Center for Adult Stem Cell Research and Regenerative Medicine
Our goal for the newly established Center for Adult Stem Cell Research and Regenerative Medicine is to shape and lead in the research, ethics, and societal implications for the field of adult...

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Ageless Derma Anti Aging Skin Care
This active ingredient won the Best Active Ingredient prize in European Innovation in 2008. Stem Cells derived from a rare Swiss Apple are part of the revolutionary technological designed...

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CAMBRIDGE, Mass., Oct. 9 (UPI) -- Patients with type 1 diabetes lack the insulin-producing cells that keep blood glucose levels in check. Currently, these patients must use insulin pumps or daily hormone injections to keep levels stable.

But in a recent breakthrough in laboratories at Harvard University, researchers came upon a new technique for transforming stem cells into pancreatic beta cells that respond to glucose levels and produce insulin when necessary. The breakthrough could lead to new less invasive, more hands-off treatment for diabetes.

Remarkably, the new technique -- a complex process which involves turning on and off specific genes and takes about 40 days and six precise steps to complete -- was replicated not only on embryonic stem cells but also on human skin cells reprogrammed to act in a stem-cell-like manner. This revelation allows scientists to produce millions of insulin-producing cells while avoiding the ethical dilemmas attached to traditional stem cell research.

Previous attempts to convert stem cells into insulin-producers have proven moderately successful, but these cells mostly produced insulin at will, unable to adjust their output on the fly. The latest techniques -- developed by Douglas Melton, co-director of the Harvard Stem Cell Institute, and his research colleagues -- produce insulin cells that react to glucose spikes by upping production, and lowering insulin output when there's not excess sugar to break down.

The breakthrough has already shown significant promise when used on lab mice. Diabetic mice who received a transplant of the stem cell beta cells had improved blood sugar levels, and were shown to be capable of breaking down sugar.

"We can cure their diabetes right away -- in less than 10 days," Melton told NPR. "This finding provides a kind of unprecedented cell source that could be used for cell transplantation therapy in diabetes."

But there's still one major issue. For reasons doctors still don't understand, the beta cells in humans with diabetes are attacked by the body's immune system. Researchers like Melton still have to figure out a way to protect the new beta cells from being killed -- otherwise the breakthrough won't become anything more than another short-term solution.

"It's taken me 10 to 15 years to get to this point, and I consider this a major step forward," Melton told TIME. "But the longer term plan includes finding ways to protect these cells, and we haven't solved that problem yet."

2014 United Press International, Inc. All Rights Reserved. Any reproduction, republication, redistribution and/or modification of any UPI content is expressly prohibited without UPI's prior written consent.

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Harvard researchers grow insulin-producing stem cells

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Research into a cure for diabetescould result in an end to insulin injections It has beenhailed as the biggest medical breakthrough since antibiotics Harvard researcher Doug Melton promised his children he'd find a cure Treatment involves making insulin-producing cells from stem cells Scientistshope to have human trials under way within a 'few years'

By Fiona Macrae for the Daily Mail

Published: 17:41 EST, 9 October 2014 | Updated: 18:12 EST, 9 October 2014

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Scientists have hailed stem-cell research into a cure for diabetes as potentially the biggest medical breakthrough since antibiotics.

It could result in an end to insulin injections, and to the disabling and deadly complications of the disease, such as strokes and heart attacks, blindness and kidney disease.

The treatment, which involves making insulin-producing cells from stem cells, was described as a 'phenomenal accomplishment' that will 'leave a dent in the history of diabetes'.

Scientists yesterday hailed stem-cell research into a cure for diabetes as potentially the biggest medical breakthrough since antibiotics

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Could this stem cell breakthrough offer an end to diabetes?

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Arthritis of shoulder; results four years after stem cell therapy by Harry Adelson, N.D.
Heavy discusses his outcome four years out from his first bone marrow stem cell treatment for his arthritic shoulders and torn rotator cuffs by Harry Adelson, N.D. http://www.docereclinics.com.

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Newswise The discovery of the cystic fibrosis (CF) gene was hailed as a trailblazing breakthrough in 1989. Now, on the 25th anniversary of the discovery of cystic fibrosis transmembrane conductance (CFTR) gene, more than two-dozen CF innovators and clinicians, including five on the Case Western Reserve University campus, took this special occasion to reflect on the discovery and the status of research and treatment during videotaped interviews.

