Category Archives: Genetic Engineering

Public release date: 26-Jul-2012 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, July 26, 2012 Administering high-doses of interleukin-2 (IL-2) has been the preferred treatment for patients with stage IV metastatic melanoma. An article published in the current issue of Cancer Biotherapy and Radiopharmaceuticals, a peer-reviewed journal from Mary Ann Liebert, Inc. (http://www.liebertpub.com), explores whether or not this regimen is still the most effective. The article is available free online at the Cancer Biotherapy and Radiopharmaceuticals website (http://www.liebertpub.com/cbr).

In the article "Should High-Dose Interleukin-2 Still Be the Preferred Treatment for Patients with Metastatic Melanoma?" (http://online.liebertpub.com/doi/full/10.1089/cbr.2012.1220) Robert Dillman and colleagues at the Hoag Institute for Research and Education and Hoag Family Cancer Institute, Newport Beach, CA concluded that until long-term survival data for some of the newer drugs are available, patients with stage IV metastatic melanoma who are well enough to be given intensive IL-2 therapy should receive it initially, either alone or in combination with one of the newer therapeutic agents.

"This is an important article that puts into perspective the reasons why IL-2 should continue to be the initial therapy in patients with metastatic melanoma," says Editor Donald J. Buchsbaum, PhD, Division of Radiation Biology, Department of Radiation Oncology, University of Alabama at Birmingham.

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About the Journal

Cancer Biotherapy and Radiopharmaceuticals (http://www.liebertpub.com/cbr), published 10 times a year in print and online, is under the editorial leadership of Editors Donald J. Buchsbaum, PhD and Robert K. Oldham, MD, Lower Keys Cancer Center, Key West, FL. Cancer Biotherapy and Radiopharmaceuticals is the only journal with a specific focus on cancer biotherapy, including monoclonal antibodies, cytokine therapy, cancer gene therapy, cell-based therapies, and other forms of immunotherapy. The Journal includes extensive reporting on advancements in radioimmunotherapy and the use of radiopharmaceuticals and radiolabeled peptides for the development of new cancer treatments. Topics include antibody drug conjugates, fusion toxins and immunotoxins, nanoparticle therapy, vascular therapy, and inhibitors of proliferation signaling pathways.

About the Publisher

Mary Ann Liebert, Inc., publishers (http://www.liebertpub.com) 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 Journal of Interferon & Cytokine Research, Human Gene Therapy and Human Gene Therapy Methods, 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 70 journals, books, and newsmagazines is available at Mary Ann Liebert, Inc. (http://www.liebertpub.com)

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Should high-dose interleukin-2 continue to be the treatment of choice for metastatic melanoma?

Public release date: 25-Jul-2012 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, July 25, 2012A novel approach to gene therapy that instructs a person's own cells to produce more of a natural disease-fighting protein could offer a solution to treating many genetic disorders. The method was used to achieve a 2- to 3-fold increase in production of a protein deficient in patients with Friedreich's ataxia, as described in an article published Instant Online in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc. (http://www.liebertpub.com) The article is available free online at the Human Gene Therapy website (http://www.liebertpub.com/hum).

The innovative gene therapy method described by Jacques Tremblay, Pierre Chapdelaine, Zo Coulombe, and Joel Rousseau, Laval University, Quebec, and University of Quebec, Canada, takes advantage of the ability of a family of proteins called Tal effector (TALE) proteins to target specific DNA sequences. As a model of how this method could be used to treat genetic disease, the authors engineered TALE proteins to target the gene that codes for the frataxin protein, which is deficient in Friedreich's ataxia. The ability to induce cells to produce more frataxin could reduce symptoms of the disease and provide an effective, long-term therapeutic strategy, conclude the authors in the article "TALE Proteins Induce the Expression of the Frataxin Gene. (http://online.liebertpub.com/doi/full/10.1089/hum.2012.034)

"This is a very clever approach to treat a recessive disease caused by decreased quantity of an otherwise normal protein," says James M. Wilson, MD, PhD, Editor-in-Chief, 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 (http://www.liebertpub.com/hum), 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 that 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. Tables of content and a free sample issue may be viewed online at the Human Gene Therapy website (http://www.liebertpub.com/hum).

