #39;s all about his kiss Contaminates my lips Our energy connects It #39;s simple genetics I #39;m the X to his Y It #39;s the colour of his eyes He can do no wrong No, he don #39;t need to try Made from the best He passes all the tests Got my heart beating fast It #39;s cardiac arrest He #39;s from a different strain That science can #39;t explain I guess that #39;s how he #39;s made In ...

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EPXbody Review - By Dan Putnam CEO Of EPXBody
getepx.com There is nothing more exciting than sharing with others a true opportunity; an opportunity that was designed from the ground up to be like no other opportunity in the 60-year history of Network Marketing! EPXbody is that opportunity! EPX is short for Epigenetics; science has shown that with proper nutrition, diet and exercise, you can improve your genetics and the genetics of generations to follow. Our products are designed to give you better health the opportunity that leads to wealth. Please visit my site for more information: getepx.com

By: Lovette DePina

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Seattle Genetics Inc. (SGEN) recently commenced two phase I clinical studies with its oncology candidate, SGN-CD19A. The two trials will assess the safety and anti-tumor response of SGN-CD19A in CD19-positive acute lymphoblastic leukemia (ALL) and B-cell non-Hodgkin lymphoma (HL) patients.

Establishing the maximum tolerated dose and examining the safety of the candidate are the two primary endpoints of the trials. The trials will also assess antitumor activity, pharmacokinetics, progression-free survival (PFS) and overall survival (:OS).

According to the American Cancer Society, more than 70,000 cases of non-HL and roughly 6,000 cases of ALL were expected to be diagnosed in the US during 2012 and almost 19,000 people were expected to die from non-HL and more than 1,400 were expected to die from ALL.

Meanwhile, Adcetris recently received approval under Health Canada's Notice of Compliance with conditions (NOC/c) for the treatment of relapsed or refractory HL and systemic anaplastic large cell lymphoma (sALCL).

For similar indications, Adcetris was approved by the US Food and Drug Administration (:FDA) in Aug 2011 and in the EU in Oct 2012.

Adcetris, Seattle Genetics' only approved drug, generated revenues of $102.8 million for the nine months ending Sep 30, 2012.

In Jan 2013, a global phase III study (ECHELON-2) was initiated on Adcetris. In this study, Adcetris plus chemotherapy will be evaluated for the front-line treatment of CD30-positive mature T-cell lymphoma (:MTCL) including patients with sALCL and other types of peripheral T-cell lymphomas.

Seattle Genetics currently carries a Zacks Rank #4 (Sell). Right now, Peregrine Pharmaceuticals, Inc. (PPHM), Agenus Inc. (AGEN) and Targacept, Inc. (TRGT) look more attractive with a Zacks Rank #1 (Strong Buy).

Read the Full Research Report on TRGT

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SGEN Starts SGN-CD19A Phase I Study

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CAMBRIDGE, MA--(Marketwire - Feb 6, 2013) - Good Start Genetics, Inc., an innovative molecular diagnostics company developing the new gold standard in carrier screening, announced today that it will present at the 2013 BIO CEO & Investor Conference at the Waldorf Astoria in New York, NY. Don Hardison, president and CEO of Good Start Genetics, is scheduled to present Monday, Feb. 11, at 2 p.m. EST.

Mr. Hardison will provide an overview of Good Start Genetics and its groundbreaking next-generation DNA sequencing based carrier screening service, as well as highlights from its first year on the market.Mr. Hardison will be available for one-on-one meetings with investors and media attending the conference.

Good Start Genetics also is proud to announce that four separate abstracts related to its technology have been accepted for presentation at the upcoming annual meetings of the Pacific Coast Reproductive Society (PCRS) and the American Congress of Medical Genetics and Genomics (ACMG).

Since its national launch in April 2012, Good Start Genetics' high-complexity, CLIA- and CAP-accredited laboratory has processed tens of thousands of test orders. The GoodStart Select next-generation gene sequencing technology detects common mutations in carriers across all 23 diseases recommended for testing by major medical societies as well as rare mutations that would go undetected by laboratories using older, traditional genotyping-based technologies. For example, most genotyping technologies detect about 100 mutations for cystic fibrosis (CF), while GoodStart Select detects 550 mutations.

Event BIO CEO & Investor Conference

Date Monday, Feb. 11th

Time 2:00 p.m. EST

Place Waldorf Astoria NY, Park South 301 Park Ave New York, NY 10022

About Good Start Genetics, Inc.

Good Start Genetics is setting the new gold standard in carrier screening by making testing for the most comprehensive set of known and novel disease-causing mutations accessible for routine clinical practice. After years of development and rigorous validation, Good Start Genetics has harnessed the power of next-generation sequencing and other best-in-class technologies to provide highly accurate, actionable and affordable tests for all disorders recommended for genetic testing by ACOG and ACMG. For these reasons, fertility specialists and their patients can have a high degree of confidence in their carrier screening results, and no longer have to compromise accuracy for price. For more information, visitwww.goodstartgenetics.com.