Much of these videotaped interviews with physicians, scientists, patients and families will appear Friday, Oct. 10, at a special satellite symposium, Ahead of the Curve: CFTR at 25, during the 28th Annual North American Cystic Fibrosis Conference in Atlanta.

The satellite symposium will be presented by continuing education firm DKBmed, LLC, in collaboration with Johns Hopkins University School of Medicine and The Institute for Johns Hopkins Nursing. All two-dozen interviews conducted this past summer for CFTR at 25 may be accessed online in late October at aotc-cf.org/cftr25 or on YouTubes eCysticFibrosis Review Channel.

Two of the interviewed Case Western Reserve School of Medicine faculty members played pioneering roles in cystic fibrosis research during the past 25 years. Pamela B. Davis, MD, PhD, dean and senior vice president for medical affairs, served as director of the Adult Cystic Fibrosis Program at University Hospitals Case Medical Center before assuming duties as dean. Mitchell Drumm, PhD, professor of pediatrics and genetics and genome sciences, participated in the CFTR gene discovery process during his graduate studies at the University of Michigan.

Others interviewed from the Case Western Reserve campus represented the perspective of clinicians actively involved in a wide spectrum of CF patient care and research: James Chmiel, MD, associate professor of pediatric pulmonology and clinician-researcher of CF inflammation at the School of Medicine and clinical director of pediatric pulmonology at UH Rainbow Babies & Children's Hospital; and Colette Bucur, RN, nurse practitioner and research coordinator of CF clinical trials, and Terri Schindler, RD, nutritionist in CF care, both of UH Rainbow Babies & Children's Hospital.

Additional interviews feature policymakers, leading educators and renowned researchers and clinicians from Johns Hopkins University, Stanford University, University of North Carolina, University of California San Diego, University of Wisconsin, National Institutes of Health (NIH), Cystic Fibrosis Foundation and Hebrew University of Jerusalem. Course Director Peter J. Mogayzel Jr., MD, PhD, director of Johns Hopkins Cystic Fibrosis Center, selected interviewees based on their knowledge and experience to address CF research and advances in care. The interviewed CF patients and their families also put a human face on this illness by sharing their experiences.

CF is an illness where thick and sticky mucus builds up in airways and blocks digestive track passages. The CFTR gene controls the movement of salt and water in and out of the bodys cells. A faulty, or mutated, CFTR gene does not make the protein necessary to control the salt and water transport function, which triggers the excess mucus leading to cystic fibrosis.

In the 1950s, children with the illness rarely lived long enough to attend elementary school. Now, thanks to advances in treatment, the median survival is about 37 years after diagnosis. There have been reports, however, of CF patients living into their 70s.

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Newswise NEW YORK, NY (October 9, 2014)Using an innovative algorithm that analyzes gene regulatory and signaling networks, Columbia University Medical Center (CUMC) researchers have found that loss of a gene called KLHL9 is the driving force behind the most aggressive form of glioblastoma, the most common form of brain cancer. The CUMC team demonstrated in mice transplants that these tumors can be suppressed by reintroducing KLHL9 protein, offering a possible strategy for treating this lethal disease. The study was published today in the online issue of Cell.

The team used the same approach to identify mutations and heritable variants that have been linked to breast cancer and Alzheimers disease, suggesting that the algorithm, combined with the researchers sophisticated computer models of cellular regulation, is a powerful method for identifying genetic drivers of a wide range of diseases.

This algorithm adds a new dimension to our ability to identify the genetic causes of complex disease. When combined with other tools that our lab has developed, it will help identify many more genes that hold potential as genetic biomarkers of disease progression and targets for treatment, said study leader Andrea Califano, PhD, the Clyde and Helen Wu Professor of Chemical Biology (in Biomedical Informatics and the Institute for Cancer Genetics), chair of the Department of Systems Biology, and director of the JP Sulzberger Columbia Genome Center, at Columbias College of Physicians and Surgeons.

In previous studies, Dr. Califano and his colleagues used high-power computer models to demonstrate that certain types of cancer have highly conserved master regulatorsgenes whose individual or synergistic activity is necessary for disease to develop and persist. However, these models provided no information on the key genetic mutations that presumably drive the abnormal activity of these master regulators.

In the current study, the team combined its existing computational tools with a new algorithm called DIGGIT (for Driver-Gene Inference by Genetical-Genomic Information Theory), which walks backward from the master regulators to find the genetic events that drive cancer.