About the Publisher

Mary Ann Liebert, Inc., publishers (http://www.liebertpub.com) 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 Tissue Engineering, Stem Cells and Development, and Cellular Reprogramming. 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 at Mary Ann Liebert, Inc. (http://www.liebertpub.com)

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New gene therapy strategy boosts levels of deficient protein in Friedreich's ataxia

Public release date: 24-Jul-2012 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, July 24, 2012A naturally occurring line of immunodeficient pigs can support the growth of human tumors injected under their skin, offering a promising new large animal model for studying human cancers and testing new drugs and treatment strategies. The ability of human melanoma cells and pancreatic carcinoma cells to grow in these pig models is described in an article in BioResearch Open Access, a new bimonthly peer-reviewed open access journal from Mary Ann Liebert, Inc. (http://www.liebertpub.com). The article is available free online at the BioResearch Open Access website (http://www.liebertpub.com/biores).

Mathew Basel and colleagues, Kansas State University (Manhattan, KS) and Iowa State University (Ames), highlight the advantages that pig disease models offer, as they are anatomically and physiologically more closely related to humans than traditional rodent animal models. As a result, findings from studies in large animal models such as pigs are more likely to translate into similar outcomes in humans. The authors present their findings in the article "Human Xenografts Are Not Rejected in a Naturally Occurring Immunodeficient Porcine Line: A Human Tumor Model in Pigs" (http://online.liebertpub.com/doi/full/10.1089/biores.2012.9902).

"This novel animal model has the potential to become a highly useful model in cancer research studies, in addition to providing significant opportunities for drug discovery and other translational applications," says Editor-in-Chief Jane Taylor, PhD, MRC Centre for Regenerative Medicine, University of Edinburgh, Scotland.

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About the Journal

BioResearch Open Access (http://www.liebertpub.com/biores) is a bimonthly peer-reviewed open access journal that provides a new rapid-publication forum for a broad range of scientific topics including molecular and cellular biology, tissue engineering and biomaterials, bioengineering, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience. All articles are published within 4 weeks of acceptance and are fully open access and posted on PubMedCentral. All journal content is available online at the BioResearch Open Access website (http://www.liebertpub.com/biores).

About the Publisher

Mary Ann Liebert, Inc., publishers (http://www.liebertpub.com) 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 Tissue Engineering, Stem Cells and Development, Human Gene Therapy and HGT Methods, and AIDS Research and Human Retroviruses. 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. website (http://www.liebertpub.com).

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Novel pig model may be useful for human cancer studies

Public release date: 17-Jul-2012 [ | E-mail | Share ]

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, July 16, 2012A small, naturally occurring nucleic acid sequence, called a microRNA, known to regulate a number of different cancers, appears to alter the activity of the androgen receptor, which plays a critical role in prostate cancer. Directly targeting microRNA-125b to block androgen receptor activity represents a novel approach for treating castrate-resistant prostate cancer. This promising new strategy for improving the effectiveness of anti-androgenic and other hormonal therapies is described in an article in BioResearch Open Access, a new bimonthly peer-reviewed open access journal from Mary Ann Liebert, Inc.. The article is available free online at the BioResearch Open Access website.

Prostate cancer is the most common non-skin cancer affecting men and the second most common cause of cancer death among men. In the article "miR-125b Regulation of Androgen Receptor Signaling Via Modulation of the Receptor Complex Co-Repressor NCOR2," Xiaoping Yang, Lynne Bernis, Lih-Jen Su, Dexiang Gao, and Thomas Flaig, University of Colorado Denver (Aurora) and University of Minnesota (Duluth), looked for targets of microRNA-125b that might shed light on its role in regulating prostate cancer and found that it directly inhibits NCOR2, which acts to repress the androgen receptor. The authors point out that "the androgen receptor is a critical therapeutic target in prostate cancer" and that alterations in the receptor are essential for the development of castrate-resistant prostate cancer, in which the disease no longer responds to hormonal therapies.

"This research provides new insight into the mechanism of miR-125b regulation of castrate-resistance prostate cancer through the identification of a novel target for miR-125b," says Editor-in-Chief Jane Taylor, PhD, MRC Centre for Regenerative Medicine, University of Edinburgh, Scotland. "The clinical implications of this study are that targeted regulation of this miRNA may lead to more effective anticancer therapies."

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About the Journal

BioResearch Open Access is a bimonthly peer-reviewed open access journal that provides a new rapid-publication forum for a broad range of scientific topics including molecular and cellular biology, tissue engineering and biomaterials, bioengineering, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience. All articles are published within 4 weeks of acceptance and are fully open access and posted on PubMedCentral. All journal content is available online at the BioResearch Open Access website.