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Good Start Genetics to Present at 15th Annual BIO CEO & Investor Conference Monday, February 11th

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Do You Know Your Mesothelioma Treatment Options?
This week we bring to you a video infographic on the available treatment options for mesothelioma cancer patients. Have you tried any of these before? Have you found a combination of treatments that has worked for you? Let us know at http://www.asbestos.com or on Facebook: http://www.facebook.com/themesocenter Transcript: "Did you know... That mesothelioma patients have a number of treatment options to extend their life expectancy and ease symptoms? The most common treatments are surgery, chemotherapy and radiation therapy. Surgery has the best chance at extending survival, especially among patients with early stage cancer. Chemotherapy and radiation therapy work best to relieve symptoms and help manage the cancer #39;s growth. When doctors combine two or more treatments, it #39;s called multimodal therapy -- which has the best history of extending survival. In one multimodal study, 45% of early stage patients survived five years or longer. Clinical trials are exploring treatments like gene therapy, immune therapy and photodynamic therapy with growing success. Also on the rise is the use of alternative therapies among mesothelioma survivors like acupuncture, dietary changes, massage and meditation to complement their traditional treatments. Even though no cure has been found yet, numerous survivor stories offer hope to newly diagnosed patients searching for treatment options. Simply knowing that mesothelioma survivors exist is inspiring, and knowing which treatments worked for them provides ...

By: TheMesoCenter

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Biotech Showcase 2012_bluebird bio
bluebird bio is developing potentially transformative, one-time gene therapies for severe genetic diseases. bluebird bio has two clinical stage products in development for childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell disease, a world-class team and a broadly applicable gene therapy platform. http://www.bluebirdbio.com

By: AllianceRegenMed

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Sanctuary Season 4 Trailer
Stem cells, gene therapy, transplants, cloning... the very meaning of the word #39;humanity #39; changes daily in the modern world. But there is a darker side to the evolution of mankind, a truth only a few brave souls are willing to face: There are monsters loose in the world. And they are the key to the future of our race. Each one-hour episode of Sanctuary follows the adventures of the beautiful and enigmatic Dr. Helen Magnus and her team at the Sanctuary as they track down, study and try to help the strange and often terrifying creatures that secretly populate our world. Aided by forensic psychiatrist Dr. Will Zimmerman (ROBIN DUNNE), techno-whiz Henry Foss (RYAN ROBBINS), her fearless daughter Ashley (EMILIE ULLERUP) along with her butler, and dear friend Bigfoot (CHRISTOPHER HEYERDAHL), Magnus and her team use their unique combination of instinct, medicine and cutting-edge science and technology to find the creatures that the world refuses to believe exist. Season two also introduces Kate Freelander (AGAM DARSHI) into the fold, a quick-talking con-artist who has more than a few tricks up her sleeve. Kate #39;s uneasy relationship to the Cabal could prove useful for Magnus in her search for Ashley, but her less-than-trustworthy track record may cause far more damage than her intel is worth. Sanctuary is helmed by Executive Producer/Creator/Writer Damian Kindler, Executive Producer/Lead Actor Amanda Tapping and Executive Producer/Director Martin Wood. In addition, for season two ...

By: TRICONFILMSANDTV

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Marie McCullough, INQUIRER STAFF WRITER Posted: Wednesday, February 6, 2013, 9:08 PM

Two months ago, Children's Hospital of Philadelphia made international headlines for using an experimental gene therapy to save the life of a Pennsylvania girl who was dying of leukemia.

On Wednesday, the hospital made international headlines - and was denounced on Facebook as "cruel" "heartless," "greedy monsters" and worse - for appearing to tack on hundreds of thousands of dollars to the original price of treating a Croatian child, Nora Situm, 5, with the same breakthrough therapy.

The online outrage built all day before the hospital responded.

It refused to discuss the specific case because of "patient privacy," but implied in its statement that Nora's parents did not understand the difference between the hospital's charges, and what the parents may have to pay for follow-up care "either at CHOP or back in the patient's home country."

Children's "estimates the costs of treatment in advance and seeks payment at the time treatment begins," the statement said. "Additional follow-up clinical treatments are sometimes necessary and can be administered over several years..."

"CHOP does not charge for this follow-up clinical treatment at the time of initial treatment. If the child is not further treated at CHOP, CHOP will never charge for the follow-up treatment. However, CHOP does explain those potential costs to patient families at the outset so they understand the financial issues they may be facing."

Even so, Nora's parents may not understand.

Nora's mother, Dana Atanosovska Situm, held a news conference in Croatia on Wednesday to say the family had obtained visas and was leaving for Philadelphia on Thursday - a day earlier than planned because Nora is so gravely ill. (A Serbo-Croatian-speaking journalist translated the conference for The Inquirer.)

Situm also thanked support groups, celebrities, individual donors, and Croatia's capital city, Zagreb, for helping to raise $837,000 - an astronomical sum in the economically distressed Balkan country.

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CHOP cost estimate for girl’s therapy stirs online outrage

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Marie McCullough, Inquirer Staff Writer Posted: Thursday, February 7, 2013, 5:41 AM

Two months ago, Children's Hospital of Philadelphia made international headlines for using an experimental gene therapy to save the life of a Pennsylvania girl who was dying of leukemia.

