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Professor: Genetic mutations can amplify drug addictions

Published: Thursday, March 14, 2013 at 5:13 p.m. Last Modified: Thursday, March 14, 2013 at 5:13 p.m.

That some people are genetically prone to addictions is nothing new, but some scientists have expressed surprise at the degree of addictive tendencies.

For example, if you have a high-risk genotype for marijuana addiction, and also suffer from neuroticism or anxiety, you have an eight- to nine-fold risk of becoming addicted to marijuana.

What's more, in utero exposure to the drug might induce long-term addictive tendencies -- at least in rats.

Professor Yasmin Hurd, a neuroscientist and professor of psychiatry at the Ichan School of Medicine in Mount Sinai, N.Y., cited these examples Thursday in a talk titled "The Vulnerable Brain: Understanding the Neurobiology of Addiction Risk." Her talk was one of two expert lectures delivered Thursday at the McKnight Brain Institute as part of brain awareness week, a global campaign to raise awareness of the brain.

Early addiction research in neuroscience focused on dopamine dependence -- the "feel-good" chemical released abundantly in the brain during drug usage. The flip side of this immediate surge is a long-term lessening of dopamine actually produced by the brain, which over time, decreases a person's ability to experience pleasure.

But scientists realized addiction in the brain was "much more complicated" than decoding dopamine circuitry, so they began looking at genetic mutations that might play a role.

"Clearly there is not one gene that makes someone a heroin abuser," Hurd said, adding, "Genetics has an important for understanding the vulnerability of heroin abusers."

Hurd noted that marijuana is the most widely abused drug in the U.S., while heroin and cocaine are the most addictive. Heroin overdose has the highest mortality rates of any drug.

One issue that interests Hurd is the effect of marijuana use by pregnant women on their babies. The scientists found that men were more vulnerable to addictions than women. Other studies have shown that people -- especially men -- with certain behavioral traits such as anxiety, when combined with a genetic vulnerability to addiction, or a mother who smoked in utero, were especially at risk.

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Professor: Genetic mutations can amplify drug addictions

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Man’s DNA Test Rocks Human Genetic Theories – Video


Man #39;s DNA Test Rocks Human Genetic Theories
The research means our Y-chromosome tree is much longer than geneticists believed mdash;and that our paternal lineage is more than twice as old as we once thought.

By: slatester

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Man's DNA Test Rocks Human Genetic Theories - Video

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MinuteEarth: The Story of Our Planet – Video


MinuteEarth: The Story of Our Planet
Subscribe to MinuteEarth - it #39;s FREE! - http://dft.ba/-minuteearth_sub Agriculture, hula hoops, SARS, and THIS video: how long did they take to get around th...

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MinuteEarth: The Story of Our Planet - Video

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Savage – Only You (Harmless Project Remix) – Video


Savage - Only You (Harmless Project Remix)
Dar viena puikiai #382;inoma daina, kuri atgaivinta nauju skambesiu. Geriausios kokyb #279;s muzik #261; rasite #269;ia, prenumeruokite ir dalinkit #279;s! Nuotrauka rasite: http:/...

By: Muzikos i scaron;krova

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Savage - Only You (Harmless Project Remix) - Video

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How Mendel’s pea plants helped us understand genetics – Hortensia Jiménez Díaz – Video


How Mendel #39;s pea plants helped us understand genetics - Hortensia Jiménez Díaz
View full lesson: http://ed.ted.com/lessons/how-mendel-s-pea-plants-helped-us-understand-genetics-hortensia-jimenez-diaz Each father and mother pass down tra...

By: TEDEducation

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How Mendel's pea plants helped us understand genetics - Hortensia Jiménez Díaz - Video

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HUNTED BY MARILYN MANSON (The Hidden) – Video


HUNTED BY MARILYN MANSON (The Hidden)
Enjoy the video? Subscribe! http://bit.ly/M0mU1V #9669; #9669; #9669; Download Here: http://www.hidden-source.com What is The Hidden? "In the early 1950s human genetics e...

By: SeaNanners

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HUNTED BY MARILYN MANSON (The Hidden) - Video

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Education Resource Teaches Population Genetics Using Current Research

Newswise BETHESDA, MD March 13, 2013 Genetics and life sciences instructors, who teach undergraduate students about population and evolutionary genetics, have a new teaching resource: the March 2013 Primer in the Genetics Society of Americas journal GENETICS uses current research on transcriptome divergence in two closely related species of field crickets to explain population genetics.

The Primer, Population Genetics and a Study of Speciation Using Next-Generation Sequencing, by Patricia J. Wittkopp, Ph.D., a professor at the University of Michigan, explains how undergraduate instructors can, in their classrooms, use the article, Patterns of Transcriptome Divergence in the Male Accessory Gland of Two Closely Related Species of Field Crickets by Andrs et al., published in the February 2013 issue of GENETICS.

The Primer details background information on the Gryllus firmus and Gryllus pennsylvanicus cricket systems and the use of transcriptome sequence variation to study speciation. Dr. Wittkopp provides cogent explanations of the sequencing technologies used as well as some of the results of the paper, but leaves most of the results for students to interpret on their own. To give students the tools they need to interpret the data, Dr. Wittkopp provides a concise and accessible overview of the necessary genetics concepts on which the research of Andrs et al. is based. For instructors, Dr. Wittkopp provides guidance on how to use the primary literature in the classroom as well as questions for student discussion.

By focusing on contemporary scientific literature, students engage in the learning process and are encouraged to make their own scientific discoveries, said Elizabeth A. De Stasio, Ph.D., a professor at Lawrence University in Appleton, Wisconsin, and editor of the Primer section in the Genetics Society of Americas journal, GENETICS.

Primers are scheduled for the April, May, and June issues of GENETICS. Providing valuable educational resources like this, which enhance the quality of genetics education, teaching and learning, is a mission of GSA, said Mark Johnston, Ph.D., Editor-in-Chief of GENETICS. These articles help educators engage students in critically analyzing current primary research, a vital part of research training.