About the Publisher

Mary Ann Liebert, Inc., 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 Tissue Engineering, Stem Cells and Development, Human Gene Therapy and HGT Methods, and AIDS Research and Human Retroviruses. 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. website.

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New therapeutic target for prostate cancer identified

Public release date: 10-Jul-2012 [ | E-mail | Share ]

Contact: John Sterling jsterling@genengnews.com 914-740-2196 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, July 9, 2012More than half (52%) of the companies comprising the tissue engineering (TE) and stem cell industries are revenue-generating, compared to about 21% four years ago, reports Genetic Engineering & Biotechnology News (GEN). Of those companies, 31% have commercial products and 21% are service-based; another 30% have products in clinical trials, according to the current issue of GEN.

The GEN article is based on interviews with leading tissue engineering researchers and on the findings of a landmark paper ("Progress in the Tissue Engineering and Stem Cell Industry, Are we there yet"), which appears in Tissue Engineering: Part B, Volume 18, Number 3, 2012, published by Mary Ann Liebert, Inc.

"Like many other biotechnologies, tissue engineering has experienced an up and down history," said John Sterling, Editor in Chief of GEN. "But with numerous technical advances moving the field forward combined now with rising revenues, this segment of bioresearch is really taking off."

The industry itself is beginning to attain profitability, with sales revenues reaching $3.5 billion and industry spending approaching $3.6 billion. The 2012 analysis by a group led by Robert Langer, Sc.D., one of the authors of the paper in the Liebert journal, reported a nearly threefold increase in commercial sales for TE and stem cell products and services compared to the previous four-year period. Furthermore, the number of companies selling products or offering services increased more than twofold to 106.

The GEN article also notes that Tissue Engineering has formed an industry council for the purpose of helping to guide the evolution of the industry and to create strategic initiatives aimed at overcoming some of the R&D, manufacturing, and regulatory challenges facing the industry.

Among the companies interviewed for the GEN article are Organogenesis, Cytograft Tissue Engineering, Scintellix, and Humacyte.

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For a copy of the July issue of GEN, please call (914) 740-2146, or email: pbartell@genengnews.com

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GEN reports on growth of tissue engineering revenues

Public release date: 16-May-2012 [ | E-mail | Share ]

Contact: Cathia Falvey cfalvey@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, May 16, 2012The inaugural issue of BioResearch Open Access, a new bimonthly peer-reviewed open access journal, was released today by Mary Ann Liebert, Inc., publishers. The Journal provides a new rapid-publication forum for a broad range of scientific topics including but not limited to molecular and cellular biology, tissue engineering and biomaterials, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience. The first issue is available on the BioResearch Open Access website at http://www.liebertpub.com/biores.

The premier issue includes research papers and a brief report from the U.S., U.K., Germany, and Korea on diverse topics such as tissue engineering, stem cells, HIV, and genetics. Forthcoming papers for the second issue include genetics, xenotransplantation, nuclear transfer, and cardiac research.

The Journal is under the leadership of Editor-in-Chief Jane Taylor, PhD, Senior Research Fellow, MRC Centre for Regenerative Medicine, University of Edinburgh, and seasoned journal editors as Section Editors, including James M. Wilson, MD, PhD, University of Pennsylvania; Antonios G. Mikos, PhD, Rice University; Professor Sir Ian Wilmut, OBE FRS FRSE, University of Edinburgh; Peter C. Johnson, MD, Scintellix, LLC, Raleigh, NC; Aubrey D.N.J. de Grey, PhD, SENS Foundation, Cambridge, UK; Alan J. Russell, PhD, Carnegie Mellon University; Thomas Hope, PhD, Northwestern University; Ganes C. Sen, PhD, Cleveland Clinic Foundation; Bruce A. Sullenger, PhD, Duke University Medical Center; Graham C. Parker, PhD, Wayne State University School of Medicine; Carol Shoshkes Reiss, PhD, New York University; Stephen C. Ekker, PhD, Mayo Clinic, Rochester, MN; John B. West, MD, PhD, University of California, San Diego; David L. Woodland, PhD, Chief Scientific Officer, Keystone Symposia on Molecular and Cellular Biology; Stephen Higgs, PhD, Kansas State University; Eugene Kolker, PhD, Seattle Children's Hospital; and Domenico Grasso, PhD, PE, DEE, University of Vermont.