Conventional techniques, like genome-wide association studies, must test all possible genetic mutations and variants in a disease cell, compared with a normal cell, said lead author James C. Chen, PhD, a postdoctoral research scientist in Dr. Califanos laboratory, who developed DIGGIT. These can number in the tens to hundreds of thousands. As a result, based on the number of patients we have profiled, we have sufficient statistical power to identify only the most striking mutations. The DIGGIT algorithm, combined with what we know about regulatory events in the cell, can help us sort through this mass of data and identify critical hidden mutations that otherwise would have gone undetected.

The new approach was tested on mesenchymal glioblastoma, the most aggressive subtype of the disease, by jointly analyzing the gene expression and mutational profile data of more than 250 patients collected by the Cancer Genome Atlas consortium.

The CUMC team found two genesC/EBP and KLHL9that appear to activate glioblastomas master regulators. C/EBP, had already been identified by the labs of Dr. Califano and of Antonio Iavarone, MD, professor of neurology and of pathology & cell biology (in the Institute for Cancer Genetics), as a master regulator of the disease, so the researchers focused on KLHL9, which had never been tied to this or any other form of cancer.

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New Computational Approach Finds Gene That Drives Aggressive Brain Cancer

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9-Oct-2014

Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News @LiebertOnline

New Rochelle, NY, October 9, 2014Laser Solid Forming (LSF) is an innovative method for direct fabrication of metallic components in additive manufacturing. Renowned researchers Weidong Huang and Lin Xin, from China's Northwestern Polytechnical University, Shaanxi, describe their progress and applications with LSF technology and the excellent mechanical properties of the metallic parts produced in a Review article in 3D Printing and Additive Manufacturing, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the 3D Printing and Additive Manufacturing website until November 9, 2014. In the article "Research Progress in Laser Solid Forming of High Performance Metallic Components at the State Key Laboratory of Solidification Processing of China," the authors review research advances toward the goal of developing LSFan additive manufacturing technique that uses laser cladding with synchronously feeding metal powdersfor obtaining fully dense metal parts with mechanical properties similar to those produced by casting or forging.

"Additive manufacturing technologies have a global reach that is impacting the manufacturing landscape worldwide, and it is critical that both technology developers and users across the planet keep abreast of each other's progress," says Editor-in-Chief Hod Lipson, PhD, Professor at Cornell University's Sibley School of Mechanical and Aerospace Engineering, Ithaca, NY.

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About the Journal

3D Printing and Additive Manufacturing is a peer-reviewed journal published quarterly online with Open Access options and in print. Spearheaded by Hod Lipson, PhD, Director of Cornell University's Creative Machines Lab at the Sibley School of Mechanical and Aerospace Engineering, the Journal explores emerging challenges and opportunities ranging from new developments of processes and materials, to new simulation and design tools, and informative applications and case studies. Spanning a broad array of disciplines focusing on novel 3D printing and rapid prototyping technologies, policies, and innovations, the Journal brings together the community to address the challenges and discover new breakthroughs and trends living within this groundbreaking technology. Tables of content and a sample issue may be viewed on the 3D Printing and Additive Manufacturing website.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative medical and biomedical peer-reviewed journals, including Big Data, Soft Robotics, New Space, Tissue Engineering, and Stem Cells and Development. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's more than 80 journals, newsmagazines, and books is available on the Mary Ann Liebert, Inc., publishers website.

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New advances in additive manufacturing using laser solid forming to produce metallic parts

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Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News @LiebertOnline

New Rochelle, NY, October 9, 2014Excessive and often lethal blood levels of bilirubin can result from mutations in a single gene that are the cause of the metabolic disease known as Crigler-Najjar syndrome type 1 (CNS1). A new gene therapy approach to correcting this metabolic error achieved significant, long-lasting reductions in bilirubin levels in a mouse model of CNS1 and is described in an Open Access article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Human Gene Therapy website at http://online.liebertpub.com/doi/full/10.1089/hum.2013.233.

In "Life-Long Correction of Hyperbilirubinemia with a Neonatal Liver-Specific AAV-Mediated Gene Transfer in a Lethal Mouse Model of CriglerNajjar Syndrome," Giulia Bortolussi and coauthors from the International Centre for Genetic Engineering and Biotechnology; Centro Studi Fegato, Fondazione Italiana Fegato; and University of Trieste (Trieste, Italy) and Charles University (Prague, Czech Republic), present details of the adeno-associated virus (AAV)-mediated gene therapy approach they used to correct the metabolic disorder that causes hyperbilirubinemia in CNS1. The researchers reported 70-80% reductions in plasma bilirubin levels early on among treated animals, with about 50% reductions maintained throughout the study. The authors compared the effectiveness of two delivery strategies: targeting the therapeutic gene directly to the liver or, preferably, to skeletal muscle. They discuss the implications of the different results they obtained with each approach.