CITATION: Patricia J. Wittkopp. Population Genetics and a Study of Speciation Using Next Generation Sequencing: An Educational Primer for Use with Patterns of Transciptome Divergence in the Male Accessory Gland of Two Closely Related Species of Field Crickets GENETICS, March 2013, Volume 193, Number 3, 671-675.

ABOUT GENETICS Primers: Primers are designed to bring cutting-edge scientific research into the classroom by making scientific papers accessible to undergraduate students and their instructors. A Primer is intended to be used with the research article, which is published in the same or a recent issue of GENETICS. Primers include topic background, explanation of genetics concepts, suggestions for using the article in the classroom, and questions for classroom discussion. The articles give instructors the opportunity to enliven student interest in genetics by teaching genetics principles in the context of current research.

ABOUT GENETICS: Since 1916, GENETICS has covered high quality, original research on a range of topics bearing on inheritance, including population and evolutionary genetics, complex traits, developmental and behavioral genetics, cellular genetics, gene expression, genome integrity and transmission, and genome and systems biology. GENETICS, a peer-reviewed, peer-edited journal of the Genetics Society of America is one of the world's most cited journals in genetics and heredity.

ABOUT GSA: Founded in 1931, the Genetics Society of America (GSA) is the professional membership organization for scientific researchers, educators, bioengineers, bioinformaticians and others interested in the field of genetics. Its nearly 5,000 members work to advance knowledge in the basic mechanisms of inheritance, from the molecular to the population level. The GSA is dedicated to promoting research in genetics and to facilitating communication among geneticists worldwide through its conferences, including the biennial conference on Model Organisms to Human Biology, an interdisciplinary meeting on current and cutting edge topics in genetics research, as well as annual and biennial meetings that focus on the genetics of particular organisms, including C. elegans, Drosophila, fungi, mice, yeast, and zebrafish. GSA publishes GENETICS, a leading journal in the field and an online, open-access journal, G3: Genes|Genomes|Genetics. For more information about GSA, please visit http://www.genetics-gsa.org. Also follow GSA on Facebook at facebook.com/GeneticsGSA and on Twitter @GeneticsGSA.

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Education Resource Teaches Population Genetics Using Current Research

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Education resource focuses on teaching population genetics using current research

Public release date: 13-Mar-2013 [ | E-mail | Share ]

Contact: Phyllis Edelman pedelman@genetics-gsa.org 301-634-7302 Genetics Society of America

BETHESDA, MD March 13, 2013 Genetics and life sciences instructors, who teach undergraduate students about population and evolutionary genetics, have a new teaching resource: the March 2013 Primer in the Genetics Society of America's journal GENETICS uses current research on transcriptome divergence in two closely related species of field crickets to explain population genetics.

The Primer, "Population Genetics and a Study of Speciation Using Next-Generation Sequencing," by Patricia J. Wittkopp, Ph.D., a professor at the University of Michigan, explains how undergraduate instructors can, in their classrooms, use the article, "Patterns of Transcriptome Divergence in the Male Accessory Gland of Two Closely Related Species of Field Crickets" by Andrs et al., published in the February 2013 issue of GENETICS.

The Primer details background information on the Gryllus firmus and Gryllus pennsylvanicus cricket systems and the use of transcriptome sequence variation to study speciation. Dr. Wittkopp provides cogent explanations of the sequencing technologies used as well as some of the results of the paper, but leaves most of the results for students to interpret on their own. To give students the tools they need to interpret the data, Dr. Wittkopp provides a concise and accessible overview of the necessary genetics concepts on which the research of Andrs et al. is based. For instructors, Dr. Wittkopp provides guidance on how to use the primary literature in the classroom as well as questions for student discussion.

"By focusing on contemporary scientific literature, students engage in the learning process and are encouraged to make their own scientific discoveries," said Elizabeth A. De Stasio, Ph.D., a professor at Lawrence University in Appleton, Wisconsin, and editor of the Primer section in the Genetics Society of America's journal, GENETICS.

Primers are scheduled for the April, May, and June issues of GENETICS. "Providing valuable educational resources like this, which enhance the quality of genetics education, teaching and learning, is a mission of GSA," said Mark Johnston, Ph.D., Editor-in-Chief of GENETICS. "These articles help educators engage students in critically analyzing current primary research, a vital part of research training."

###

CITATION: Patricia J. Wittkopp. Population Genetics and a Study of Speciation Using Next Generation Sequencing: An Educational Primer for Use with "Patterns of Transciptome Divergence in the Male Accessory Gland of Two Closely Related Species of Field Crickets" GENETICS, March 2013, Volume 193, Number 3, 671-675.

ABOUT GENETICS Primers: Primers are designed to bring cutting-edge scientific research into the classroom by making scientific papers accessible to undergraduate students and their instructors. A Primer is intended to be used with the research article, which is published in the same or a recent issue of GENETICS. Primers include topic background, explanation of genetics concepts, suggestions for using the article in the classroom, and questions for classroom discussion. The articles give instructors the opportunity to enliven student interest in genetics by teaching genetics principles in the context of current research.

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Education resource focuses on teaching population genetics using current research

Recommendation and review posted by Bethany Smith

Atossa Genetics Announces National Agreement With Network Provider FedMed

SEATTLE, WA--(Marketwire - Mar 13, 2013) - Atossa Genetics, Inc. ( NASDAQ : ATOS ), The Breast Health Company, has entered into a contractual agreement with FedMed, Inc., one of the largest proprietary Preferred Provider Organization (PPO) networks in the U.S., for diagnostic laboratory testing. FedMed's network is comprised of more than 550,000 providers, including 4,000 hospitals and more than 60,000 ancillary facilities, serving over 40 million Americans.

Atossa's agreement with FedMed will give FedMed's participating providers and its clients' members greater access to Atossa's tests, including the ForeCYTE Breast Health Test and the ArgusCYTE Breast Health Test.