On Wednesday, the hospital made international headlines - and was denounced on Facebook as "cruel" and "heartless," as being "greedy monsters" and worse - for appearing to tack on hundreds of thousands of dollars to the original price of treating a Croatian child, Nora Situm, 5, with the same breakthrough therapy.

The online outrage built all day before the hospital responded.

It would not discuss the specific case because of patient privacy issues, but implied in its statement that Nora's parents did not understand the difference between the hospital's charges, and what the parents may have to pay for follow-up care "either at CHOP or back in the patient's home country."

Children's "estimates the costs of treatment in advance and seeks payment at the time treatment begins," the statement said. "Additional follow-up clinical treatments are sometimes necessary and can be administered over several years . . . .

"CHOP does not charge for this follow-up clinical treatment at the time of initial treatment. If the child is not further treated at CHOP, CHOP will never charge for the follow-up treatment. However, CHOP does explain those potential costs to patient families at the outset so they understand the financial issues they may be facing."

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Pet Surgery | Las Vegas | Veterinary Stem Cell Therapy | TPLO
veterinary-stemcell.com 702-250-1888 What is stem cell therapy? Stem cells are the body #39;s repair cells. They have the ability to divide and differentiate into many different types of cells based on where they are needed throughout the body. Stem cells can divide and turn into tissues such as skin, fat, muscle, bone, cartilage, and nerve to name a few. They even possess the ability to replicate into organs such as the heart, liver, intestines, pancreas, etc. Call Dr. Mauer or visit his site today.. Find out if stem cell therapy is right for your pet.

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Stem Cell Therapy For Your Pet | Las Vegas, Nevada | Dr. Roger A. Mauer DVM
veterinary-stemcell.com Dr. Mauer is very excited to now be offering Stem Cell Therapy. This innovative and affordable treatment has been making news all over the country. This treatment is completely natural and there are no side effects other than occasional minor swelling at injected joint sites. Most animal owners see results in just days after treatment and their pets benefiting with less or no pain. Stem cells are the body #39;s repair cells. They have the ability to divide and differentiate into many different types of cells based on where they are needed throughout the body. Stem cells can divide and turn into tissues such as skin, fat, muscle, bone, cartilage, and nerve to name a few. They even possess the ability to replicate into organs such as the heart, liver, intestines, pancreas, etc.

By: Roger Mauer

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Public release date: 7-Feb-2013 [ | E-mail | Share ]

Contact: Mark Michaud mark_michaud@urmc.rochester.edu 585-273-4790 University of Rochester Medical Center

A study out today in the journal Cell Stem Cell shows that human brain cells created by reprogramming skin cells are highly effective in treating myelin disorders, a family of diseases that includes multiple sclerosis and rare childhood disorders called pediatric leukodystrophies.

The study is the first successful attempt to employ human induced pluripotent stem cells (hiPSC) to produce a population of cells that are critical to neural signaling in the brain. In this instance, the researchers utilized cells crafted from human skin and transplanted them into animal models of myelin disease.

"This study strongly supports the utility of hiPSCs as a feasible and effective source of cells to treat myelin disorders," said University of Rochester Medical Center (URMC) neurologist Steven Goldman, M.D., Ph.D., lead author of the study. "In fact, it appears that cells derived from this source are at least as effective as those created using embryonic or tissue-specific stem cells."

The discovery opens the door to potential new treatments using hiPSC-derived cells for a range of neurological diseases characterized by the loss of a specific cell population in the central nervous system called myelin. Like the insulation found on electrical wires, myelin is a fatty tissue that ensheathes the connections between nerve cells and ensures the crisp transmission of signals from one cell to another. When myelin tissue is damaged, communication between cells can be disrupted or even lost.

The most common myelin disorder is multiple sclerosis, a condition in which the body's own immune system attacks and destroys myelin. The loss of myelin is also the hallmark of a family of serious and often fatal diseases known as pediatric leukodystrophies. While individually very rare, collectively several thousand children are born in the U.S. with some form of leukodystrophy every year.

The source of the myelin cells in the brain and spinal cord is cell type called the oligodendrocyte. Oligodendrocytes are, in turn, the offspring of another cell called the oligodendrocyte progenitor cell, or OPC. Myelin disorders have long been considered a potential target for cell-based therapies. Scientists have theorized that if healthy OPCs could be successfully transplanted into the diseased or injured brain, then these cells might be able to produce new oligodendrocytes capable of restoring lost myelin, thereby reversing the damage caused by these diseases.

However, several obstacles have thwarted scientists. One of the key challenges is that OPCs are a mature cell in the central nervous system and appear late in development.

"Compared to neurons, which are among the first cells formed in human development, there are more stages and many more steps required to create glial cells such as OPCs," said Goldman. "This process requires that we understand the basic biology and the normal development of these cells and then reproduce this precise sequence in the lab."

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Cells forged from human skin show promise in treating MS, myelin disorders

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