The Journal welcomes basic science and translational research in the form of original research articles, comprehensive review articles, mini-reviews, rapid communications, brief reports, technical reports, hypothesis articles, perspectives, and letters to the editor. All articles in BioResearch Open Access will be published online within 4 weeks of acceptance. Articles will be fully open access and posted on PubMedCentral. All articles submitted through July 15, 2012 will be made open access without article processing charges. BioResearch Open Access is fully NIH-, HHMI-, and Wellcome Trust compliant.

"BioResearch Open Access is a fully refereed multidisciplinary journal and provides all the checks and balances that rigorous peer review ensures," says Mary Ann Liebert, president of Mary Ann Liebert, Inc., publishers. "An outstanding editorial team comprised of experienced journal editors guarantees the integrity of the Journal."

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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 Tissue Engineering, Human Gene Therapy, Nucleic Acid Therapeutics, Stem Cells and Development, Viral Immunology, DNA and Cell Biology, and Antioxidants & Redox Signaling. 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. website at http://www.liebertpub.com.

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Premier issue of BioResearch Open Access launched by Mary Ann Liebert Inc. publishers

Hematoxylin and eosin (H&E) staining of sections of wild-type (top row) and H1 triple-knockout (bottom row) embryoid bodies. After 14 days in rotary suspension culture, the wild-type embryoid bodies showed more differentiated morphologies with cysts forming (black arrows) and the knockout embryoid bodies failed to form cavities (far right). (Credit: Yuhong Fan)

(Phys.org) -- New research findings show that embryonic stem cells unable to fully compact the DNA inside them cannot complete their primary task: differentiation into specific cell types that give rise to the various types of tissues and structures in the body.

Researchers from the Georgia Institute of Technology and Emory University found that chromatin compaction is required for proper embryonic stem cell differentiation to occur. Chromatin, which is composed of histone proteins and DNA, packages DNA into a smaller volume so that it fits inside a cell.

A study published on May 10, 2012 in the journal PLoS Genetics found that embryonic stem cells lacking several histone H1 subtypes and exhibiting reduced chromatin compaction suffered from impaired differentiation under multiple scenarios and demonstrated inefficiency in silencing genes that must be suppressed to induce differentiation.

While researchers have observed that embryonic stem cells exhibit a relaxed, open chromatin structure and differentiated cells exhibit a compact chromatin structure, our study is the first to show that this compaction is not a mere consequence of the differentiation process but is instead a necessity for differentiation to proceed normally, said Yuhong Fan, an assistant professor in the Georgia Tech School of Biology.

Fan and Todd McDevitt, an associate professor in the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory University, led the study with assistance from Georgia Tech graduate students Yunzhe Zhang and Kaixiang Cao, research technician Marissa Cooke, and postdoctoral fellow Shiraj Panjwani.

The work was supported by the National Institutes of Healths National Institute of General Medical Sciences (NIGMS), the National Science Foundation, a Georgia Cancer Coalition Distinguished Scholar Award, and a Johnson & Johnson/Georgia Tech Healthcare Innovation Award.

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Phase contrast images showing that H1 triple-knockout (bottom) embryonic stem cells were unable to adequately form neurites and neural networks compared to wild-type embryonic stem cells (top). (Click image for high-resolution version. Credit: Yuhong Fan)

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Genetic packing: Successful stem cell differentiation requires DNA compaction, study finds

With some clever genetic engineering but without ever touching a cell or an animal, scientist can remotely control cells using ultrasound, light,and, now, also radio waves. The electromagnetic waves can be used to selectivelyheat up parts of cells and activate a gene to make insulin in mice, according to a recent study published in Science.

But why care about radio waves if we have light and ultrasound?Radio waves have a couple distinct advantages over existing techniques.

In the current study, the radio waves didnt heat up a whole patch of tissue or even a whole cellit only affected specific pores in the cell, calledTRPV1,that open in response to heat. To get this specificity, the scientists made special iron oxide nanoparticles attached to an antibody that only sticks to TRPV1. When they turned on the radio waves, the iron oxide particles warmed up and opened the TRPV1 channel, minimally affecting the rest of the cell or surrounding cells.Ultrasound, on the other hand, heats up a whole patch of tissue to 42 Celsius, which could have damaging or confounding effects on the cells.

Radio waves, unlike light, can also penetrate deep into tissue. To show how the radio could safely work inside an animal, scientists injected mice with special cells that had been genetically engineered to include both the TRPV1 pore and a gene switch that would release insulin when exposed to calcium. Then they got those cells in the mice to start making insulin with a little Rube Goldberg-esque cellular machine: heat from the radio waves opened the TRPV1 channels, calcium rushed into the cells through the open TRPV1, the flood of calcium turned on the insulin gene switch, and, finally, the cells began making insulin. (The whole chain of events makes you appreciate the complexity of biology, right?)