"There is a significant unmet clinical need in the medical community for more effective ways to identify women at high risk of breast cancer," stated Steven C. Quay, M.D., Ph.D., FCAP, Chairman, CEO & President of Atossa Genetics. "Our agreement with FedMed will help ensure that more doctors and their patients have access to the ForeCYTE Breast Health Test, a risk stratification test akin to the cervical Pap smear, and the ArgusCYTE Breast Health Test, a blood test for recurrence targeted at the 3.2 million breast cancer survivors in the U.S."

Dr. Quay added, "At Atossa, our goal is to help physicians manage their patients' breast health by offering a suite of products and services that address the most pressing clinical decisions. Armed with better information, physicians will be able to customize individualized treatment plans to reduce women's risk of breast cancer or cancer recurrence, to improve patient therapeutic compliance and ultimately to decrease the overall cost of care."

About Atossa Genetics, Inc.

Atossa Genetics, Inc. ( NASDAQ : ATOS ), The Breast Health Company, is based in Seattle, WA, and is focused on preventing breast cancer through the commercialization of patented diagnostic medical devices and patented, laboratory developed tests (LDT) that can detect precursors to breast cancer up to eight years before mammography, and through research and development that will permit it to commercialize treatments for pre-cancerous lesions.

The National Reference Laboratory for Breast Health (NRLBH), a wholly owned subsidiary of Atossa Genetics, Inc., is a CLIA-certified high-complexity molecular diagnostic laboratory located in Seattle, WA, that provides the patented ForeCYTE Breast Health Test and the ArgusCYTE Breast Health Test.

About the ForeCYTE Breast Health Test

The ForeCYTE Breast Health Test provides personalized information about the 10-year and lifetime risk of breast cancer for women between ages 18 and 73. It involves collecting a specimen of nipple aspirate fluid, or NAF, using our patented Mammary Aspirate Specimen Cytology Test, or MASCT, System. The NAF specimen is collected by a physician and returned to our CLIA-certified laboratory. We study the patient's NAF specimen and use a proprietary molecular and cellular biomarker test that detects basal or luminal cells to identify the presence of atypical ductal hyperplasia, or ADH, which is considered a precursor to breast cancer.If ADH is detected, steps can be taken to reduce the risk of breast cancer.

About the ArgusCYTE Breast Health Test

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Atossa Genetics Announces National Agreement With Network Provider FedMed

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Gene therapy helped mice withstand osteoarthritis

By Randy Dotinga HealthDay Reporter

THURSDAY, March 14 (HealthDay News) -- In a very early sign of medical progress on the osteoarthritis front, scientists report they've used injections of modified genes to reduce the risk that mice will develop the painful, debilitating condition.

There's no way to know if the gene therapy treatment will help humans, and scientists are far from understanding the treatment's side effects and potential cost. But the findings are more than just good news for mice with creaky joints.

"This work identifies an approach that can make a difference," explained study co-author Dr. Brendan Lee, director of the Rolanette and Berdon Lawrence Bone Disease Program of Texas. "There's a great need for treating and preventing osteoarthritis."

The disease, the most common form of arthritis, appears as your joints deteriorate with aging. It often strikes the hands, knees, neck and hips, causing pain, stiffness and difficulty moving.

Seventy percent of Americans aged 55 to 70 struggle with osteoarthritis, for which there is no cure. Doctors try to treat the pain and improve the ability of patients to move, Lee said, and may turn to joint replacement surgeries in advanced cases.

In the new study, researchers examined a protein that diminishes in people with a rare joint disorder. The protein appears to be crucial to the lubrication of joints.

Researchers injected a gene related to the protein into mice and found that the rodent bodies began producing it. The mice appeared to be resistant -- but not immune -- to damage to the cartilage of joints from injury and aging, Lee said.

There are plenty of caveats.

The research is in mice, not humans; the next step is to test the approach in horses, whose joints are similar to those of people. And the gene therapy doesn't seem to do anything for damage that's already occurred.

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Gene therapy helped mice withstand osteoarthritis

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Families of SMA and Nationwide Children’s Announce Multi-Million Dollar Award From NINDS to Advance CNS Gene Therapy …

ELK GROVE VILLAGE, Ill., March 13, 2013 (GLOBE NEWSWIRE) -- Nationwide Children's Hospital (Columbus, OH) and Families of Spinal Muscular Atrophy (Elk Grove Village, IL) announce the award of a multi-million dollar cooperative agreement from the National Institute of Neurological Disorders and Stroke (NINDS) to advance a gene therapy development program for Spinal Muscular Atrophy (SMA).

This three-year multi-million dollar cooperative agreement to Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children's Hospital in the amount of $3,752,462, funds pre-clinical drug development up to the filing of an Investigational New Drug Application (IND) to the Food and Drug Administration (FDA). This agreement represents an innovative collaboration between Government, Advocacy and Academic groups to advance a promising new therapy for SMA.

In May 2012, Families of SMA (FSMA) announced the award of up to $750,000 to Dr. Kaspar. This ongoing award supports the preclinical development of a Central Nervous System (CNS)-delivered gene therapy for SMA. Direct CNS delivery likely allows for less virus to be used, which significantly increases the likelihood that older and larger SMA patients can be treated with gene therapy. With the funding from FSMA, Dr. Kaspar's team initiated studies to jumpstart the research prior to obtaining government and later commercial involvement. This cooperative award from the NINDS will now support advancing the program to the point of human clinical trials. The program will be evaluated using quantitative go/no-go milestones, determined by Nationwide Children's and NINDS.

SMA is an often-fatal genetic disorder resulting from the loss of both copies of the Survival Motor Neuron (SMN1) gene. This causes a chronic deficiency in the production of the SMN protein, which is essential to the proper functioning of the motor neurons in the spinal cord to the control of muscles in the limbs, neck and chest. SMA is typically marked by the deterioration of the muscles that control crawling, walking, swallowing or breathing. There are no approved therapies for the treatment of SMA. Approximately 1 in 6,000 babies born is affected. One in 40 people, or approximately 8 million in the United States, are genetic carriers of the disease.