"CNS1 is an outstanding model for in vivo gene therapy with easily measured and clinically relevant metabolic endpoints," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

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About the Journal

Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its sister journals, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of contents and sample issues for all three publications may be viewed on the Human Gene Therapy website at http://www.liebertpub.com/hgt.

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Long-term treatment success using gene therapy to correct a lethal metabolic disorder

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8-Oct-2014

Contact: Holly Ernest hernest@uwyo.edu 307-766-6605 University of California - Davis @ucdavis

Cut off by freeways and human development, mountain lions in southern California are facing a severe loss of genetic diversity, according to a new study led by the University of California, Davis in partnership with The Nature Conservancy.

The study, published today in the journal PLOS ONE, represents the largest genetic sampling of mountain lions, or pumas, in southern California. It raises concerns about the current status of mountain lions in the Santa Ana and Santa Monica mountains, as well as the longer-term outlook for mountain lions across southern California.

UC Davis School of Veterinary Medicine scientists collected and analyzed DNA samples from 354 mountain lions statewide, including 97 from southern California. Pumas in the Santa Ana Mountains displayed lower genetic diversity than those from nearly every other region in the state.

Santa Ana mountain lions show dramatic genetic isolation and have less in common with their neighbors in the Santa Monica Mountains than with those in the Sierra Nevada, underscoring the increasing seclusion of pumas in southern California.

The Santa Ana Mountain range, located south of Los Angeles and north of San Diego, is surrounded by urbanization and a growing population of about 20 million people. A small habitat linkage to the southeast connects pumas to the Peninsular Range, but it is bisected by Interstate 15 -- a busy 10-lane highway -- and associated human development. The study highlights the urgency to maintain and enhance the little connectivity remaining for coastal mountain lions, particularly across I-15.

The study also showed that the Santa Ana pumas recently went through a "population bottleneck," when the population's size sharply decreased to a fraction of its original size.

"The genetic samples give us a clear indication that there was a genetic bottleneck in the last 80 or so years," said lead author Holly Ernest, a professor with the Karen C. Drayer Wildlife Health Center and the Veterinary Genetic Laboratory at UC Davis at the time of the study. She is now a professor at the University of Wyoming, Laramie. "That tells us it's not just natural factors causing this loss of genetic diversity. It's us people impacting these environments."

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A highway runs through it: Mountain lions in southern California face genetic decay

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Genetics - The lamb lies down on Broadway - Coliseo - 04/10/2014
Genetics en el Teatro Coliseo el sabado 4/10/2014. Tributo al album "The lamb lies down on Broadway" en su 40 aniversario Imagenes: Panasonic FZ200 Audio: Z...

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Genetics - The lamb lies down on broadway-Fly on the Windshield - Live from Teatro Coliseo
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How to solve quantitative genetics problems

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Let #39;s play Sims 3 Perfect genetics challenge #4 C #39;est une fille !
Nous nous retrouvons pour un tout nouveaux let #39;s play sur les sims 3 le Prefect genetics challenge j #39;espere qu #39;il vous plaira n #39;hesitez pas a laisser vos avi...

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#95 Intro Genetics Artifacts (Vai ter v2) (Element 3D)
LEIA A DESCRO - Musica : Magnus H Tellmann Ancient Momento SKYPE : Auxy Dzn REQUISITOS : http://youtu.be/jxy64Dvba6g ============================...

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Conversation with Carolyn Hax: Ask Me About Health, Genetics and Dealing with Diseases
September 30, 2014 - Genomics and Our Health: What does the future hold? A closing symposium for the exhibition - Genome: Unlocking Life #39;s Code - that explores the potential of genomics to...

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French touch genetics douce nuit
French touch dropping them beans.

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Fertility Center Applied Genetics Sarasota Call (941) 342-1568 Exceptional New Review
http://GeneticsAndFertility.com 941-342-1568 Fertility Center Applied Genetics Sarasota reviews. Excellent Review We love Dr. Pabon and his entire staff! After 3 years of trying to get...