In one last step, the scientists did away with the synthetic iron oxide nanoparticles altogther. They got cells to produce their own iron nanoparticles, a iron storage protein calledferritin. When they tested ferritin in cells, it was 2/3 as effective at inducing insulin production as the synthetic nanoparticles.

There could be medical applications for activating genes in stem cell therapy in the future, but for now, this is just pretty cool: scientists can turn on some radio waves and hack right into the cellular machinery of a mouse.

[via Nature News]

Mouse image via Shutterstock / lculig

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Now *This* Is a Cell Phone: Using Radio Waves to Control Specific Genes in Mice | 80beats

Devangshu Datta: Towards an HIV cure Advances in genetic engineering techniques may finally help us win the battle against this global scourge Devangshu Datta / New Delhi May 04, 2012, 00:53 IST

Since AIDS, or acquired immune deficiency syndrome, was identified in 1981, there has been only one medically-certified cure. That occurred under unusual circumstances and it gave researchers an important clue about new ways to attack the disease. Recent advances in genetic engineering techniques have aided in this process. Some studies offer new hope of a cure for the 35 million estimated to be infected worldwide.

No disease inspires as much superstitious dread. So far, AIDS is estimated to have killed over 30 million people and it infects millions every year. It is especially prevalent in Sub-Saharan Africa.

HIV is transmitted through the exchange of body fluids. Common causes of infection (not necessarily in order) include unprotected sex, blood transfusions, sharing needles and so on. The associations with promiscuity and drug addiction make it hard to implement policies to stop HIV-spread. What works best is a combination of sex education and drug awareness programmes, coupled with easy availability of condoms and disposable needles. But in conservative societies like India, people object to sex education. Some religions also discourage the use of condoms.

Someone infected with HIV (HIV-positive) may survive years, without symptoms. The virus attacks a class of white blood cells called CD4 T-cells. It inserts itself into the cell and replicates. T-cells are part of the natural immune system. Once AIDS develops owing to HIV taking over T-cells, the immune system shuts down. Most AIDS patients die of cancer, pneumonia, or some other infection.

The new approaches involve inserting immune genes into HIV-positive patients, through genetic engineering of stem cells. Every researcher is cautious about claims of cures. The characteristic long symptom-less periods and HIVs ability to hide can be cruelly deceptive. HIV-positive people are also vulnerable to quacks. Many charlatans, including a cross-dresser who teaches yoga on Indian television, have claimed at various times to have found AIDS cures.

Some people have natural genetic immunity for various reasons. Advances in understanding of genomes have helped identify some of the causes of immunity. Researchers have known for a while that a mutated gene called CCR5 Delta 32 offers natural immunity to HIV.

The mutation is rare and found only in a few northern Europeans. The normal CCR5 gene, which most people possess, is the receptor HIV uses to enter T-cells. HIV cannot use the Delta-32 mutated gene and, hence, cannot replicate in a host who has two copies of the CCR5 Delta 32 gene (one inherited from each parent). Even one copy of Delta 32 seems to offer some protection. Only about one per cent of northern Europeans possess both copies.

In 2007, Timothy Ray Brown, an American resident in Berlin, was HIV-positive and also under treatment for leukaemia. Leukaemia causes an abnormal increase in white blood cells and a drop in red cells. Blood cells are produced by bone marrow. One drastic treatment is a bone marrow stem cell transplant from a healthy person. This helps regenerate healthy blood with a good haemoglobin ratio, and a new immune system. Its dangerous since the patients entire immune system must be destroyed prior to the transplant.

Browns doctors at the Charite University Medicine Berlin, Kristina Allers and Gero Hutter, found a compatible donor who belonged to that rare one per cent with the Delta-32 mutation. Five years later, after the transplant procedures, the Berlin Patient, as Brown is called in medical journals, is still HIV-free and doctors concur that this is a functional cure.