Gene therapy is an approach to treating diseases by replacing faulty genes. In the case of SMA, the most direct approach for a gene therapy is to replace the mutated SMN1 gene. In the past, the challenge with gene therapy for SMA has been to find a way to deliver the genetic material efficiently to motor neurons. In recent years, Dr. Kaspar's group was the first to demonstrate Adeno-Associated Virus 9 (AAV9) targeted motor neurons effectively. Administration of AAV9-SMN into one day-old SMA mice resulted in increased SMN protein levels in motor neurons, correction of synaptic function, and a significant extension of life span.

"At Families of SMA we are extremely pleased that our initial investment at an early stage of this program has provided the preliminary data to leverage larger funding from the NIH. We feel this grant award is positive validation of the Families of SMA research funding and partnering strategy, as well as for this approach for gene therapy in SMA," said Jill Jarecki PhD, Research Director at Families of SMA. "The Families of SMA funding strategy for preclinical drug development is to invest seed funds to begin early-stage programs for SMA. As programs advance, we look for funding to transition from non-profit to government and commercial sources."

"My research team at Nationwide Children's Hospital is excited to advance this promising cerebrospinal fluid delivery approach of AAV9-SMN to the clinic for SMA patients and we are extremely grateful to FSMA and NINDS for the support of this important work," said Dr. Kaspar, also a faculty member at The Ohio State University College of Medicine. "We stand committed to bring SMA experimental therapeutics to the clinic in the most rapid and safe manner."

"Development of therapies requires collaboration of academics, advocacy, industry, and government--no single party has the resources to do this alone. The collaboration between Dr. Brian Kaspar, Families of SMA, and the NIH is an exciting model in leveraging resources and expertise in the hope of accelerating therapy development for SMA," said Dr. John Porter, PhD, Program Director at the National Institute of Neurological Disorders and Stroke.

About Families of SMA:

Families of SMA is the world's leader focused on funding SMA research to develop a treatment and cure for the disease. The successful results and progress that the organization has delivered, from basic research to drug discovery to clinical trials, provide real hope for families and patients impacted by the disease. The charity has invested over $55 million in research and has been involved in funding half of all the ongoing novel drug programs for SMA. Families of SMA is a nonprofit 501(c)3 organization, with 31 Chapters and 90,000 members and supporters throughout the United States. The organization's work has produced major discoveries, including identification of the underlying cause and a back-up gene for the disease, which provides a clearly defined target for disease altering therapies. The organization is also dedicated to supporting SMA families through networking, information and services and to improving care for all SMA patients. http://www.curesma.org.

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Families of SMA and Nationwide Children's Announce Multi-Million Dollar Award From NINDS to Advance CNS Gene Therapy ...

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Gene therapy studies show potential for HIV control without drugs

In the first paper, researchers from the Fred Hutchinson Cancer Research Institute in Seattle took haematopoeic stem cells (HSCs), bone-marrow cells that are the progenitors of all blood cells, from two pigtail macaque monkeys and transformed them genetically by splicing an inserted gene sequence for mC46 into their CCR5 receptor gene.

They then injected the cells back into the monkeys. One monkey had 20%of its HSCs replaced by the C34-producing cells and the second had over 50% replaced.

A week later, they infected them and two control monkeys with a particularly lethal strain of genetically engineered human/monkey SHIV, which destroys CD4 cells fast and usually develops a steady-state viral load in the order of several million copies/ml.

In the control animals, their CD4 counts declined from around 600 cells/mm3 before infection to between 10 and 50 cells/mm3 within two to three weeks. In the monkeys with mC46, the CD4 cell count dipped to about 100 cells/mm3 within two weeks of infection, but then rose slowly back to pre-infection levels over the next six months.

At the time of highest SHIV viral load and fewest CD4 cells, 90% of the CD4 cells in the mC46 monkeys had the fusion-inhibitor-generating insert in them, which is what one would expect, given that SHIV so decimates non-mutated CD4 cells.

What was unexpected to the scientists, though, was that after the period of peak viral load, the non-mutated CD4 cells made a partial recovery in one monkey to about 60% of all CD4 cells and in the other about 20%. This is promising, as it shows that one would not need to replace all or even most of the CD4 cells in the body with HIV-resistant ones in order to contain an HIV infection, and that increasing numbers of non-resistant cells does not lead to a new burst of virus.

This may also mean that it would not be necessary to take the dangerous step of having to destroy a persons immune system with whole-body radiation, as happened to the 'Berlin patient' Timothy Ray Brown, in order for the new cells to repopulate the immune system.

As we said, SHIV reproduces furiously and peak viral load in all monkeys ten days after infection was one billion copies/ml. After that, viral load in the control monkeys declined to about half a million in one and about ten million in the other. In the mC46 monkeys, it fell to about 100,000 in the monkey with 20% of its cells replaced by mC46 cells and down to a few hundred in the one with more than 50% of its cells replaced.

Viral load was about 320-fold lower (2.5 logs) in the first monkey and about 1400-fold (3.15 logs) lower in the second. This would transform a typical HIV viral load in an untreated human of 70,000 copies/ml to 50 copies/ml before any ARVs were taken.

Although there was a partial recovery of unmutated CD4 cells in the blood, memory cells in the lymph nodes that form the reservoir of proviral HIV DNA remained predominantly the HIV-resistant mC46 cells, which is exactly where one would want them to be.

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Gene therapy studies show potential for HIV control without drugs

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Gene Therapy Could Prevent Arthritis

By Lisa Raffensperger | March 13, 2013 1:22 pm

Most people who live to old age will suffer from arthritis. The conditions prevalence is growing alongside a graying world population.

However the only treatments at the moment address the symptoms rather than the causethe loss of cartilage in joints. Joint replacement is a last-ditch solution for some sufferers. Now a gene therapy approach has demonstrated promise in staving off arthritis in mice, opening the door to human testing.

The inspiration for the research came from studying children with a genetic form of arthritis that strikes early. These children are deficient in the gene for a protein called lubricin. Lubricin is thought to act as a lubricant between the bones in a joint.

Since a lack of lubricin caused arthritis, researchers thought perhaps additional lubricin could stave it off.