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9th Grade Biology: Genetics - Meiosis
We #39;ll be going over the basics of the process of genetic recombination and the creation of sex cells. It should be interesting...

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PUBLIC RELEASE DATE:

9-Oct-2014

Contact: Kevin Jiang kevin.jiang@uchospitals.edu 773-795-5227 University of Chicago Medical Center @UChicagoMed

The National Institute on Drug Abuse (NIDA) has awarded the University of Chicago a $12 million, five year grant to establish a national Center of Excellence to study drug abuse-associated behaviors by conducting research with rats.

Led by Abraham Palmer, PhD, associate professor of human genetics, the NIDA Center for Genome-Wide Association Studies in Outbred Rats will combine complex behavioral studies with recent technological advances in rat genetics to help scientists shed light on the genes behind drug addiction.

Rats have a long and storied history as an important animal model for research, especially in behavioral studies. But in recent decades, the use of rats has given way to mice because of innovations in the manipulation of mouse genomes. This shift has affected certain research fields, particularly the study of drug abuse and addiction, where behavioral tasks are often too complex for mice to perform. That's led to a slowdown in research aimed at revealing the genetics thought underlie drug abuse-related behaviors.

"The odds of permanently recovering from drug addiction are low and there is currently very little understanding of why that is," Palmer said. "With an animal system, we have a powerful advantage in that once we've found a genetic location or pathway, we can easily manipulate the gene and measure the resulting effects. The use of rats is critical because many of the behaviors we will study have proven difficult or impossible to adapt for mice."

A rat revival

To shed light on the genetics behind complex traits such as drug abuse behavior, the researchers will utilize genome-wide association studies (GWAS) an examination of the entire genomes of different individuals to reveal genetic variants linked with particular traits. Research groups around the country will perform experiments exploring separate behaviors, and send samples to UChicago for genetic analysis. This allows the center to study the genetics of multiple aspects of drug abuse efficiently and at a much more rapid pace than previously possible.

While most animal studies use almost genetically identical subjects, GWAS studies require large numbers of unrelated individuals. The center will support a comprehensive breeding program that provides researchers with a unique population of rats that have been bred to maintain as much genetic diversity as possible. Studies will be performed on both male and female rats to explore the relationship between gender, drug abuse behavior and genetics.

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University of Chicago establishes national center to study genetics of drug abuse in rats

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Amy Norton HealthDay Reporter Posted: Thursday, October 9, 2014, 5:00 AM

(HealthDay News) -- A new form of gene therapy may offer a safe and effective way to treat "bubble boy" disease -- a severe immune deficiency that is fatal unless treated in infancy.

Researchers have long known that gene therapy can cure the disease, known medically as severe combined immunodeficiency, or SCID. Over a decade ago, trials in Europe showed that gene therapy worked -- but five of the 20 children treated developed leukemia (a type of cancer) within two to five years, according to background information in the study.

In the new trial, reported in the Oct. 9 New England Journal of Medicine, researchers refined the gene therapy approach to hopefully negate the leukemia risk.

Eight of nine children who received the therapy are still alive one to three years later, the investigators report. And so far, none has developed leukemia.

It's too early to say the therapy carries no leukemia risk, cautioned researcher Dr. David Williams, a pediatric hematologist/oncologist at Dana-Farber Cancer Institute and Boston Children's Hospital.

"We'll continue to follow these children for 15 years," Williams said.

But based on the early results, he noted, the tweaked gene therapy appears as effective at generating a functional immune system as the earlier form of treatment.

SCID refers to a group of rare genetic disorders that all but eliminate the immune system, according to the Immune Deficiency Foundation (IDF). That leaves children at high risk of severe infections.

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Gene Therapy Shows Potential for 'Bubble Boy' Disease

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TIME Health Genetics Gene-Therapy Trial Shows Promise Fighting Bubble Boy Syndrome The immune system-related disease affects about 1 in 100,000 babies each year

A new gene-therapy treatment is showing promise in treating a rare and severe congenital condition that involves extreme immune-system deficiencies.

Bubble boy syndrome, an X-linked condition, takes its name from a famous case in which an affected boy, vulnerable to infection, lived inside a plastic bubble that protected him from the worlds germs. Outside of such sterile environments, babies with the syndrome seldom live longer than a year, the Wall Street Journal reports.

The condition has for decades bested medical researchers, despite occasional bouts of optimism hope for one previous gene-therapy treatment was felled when some recipients developed leukemia.