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Devangshu Datta: Towards an HIV cure

St. Louis /PRNewswire/ -- Sigma-Aldrich Corporation (Nasdaq: SIAL) today announced that Sigma Advanced Genetic Engineering (SAGE) Labs, an initiative of Sigma Life Science, extended CompoZr Zinc Finger Nuclease (ZFN) technology to achieve the first tissue-specific conditional knockout of an endogenous gene in rats. For two decades this approach for generating sophisticated disease models could be performed only in mice. Rats, however, are preferred by drug discovery and basic researchers because the animal's physiology, neurobiology and other features are more predictive of human conditions. Rats engineered to contain tissue-specific conditional gene knockouts are available exclusively through the SAGEspeed Custom Model Development Service. Details are available at http://www.sageresearchmodels.com/conditional-KO.

Conventional gene knockout eliminates a gene throughout an entire animal. In contrast, conditional gene knockout can eliminate a gene solely in the relevant tissue or organ, leading to a more accurate understanding of the gene's function. Conditional gene knockout can also knock out genes at certain points in development, enabling studies of genes whose absence in embryos is lethal, but whose loss of function in adulthood is critical to investigate for many human diseases.

"Almost 89% of drug candidates fail to achieve approval," said Edward Weinstein, Director of SAGE Labs. "Basic and drug discovery researchers need access to more predictive animal models whose physiology, biology, and genetics more closely reflect specific human conditions. SAGE Labs is applying ZFN technology to achieve previously impossible genetic manipulations, such as tissue-specific gene deletion in rats."

Using the conditional knockout methodology, scientists at SAGE Labs have generated a pair of rat lines in which two important neuronal genes, Crhr1 and Grin1, were removed in specific neuronal populations. Crhr1 and Grin1 have been implicated as playing a role in depression and schizophrenia, respectively. The rat lines were developed through the SAGEspeed model creation process, which uses Sigma's CompoZr ZFN technology to create sophisticated genetic modifications in rats, mice, rabbits, and other organisms. CompoZr ZFN technology is the first to enable highly efficient, targeted editing of the genome of any species.

For more information and to request pricing, visit http://www.sageresearchmodels.com.

Cautionary Statement: The foregoing release contains forward-looking statements that can be identified by terminology such as "enable," "enabling," "leading to," "achieve," "predictive" or similar expressions, or by expressed or implied discussions regarding potential future revenues from products derived there from. You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of management regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that iPS cells, iPS-cell derived primary cell lines, novel assays, or related custom services will assist the Company to achieve any particular levels of revenue in the future. In particular, management's expectations regarding products associated iPS cells, iPS-cell derived primary cell lines, novel assays, or related custom services could be affected by, among other things, unexpected regulatory actions or delays or government regulation generally; the Company's ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry and general public pricing pressures; the impact that the foregoing factors could have on the values attributed to the Company's assets and liabilities as recorded in its consolidated balance sheet, and other risks and factors referred to in Sigma-Aldrich's current Form 10-K on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Sigma-Aldrich is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Sigma Life Science: Sigma Life Science is a Sigma-Aldrich business that represents the Company's leadership in innovative biological products and services for the global life science market and offers an array of biologically-rich products and reagents that researchers use in scientific investigation. Product areas include biomolecules, genomics and functional genomics, cells and cell-based assays, transgenics, protein assays, stem cell research, epigenetics and custom services/oligonucleotides. Sigma Life Science also provides an extensive range critical bioessentials like biochemicals, antibiotics, buffers, carbohydrates, enzymes, forensic tools, hematology and histology, nucleotides, amino acids and their derivatives, and cell culture media.

About Sigma-Aldrich: Sigma-Aldrich is a leading Life Science and High Technology company whose biochemical, organic chemical products, kits and services are used in scientific research, including genomic and proteomic research, biotechnology, pharmaceutical development, the diagnosis of disease and as key components in pharmaceutical, diagnostics and high technology manufacturing. Sigma-Aldrich customers include more than 1.3 million scientists and technologists in life science companies, university and government institutions, hospitals and industry. The Company operates in 40 countries and has nearly 9,000 employees whose objective is to provide excellent service worldwide. Sigma-Aldrich is committed to accelerating customer success through innovation and leadership in Life Science and High Technology. For more information about Sigma-Aldrich, please visit its website at http://www.sigma-aldrich.com.

Sigma-Aldrich and Sigma are trademarks of Sigma-Aldrich Co, LLC registered in the US and other countries. SAGE and CompoZr are registered trademarks of Sigma-Aldrich Co. LLC. SAGEspeed is a trademark of Sigma-Aldrich Co. LLC.

SOURCE: Sigma-Aldrich Corporation

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SAGE® Labs Creates The First Tissue-Specific Gene Deletion In Rats