They tested this hypothesis by creating a strain of mice with an additional lubricin gene in their DNA. When these mice suffered an injury to their knees they didnt develop injury-induced arthritis. Inspection of the mices joints found that their cartilage resembled mice whod never been injured in the first place. Non-modified mice, on the other hand, had symptoms of arthritis just a month after injury.

Whats more, as the mice that made extra lubricin aged, their cartilage stayed youthful. That suggests the protein may protect against both common forms of arthritis: injury-related and age-related.

The treatment also works if the replacement genes are injected right into the joint itself, the researchers report in Science Translational Medicine today. Its delivery to human patients, then, could be similar to the injection of joint lubricants that some arthritis sufferers currently rely on.

However no gene therapies are currently approved by the FDA for human treatment, so this research will likely stay in the lab for some time yet.

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Gene Therapy Could Prevent Arthritis

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Research and Markets: Gene Therapy : Technologies, Markets and Companies 2013 Update with Added Company Profiles

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/2nfn6s/gene_therapy) has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering.

Gene therapy can be broadly defined as the transfer of defined genetic material to specific target cells of a patient for the ultimate purpose of preventing or altering a particular disease state. Genes and DNA are now being introduced without the use of vectors and various techniques are being used to modify the function of genes in vivo without gene transfer. If one adds to this the cell therapy particularly with use of genetically modified cells, the scope of gene therapy becomes much broader. Gene therapy can now combined with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2012, over 2030 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the areas of application.

Since the death of Jesse Gelsinger in the US following a gene therapy treatment, the FDA has further tightened the regulatory control on gene therapy. A further setback was the reports of leukemia following use of retroviral vectors in successful gene therapy for adenosine deaminase deficiency. Several clinical trials were put on hold and many have resumed now. The report also discusses the adverse effects of various vectors, safety regulations and ethical aspects of gene therapy including germline gene therapy.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2012-2022. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets

Profiles of 179 companies involved in developing gene therapy are presented along with 203 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report. John Wiley & Sons published the book in 2000 and from 2001 to 2003, updated versions of these companies (approximately 160 at mid-2003) were available on Wiley's web site. Since that free service was discontinued and the rights reverted to the author, this report remains the only authorized continuously updated version on gene therapy companies.

Benefits of this report

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Research and Markets: Gene Therapy : Technologies, Markets and Companies 2013 Update with Added Company Profiles

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Stem Cell Treatment for Spinal Cord Injury at the Stem Cell Institute in Panama – Daniel Campbell – Video


Stem Cell Treatment for Spinal Cord Injury at the Stem Cell Institute in Panama - Daniel Campbell
After stem cell therapy at the Stem Cell Institute in Panama, Daniel Campbell describes how his condition has improved since his first treatment. Daniel is i...

By: cellmedicine

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Stem Cell Treatment for Spinal Cord Injury at the Stem Cell Institute in Panama - Daniel Campbell - Video

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Dr Alok Sharma Stem Cell Therapy Treatment for Muscular Dystrophy – Video


Dr Alok Sharma Stem Cell Therapy Treatment for Muscular Dystrophy
Dr Alok Sharma Stem Cell Therapy Treatment for Muscular Dystrophy She is a known case of MD with history of gradual onset of progressive lower extremities muscle weakness noticed since 27 years of age with complaints of imbalance while walking foot drop and difficulty in stair case climbing So she was investigated and muscle biopsy confirmed diagnosis of MD After Stem Cell Therapy 1 Stamina has increased Exercise tolerance has improved 2 She can lift her leg more up while in standing 3 Hip ...

By: neurogenbsi

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Dr Alok Sharma Stem Cell Therapy Treatment for Muscular Dystrophy - Video

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Dr Alok Sharma Stem Cell Therapy Treatment for Cerebral Palsy – Video


Dr Alok Sharma Stem Cell Therapy Treatment for Cerebral Palsy
Dr Alok Sharma Stem Cell Therapy Treatment for Cerebral Palsy He is a known case of Diplegic CP with history of full term normal delivery (vaccum) delivery a...

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Dr Alok Sharma Stem Cell Therapy Treatment for Cerebral Palsy - Video

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Stem Cell Therapy Now In Davao

With its growing popularity in the country, the so-called stem cell therapy, which was recently approved by the Department of Health (DOH), is now available in Davao City.

Dr. Luz Acosta, a Davao-born physician specializing in Ophthalmology, Oculoplastic Reconstructive Surgery, Cosmetic Surgery and recently Stem Cell Treatment, conducts the procedure after acquiring equipment from Australia and the USA. She is supported by a team that includes cardiologists, internists, and anesthesiologists who administer the laboratory tests and cardiopulmonary clearance to determine if an individual can safely undergo stem cell therapy. Having trained in this discipline abroad, she recently decided to offer this treatment after it was approved by the DoH.

As a founding member of the Philippine Society for Stem Cell Medicine that was only established last January 18, 2013 with DOH Sec. Dr. Enrique Ona as its honorary chair, Acosta is joined by doctors Jose Sabili, Melchor Santos, Christian Mancao, Leo Olarte, Bu Castro, Oscar Tinio, and Almond Derla as well as Mr. Rico Colayco as organizers of the society.

"This means that the Philippine Society for Stem Cell Medicine is the regulating body for the practice of stem cell treatment in the country," Acosta says. "Stem cell therapy is legal and safe for every medically cleared patient who wants to undergo such treatment to be rejuvenated and treated of his/her illnesses and diseases."

Acosta said that based on what was stipulated in the approved directions for stem cell therapy, the stem cell could just come from the person himself and not from the other sources like black sheep or aborted fetus, adding that sources of stem cells are fats, blood, bone marrow and umbilical cord.

"It could take five to six hours for one to undergo stem cell therapy. Harvesting is done in the first two hours, then another one to two hours for stem cell processing and activation, and the last one to two hours for treatment of stem cells back to the same patient," she said.

Popular personalities who have already publicly admitted that they had undergone stem cell therapy are former President Joseph Estrada, Senate President Juan Ponce Enrile and former Senator Ernesto Maceda, who said that they spent millions to pay for the treatment.