Gene-therapy treatment works, essentially, by replacing unperforming genes with functional ones. Dysfunctional cells are removed from the childs immune system and exposed to a genetically engineered virus that can reprogram the cells to function properly, explains Reuters. Those cells are then reinserted back into the patient.

In the earlier treatment, the virus to which the cells were exposed apparently activated a part of their genetic code that leads to leukemia, Reuters says.

But initial results reported in the New England Journal of Medicine show that none of the nine babies from the U.S. and Europe who received the latest treatment are exhibiting any signs of cancer.

Of the nine infant participants in the research who were between 4 and 10 months old when they began receiving the therapy eight were still alive 16 to 43 months later, without living in a protective bubble. (The ninth child died four months after treatment began from an earlier infection he had been fighting.)

Out of the eight boys still living, the treatment upped blood T-cell levels, rebuilding the immune system, of seven. In the case of the eighth child, the treatment did not rebuild his immune system, but a successful stem-cell transplant has kept him in improved health, Reuters reports.

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Date:

October 8, 2014

Source:

NIH/National Institute of Allergy and Infectious Diseases

Summary:

Gene therapy using a modified delivery system, or vector, can restore the immune systems of children with X-linked severe combined immunodeficiency (SCID-X1), a rare, life-threatening inherited condition that primarily affects boys, researchers have discovered.

Researchers have found that gene therapy using a modified delivery system, or vector, can restore the immune systems of children with X-linked severe combined immunodeficiency (SCID-X1), a rare, life-threatening inherited condition that primarily affects boys. Previous efforts to treat SCID-X1 with gene therapy were initially successful, but approximately one-quarter of the children developed leukemia two to five years after treatment. Results from a study partially funded by the National Institute of Allergy and Infectious Diseases (NIAID), a component of the National Institutes of Health (NIH), suggest that the new vector is equally effective at restoring immunity and may be safer than previous approaches.

In SCID-X1, mutations in a specific gene prevent the development of infection-fighting T cells. The standard therapy for SCID is transplantation of blood-forming stem cells, but some patients lack a suitable donor. In gene therapy, doctors remove stem cells from the patient's bone marrow, use a vector to insert a corrected gene and then return the corrected cells to the patient. Scientists suspect that the vectors used in earlier studies may have activated genes that control cell growth, contributing to leukemia.

In the current study, nine boys with SCID-X1 underwent gene therapy using a vector engineered by the study researchers. Seven boys developed functional T cells at levels comparable to those seen in previous studies and have remained healthy for one to three years after treatment. Analyses of the children's T cells suggest that the new vector causes fewer genomic changes that could be linked to leukemia. Researchers will continue to monitor the boys for leukemia development. Of the two other boys, one died of a pre-existing viral infection shortly after receiving the therapy, and one failed to develop corrected T cells and was given a stem cell transplant from an unrelated donor.

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Gene therapy shows promise for severe combined immunodeficiency

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Seminar Series - Dr. Susan Clarke on Regenerative Medicine in Orthopaedics

By: QUB School of Nursing Midwifery

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A combined LifeSource blood drive and Be The Match bone marrow registry event will honor two local women battling different forms of leukemia from 8 a.m. to 2 p.m. Saturday, Oct. 11, at The Society of Danube Swabians, 625 E. Seegers Road, Des Plaines.

The event will co-honor Des Plaines resident Amy Charewicz, 24, and Prospect Heights resident Anni Mayer, who is a Society of Danube Swabians board member. Mayer, a St. Alphonsus Ligouri parishioner and national society youth group leader, has Myelodyplastic Syndrome, a blood cancer that prevents her bone marrow from making enough healthy blood cells. Charewicz has aggressive Acute Myeloblastic Leukemia, a disease that causes her bone marrow to produce abnormal white and red blood cells, as well as platelets.

The Northern Illinois University graduate will receive a stem cell transplant Thursday from a 23-year-old man located by Be the Match at Northwestern Memorial Hospital. "Amy has been fortunate to have what she needs available because others have donated and registered," says Doris Charewicz, her mother. "She wants to 'pay it forward' by increasing awareness of the need for both blood donors and stem cell donors."

Be The Match registrants are limited to ages 18-44 and don't need to preregister. Blood donor walk-ins are welcome, but appointments are preferred at (877) 543-3768 or http://www.lifesource.org using code 650B.

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Bone marrow registry drive Saturday in Des Plaines

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