But Acosta said that it could be a lot cheaper here than abroad, as the cost of treatment will be in accordance to the guidelines of the Phil. Society for Stem Cell Medicine.

She said there have been very good clinical outcomes from stem cell treatments on autism, auto-immune diseases, cerebral palsy, diabetes, heart disease, liver cirrhosis, macular degeneration, multiple sclerosis, nerve damage, osteoarthritis, spinal cord injury and stroke.

"But we have to clarify here that we are not claiming stem cell therapy as a cure for cancer, though it can alleviate pain and improve patients' wellbeing while undergoing cancer therapy," she said.

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Stem Cell Therapy Now In Davao

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State Sen. Gene Yaw re-elected board chairman for Center for Rural Pennsylvania

HARRISBURG The Center for Rural Pennsylvania has re-elected state Senator Gene Yaw, R-23 of Lycoming County, to serve as Chairman of its 11 member Board of Directors.

The Center is a bipartisan, bicameral legislative agency that serves as a resource for rural policy within the Pennsylvania General Assembly.

I am again very humbled to be re-elected Chairman of the Board for the Center for Rural Pennsylvania, said Yaw. As a state Senator and resident of rural Pennsylvania, I find the research and reports conducted and distributed by the Center for Rural Pennsylvania invaluable to our work in Harrisburg and also to many individuals, groups and organizations in my district, and across the Commonwealth.

The Center works with the legislature, educators, state and federal executive branch agencies, and national, statewide, regional and local organizations to maximize resources and strategies that can better serve Pennsylvanias 3.4 million rural residents.

Approximately 27 percent of the states 12.7 million residents live in 48 rural counties, Yaw added. By 2030, Pennsylvania rural counties are projected to have a total population of 3.57 million people, a 3 percent increase from 2010. This further amplifies the importance of the work provided by the Center for Rural Pennsylvania.

Created in 1987 under Act 16, the Rural Revitalization Act, the Center for Rural Pennsylvania promotes and sustains the vitality of Pennsylvanias rural and small communities by:

- Sponsoring research projects to identify policy options for legislative and executive branch consideration and action

- Collecting data on trends and conditions to understand the diversity of rural Pennsylvania

- Publishing information and research results to inform and educate audiences about the diverse people and communities of rural Pennsylvania; and

- Participating in local, state and national forums on rural issues to present and learn from best practices.

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State Sen. Gene Yaw re-elected board chairman for Center for Rural Pennsylvania

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Horse Gene Discovery Points to Asthma Relief: Guelph Study

March 12, 2013 - News Release

A discovery by University of Guelph researchers will help in understanding how horses develop recurrent airway obstruction (RAO) and offers hope of potential solutions for people with asthma.

In a paper in a recent issue of BMC Genomics, the researchers discuss their discovery that horses have three copies of a gene normally found as a single copy in mammals. This gene, called secretoglobin family 1A member 1 (SCGB-1A1), produces a protein secreted in large amounts in the airway.

RAO is a chronic inflammatory lung disease. It is especially prevalent during winter in horses kept in barns and fed hay. The researchers found that RAO-susceptible horses have much less SCGB1A1 protein in their airways, which enhances inflammation.

Symptoms in horses with RAO resemble those of humans with environmentally induced asthma.

The researchers found that two of the gene copies could play a significant role in treating RAO. The third copy has no recognized function and may have evolved into a pseudo-gene.

Lead author Olivier Ct, a PhD candidate in the Department of Pathobiology, says the study could have larger implications than treating horses.

Were able to use the horse as a model for asthma in humans, said Ct. We found through our research that horses suffering from RAO had reduced SCGB1A1 levels. Since an obvious suggestion for treating RAO is to increase protein levels of SCGB1A1, we made a synthetic version of it in the lab. We are currently testing the proteins function. While it would not be possible to simply provide humans with this protein to reduce asthma, as humans and horses are different species, these findings do give hope that we can find novel treatments for asthma.

Study co-author Prof. Dorothee Bienzle said it was challenging to isolate and assess the individual genes. The researchers also faced challenges because of the unusual nature of the gene triplication.

Other mammals do not have multiple SCGB1A1 copies, except for some other equidae, such as Przewalskis horses and donkeys, she said. So it is difficult to know where the gene came from. We can speculate that it was an evolutionary response that took place over many years. We dont know why the pseudo-gene exists or what its purpose is. The distribution of the other two gene transcripts and proteins indicates they are extremely prevalent in the lung and reproductive organs.

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Horse Gene Discovery Points to Asthma Relief: Guelph Study

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GIANT GENETIC MUTANT RATS 2013 – Video


GIANT GENETIC MUTANT RATS 2013
WERE THEY RELEASED THERE? ARE THEY INFECTED WITH HORRIBLE DISEASES? THIS SOUNDS VERY SUSPICIOUS TO ME! LINK: http://www.ibtimes.co.uk/articles/437326/2013022...

By: faceintree

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GIANT GENETIC MUTANT RATS 2013 - Video

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Synthetic biologists standardize genetic parts to engineer cells

Palo Alto and Berkeley, Calif. March 10, 2013 An unprecedented collaboration among academia, industry, government and civil society has resulted in the launch of a professional-grade collection of public domain DNA parts that greatly increases the reliability and precision by which biology can be engineered. Researchers at the International Open Facility Advancing Biotechnology (aka, BIOFAB) have just announced that they have, in effect, established rules for the first language for engineering gene expression, the layer between the genome and all the dynamic processes of life. The feat is all the more remarkable considering that just a few years ago several prominent scientists claimed that it would be impossible to develop frameworks enabling reliably reusable standard biological parts. Collectively, the BIOFAB team has produced thousands of high quality standard biological parts. The DNA sequences that encode all parts and the data about them are free and available online.

The project is detailed in three research papers, Precise and Reliable Gene Expression via Standard Transcription and Translation Initiation Elements, and Quantitative Estimation of Activity and Quality for Collections of Functional Genetic Elements, published simultaneously in Nature Methods, and Measurement and Modeling of Intrinsic Transcription Terminators, forthcoming in Nucleic Acids Research (see full citations below).

The BIOFABs rules for engineering expression come in the form of mathematical models that can be used to predict and characterize the individual parts used in synthetic biology. The work establishes a much-needed technological foundation for the field, allowing researchers to engineer the function of DNA more precisely, and to better predict the resultant behavior.

Dr. Vivek Mutalik, a BIOFAB team leader, says that synthetic biology has been plagued by a lack of reliability and predictability. Until now, virtually every project has been a one-off we havent figured out how to standardize the genetic parts that are the building blocks of this new field. Researchers produce amazing new parts all the time, but much like trying to use someone elses house key in your own door, its been difficult to directly reuse parts across projects. Without the ability to characterize parts that is, to understand how they will behave in multiple contexts biotech researchers are doomed to a lengthy process of trial-and-error. Fortunately, notes Mutalik, Our work in the BIOFAB changes all that.

The plan for establishing the rules for how genetic parts fit together was ambitious and complex. First, researchers needed to figure out the functional patterns of genetic parts. They had to ask, To what extent do the basic genetic parts that control gene expression misbehave when reused over and again in novel combinations, said Mutalik. BIOFAB researchers had to make and test hundreds of combinations of frequently used parts, then take the resulting data and build mathematical models that demonstrated part quality. Joao Guimaraes, a member of the BIOFAB team and graduate student in computational biology, explains that difficult-to-predict parts are deemed to be low quality, while high quality parts behave the same when reused. Once they found a way to determine part quality, the BIOFAB team set to work on establishing rules for precision control of gene expression, a process that underlies all of biotechnology. They learned by observing natural examples of genetic junctions, and built reliable transcription and translation initiation elements. We also created standard junctions for transcription terminators, a molecular stop sign for gene expression, said Dr. Guillaume Cambray, a BIOFAB team leader.

While the initial BIOFAB project was able to tame three types of core genetic parts, much more work remains. We ask that others expand upon the genetic grammar initiated here, to incorporate additional genetic functions and to translate the common rule set beyond E. coli, says Stanford professor and BIOFAB co-director Drew Endy. (Endy also serves as president of the BioBricks Foundation.)

The BIOFABs seed money came from the National Science Foundation, but this funding came only after 10 years of knocking on doors. Part of the difficulty was that the BIOFAB represented a fundamental engineering research project. Its not the kind of work that is suitable for a single graduate student thesis, and it wasnt economically practical for a biotechnology company to take it on. UC Berkeley professor and BIOFAB co-director Adam Arkin noted that, We knew that we would only be successful if we could bring together the skills represented by both academia and industry to establish a professional team that could specify and solve the fundamental engineering puzzles that slow the development of effective biotechnologies

The BIOFABs collaboration with not only the NSF, but also with industry, has been one of the keys to its success. Pre-competitive and unrestricted partnerships with industry were essential to guide the work and help secure and extend public funding, said UC Berkeley professor and BIOFAB advisor Jay Keasling. (Both Arkin and Keasling are also affiliated with Lawrence Berkeley Lab; Arkin is Director of the Physical Biosciences Division, and Keasling is an Associate Lab Director for Biosciences.)

Other partners came from civil society, including the BioBricks Foundation, a public-benefit organization that helps to advance best practices in the emerging field of synthetic biology. We were thrilled to help make all BIOFAB engineered parts free-to-use via the BioBrick Public Agreement and the public domain, said Holly Million, the foundations executive director.

The BIOFABs standardized parts are specific for E. coli but the grammar the way in which the rules are constructed for how the parts fit together should apply to nearly any organism; many of the BIOFABs rules for E.coli are expected to apply to other prokaryotes.

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Synthetic biologists standardize genetic parts to engineer cells

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Can energy drinks improve the physical and mental performance of cyclists?

Public release date: 11-Mar-2013 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, March 11, 2013Consumption of energy drinks containing caffeine may have beneficial effects on exercise but probably not for mental function. The effects of pre-exercise caffeine consumption by trained cyclists on racing times and cognitive performance were measured and are reported in Journal of Caffeine Research, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Journal of Caffeine Research website at http://www.liebertpub.com/jcr.

Race performance improved for all study participants after consuming an energy drink, even if they already had an elevated blood caffeine level before the energy drink. Cycling times improved by an average of 3% for the group. David Gray Lassiter and coauthors from University of Texas at Austin also reported improvements in certain aspects of cognitive function, but these were probably not due to the energy drink. They present their findings in the article "Effect of an Energy Drink on Physical and Cognitive Performance in Trained Cyclists."

"While it is not certain from this one study whether energy drink improves physical performance, the study is important in pointing the way to further research in this area," says Jack E. James, PhD, Editor-in-Chief of Journal of Caffeine Research.

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About the Journal

Journal of Caffeine Research: The International Multidisciplinary Journal of Caffeine Science is a quarterly journal published in print and online that covers the effects of caffeine on a wide range of diseases and conditions, including mood disorders, neurological disorders, cognitive performance, cardiovascular disease, and sports performance. The Journal explores all aspects of caffeine science including the biochemistry of caffeine; its actions on the human body; benefits, dangers, and contraindications; and caffeine addiction and withdrawal, across all stages of the human life span from prenatal exposure to end-of-life. Tables of content and a sample issue may be viewed on the Journal of Caffeine Research website at http://www.liebertpub.com/jcr.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Breastfeeding Medicine, Journal of Medicinal Food, and Journal of Child and Adolescent Psychopharmacology. 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 70 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website at http://www.liebertpub.com.

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Can energy drinks improve the physical and mental performance of cyclists?

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Low-cost nano-biosensor to detect foodborne pathogen that causes listeriosis

Public release date: 12-Mar-2013 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, March 12, 2013The foodborne bacteria Listeria monocytogenes sickens about 2,500 people in the U.S. each year and many more worldwide, killing about 25-30% of those infected. Listeriosis is caused by eating food contaminated with L. monocytogenes, and current methods for detecting the bacteria are costly and time consuming. An innovative nanotechnology-based method for developing an inexpensive biosensor to detect the pathogen in food is described in Industrial Biotechnology, a peer-reviewed journal from Mary Ann Liebert Inc., publishers (http://www.liebertpub.com). The articles are available free on the Industrial Biotechnology (http://www.liebertpub.com/ind) website.

Vivian C.H. Wu, PhD led a group of scientists from University of Maine (Orono), National Chio Tung University, and Apex Biotechnology Corp. (Hsinchu, Taiwan), in producing a highly specific, antibody-based immunobiosensing strip with the potential for low-cost commercial development. Danielle Davis, et al. describes their work in the article "Gold Nanoparticle-Modified Carbon Electrode Biosensor for the Detection of Listeria monocytogenes (http://online.liebertpub.com/doi/full/10.1089/ind.2012.0033)."

The article is part of an IB Special Section on Nanobiotechnology, Part 2, led by Co-Guest Editors Norman Scott, PhD, Professor, Cornell University (Ithaca, NY) and Hongda Chen, PhD, National Program Leader, National Institute of Food and Agriculture, USDA (Washington, DC). In their Overview article "Nanoscale Science and Engineering for Agriculture and Food Systems (http://online.liebertpub.com/doi/full/10.1089/ind.2013.1555)," they describe the emerging opportunities and challenges for nanotechnology and nanomaterials research in industrial biotechnology.

The special section also includes two Review articles: "Time Analysis of Poly(Lactic-Co-Glycolic) Acid Nanoparticle Uptake by Major Organs Following Acute Intravenous and Oral Administration in Mice and Rats (http://online.liebertpub.com/doi/full/10.1089/ind.2012.0032)" by Lacey Simon and Cristina Sabliov, Louisiana State University (Baton Rouge, LA); and "Biomarker-Based Nanotechnology for the Improvement of Reproductive Performance in Beef and Dairy Cattle (http://online.liebertpub.com/doi/full/10.1089/ind.2012.0035)" by Peter Sutovsky and Chelsey Kennedy, University of Missouri-Columbia, MO.

Additional original research articles include "Pueraria lobata (Kudzu) Photosystem I Improves the Photoelectrochemical Performance of Silicon (http://online.liebertpub.com/doi/full/10.1089/ind.2012.0036)" by Darlene Gunther, Gabriel LeBlanc, David Cliffel, and G. Kane Jennings, Vanderbilt University (Nashville, TN); and "An Aptasensor Based on Polymer-Gold Nanoparticle Composite Microspheres for the Detection of Malathion Using Surface-Enhanced Raman Spectroscopy (http://online.liebertpub.com/doi/full/10.1089/ind.2012.0029)" by Francisco Barahona, Cameron Bardliving, Adrienne Phifer, John Bruno, and Carl Batt, Cornell University (Ithaca, NY) and Operational Technologies Corp. (San Antonio, TX).

"Nanoscale science continues to play a major role in catalyzing biotechnology innovation, yielding a broad spectrum of devices and products that are addressing many pressing social needs," says Larry Walker, PhD, Co-Editor-in-Chief and Professor, Biological & Environmental Engineering, Cornell University, Ithaca, NY.

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About the Journal

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Low-cost nano-biosensor to detect foodborne pathogen that causes listeriosis

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DNA tool kit goes live online

BIOFABs directors Drew Endy (left) and Adam Arkin hope that their facility will help to industrialize synthetic biology.

Margot Hartford

The latest shopping website is open for business, offering unusual wares: DNA tools to help biologists to engineer life.

The DNA sequences which allow precise control of gene activity in the bacterium Escherichia coli are the first output of BIOFAB, based in Emeryville, California, which calls itself the worlds first biological design-build facility. Launched in 2009 with a US$1.4-million grant from the US National Science Foundation, BIOFAB aims to advance synthetic biology by creating standard biological parts in the form of DNA sequences that control gene expression. These standard sequences should allow biologists to engineer cells that can make medicines and perform other useful tasks simply by plugging in various sets of genes.

The sequences are meant to overcome a key barrier to synthetic biology: genes inserted into an organism do not behave predictably, even in such a well-understood workhorse as E. coli. You would think after a generation of genetic engineering, expressing genes with precision in an organism as well utilized as E. coliwould be pretty straightforward. It turns out its not, says BIOFAB co-director Drew Endy, a synthetic biologist at Stanford University in California.

For a cell to express a gene that is, transcribe it into an RNA molecule and then translate that RNA into a protein other sequences recognized by the cells machinery must precede it. A promoter sequence is needed to make an RNA transcript, and a ribosome binding site (RBS) is crucial for protein translation.

Over the past three decades, scientists have amassed collections of these sequences and used them to express genes in which they are interested. Some sequences tend to be strong and others weak, resulting in varying levels of RNA and protein being produced.

But a team led by Endy and BIOFAB co-director Adam Arkin, of Lawrence Berkeley National Laboratory in Berkeley, California, has found that the activities of those sequences are far from predictable. In two papers published online this week in Nature Methods1, 2, the team reports inserting many different combinations of promoters and RBS sequences in front of genes encoding fluorescent proteins, and then measuring the level of protein that was made. It was a bloody mess, says Arkin, with each promoterRBS combination having varying effects depending on the gene.

He and Endy also cite an earlier finding that a scientist hoping to express a protein at a particular level has just a 50% chance of producing the required amount within a factor of two. Such hit-or-miss expression poses a major challenge to synthetic biologists who would like to create genetic circuits involving dozens of genes.

As a solution, the BIOFAB team designed promoter and RBS sequences for E. colithat do not interfere with downstream DNA, so that their effects are independent of the specific gene they are paired with. The sequences should provide scientists with a much tighter grip on gene expression, offering around a 93% chance of hitting a desired level of expression within a factor of two2. Researchers can obtain the sequences for free online (see http://www.biofab.org/data), and Arkin says that some of his colleagues are already finding them useful.

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DNA tool kit goes live online

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