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Enlarge Image Gene fix. Red cells in this slice of mouse liver are making a human protein called A1At. Credit: K. YUSA ET AL., NATURE (ADVANCED ONLINE EDITION) ©2011 MACMILLAN PUBLISHERS LTD. Researchers have taken a step toward showing how stem cells might one day be used to help patients born with a deadly liver disease. The researchers corrected a DNA spelling error in patient skin cells that had been converted into so-called induced pluripotent stem (iPS) cells, then coaxed the cells to form liver cells that seemed to function normally in mice. The approach is still a long...

Two of the holy grails of medicine - stem cell technology and precision gene therapy - have been united for the first time in humans, say scientists.It means patients with a genetic disease could, one day, be treated with their own cells. A study in Nature corrected a mutation in stem cells made from a patient with a liver disease. Researchers said this was a "critical step" towards devising treatments, but safety tests were still needed. At the moment, stem cells created from a patient with a genetic illness cannot be used to cure the disease as those cells would...

A gene silencing approach can save monkeys from high doses of the most lethal strain of Ebola virus in what researchers call the most viable route yet to treating the deadly and frightening infection. They used small interfering RNAs or siRNAs, a new technology being developed by a number of companies, to hold the virus at bay for a week until the immune system could take over. Tests in four rhesus monkeys showed that seven daily injections cured 100 per cent of them. U.S. government researchers and a small Canadian biotech company, Tekmira Pharmaceuticals, worked together to develop the new...

Functionalised gold nanoparticles make a more efficient and versatile delivery system for use in gene therapy and cancer treatment, claim Korean scientists. Gold nanoparticles are non-toxic and easily made and so provide an effective way to deliver therapeutic antisense DNA to disease causing genes. Antisense DNA that is covalently attached to the nanoparticles will bind to messenger RNA of a disease causing gene, preventing translation and therefore inactivating it. But previous systems could deliver only antisense DNA that was covalently cross-linked to gold nanoparticles, which needed to be individually synthesized for each gene of interest. Now, Kangseok Lee and Min Su Han at...

Veterinary ophthalmology researchers from the University of Pennsylvania have used gene therapy to restore retinal cone function and day vision in two canine models of congenital achromatopsia, also called rod monochromacy or total color blindness. Achromatopsia is a rare autosomal recessive disorder with an estimated prevalence in human beings of about 1 in 30,000 to 50,000. It primarily affects the function of the cone photoreceptors in the retina and serves as a representative model for other more common inherited retinal disorders affecting cones. Cone function is essential for color vision, central visual acuity and most daily visual activities, which underlines...

Gene therapy for a severe inherited blindness, which produced dramatic improvements last year in 12 children and young adults who received the treatment in a clinical trial, has cleared another hurdle. The same research team that conducted the human trial now reports that a study in animals has shown that a second injection of genes into the opposite, previously untreated eye is safe and effective, with no signs of interference from unwanted immune reactions following the earlier injection. These new findings suggest that patients who benefit from gene therapy in one eye may experience similar benefits from treatment in the...

(BRONX, NY) — A team led by researchers at Albert Einstein College of Medicine of Yeshiva University has found a clear link between living to 100 and inheriting a hyperactive version of an enzyme that rebuilds telomeres — the tip ends of chromosomes. The findings appear in the latest issue of the Proceedings of the National Academy of Sciences. Telomeres play crucial roles in aging, cancer and other biological processes. Their importance was recognized last month, when three scientists were awarded the 2009 Nobel Prize in Physiology and Medicine for determining the structure of telomeres and discovering how they protect...

A treatment based on HIV finds first success in humans.Researchers have halted a fatal brain disease by delivering a therapeutic gene to the stem cells that mature into blood cells. The gene was transferred using a virus derived from HIV, a technique that researchers have pursued for more than a decade but has not been successful in humans until now. Together with his colleagues, paediatric neurologist Patrick Aubourg at INSERM — France's main biomedical research agency — and at the Saint-Vincent de Paul Hospital in Paris, developed the system to treat X-linked adrenoleukodystrophy (ALD), a neurodegenerative disease that affects young...

Introducing three genes corrects motor defects in monkeys.A potential gene therapy for Parkinson's disease can correct motor deficits in monkeys without causing the jerky, involuntary movements that often accompany long-term treatments for the disease. The approach is undergoing preliminary testing in a handful of human patients, who have all shown promising signs of improvement.At present, the most common remedy for Parkinson's disease involves replacing dopamine — the neurotransmitter that is depleted in patients with the disease — by administering the dopamine precursor levodopa, or L-DOPA. Most patients initially regain near-normal motor control, but after several years on L-DOPA the majority...

Dalton, a squirrel monkey treated with gene therapy, enjoys his new colour sense.Neitz Laboratory Researchers have used gene therapy to restore colour vision in two adult monkeys that have been unable to distinguish between red and green hues since birth — raising the hope of curing colour blindness and other visual disorders in humans."This is a truly amazing study," says András Komáromy, a vision researcher and veterinary ophthalmologist at the University of Pennsylvania in Philadelphia, who was not involved in the research. "If we can target gene expression specifically to cones [in humans] then this has a tremendous implication."About 1...

Enlarge ImageOn track. Colonies of genetically corrected cells taken from Fanconi anemia patients show red and yellow, markers associated with pluripotency. Credit: Juan Carlos Izpisúa Belmonte Two papers published this week appear to bring closer the day when embryonic-like stem cells can be used to treat human diseases. One study describes what scientists say is the safest method yet to produce these cells. The other reports success in using the cells to begin correcting a rare genetic disorder known as Fanconi anemia. Induced pluripotent stem (iPS) cells were first reported in 2006 by Shinya Yamanaka, a researcher at Kyoto...

Enlarge ImageDesigner genes. Monkeys given a virus that holds the DNA for lab-made antibodies dodged SIV.Credit: Malcolm Linton Researchers are reporting that a new antiviral strategy powerfully protects monkeys from SIV, the simian cousin of HIV. The approach combines elements of vaccines and gene therapy, and experts say the development could eventually lead to a vaccinelike weapon against AIDS--a goal that has thus far proved elusive. Vaccines work by priming the "adaptive" immune system to recognize and attack a specific invader. But despite 2 decades of research, several potential AIDS vaccines have failed to teach the immune system to...

A radical technique for treating diabetes could recruit cells in the gut to make insulin SAN DIEGO — If your pancreas fails you, go with your gut. Inserting a gene into gut cells in mice enabled those cells to take over the pancreas’s job, producing insulin after meals, according to unpublished research announced June 18 in San Diego at the Biotechnology Industry Organization International Convention. The work may offer a novel way to treat diabetes. "This is the first time that we've engineered a tissue that is not the pancreas to manufacture insulin" in animals, says researcher Anthony Cheung, a...

Enlarge ImageSeeing the light.Steven Howarth, 18, a patient in a U.K. gene-therapy trial for blindness, says he is now more comfortable walking home at dusk.Credit: BBC In what eye researchers are hailing as a major advance, gene therapy has partially restored the sight of four young adults born with severe blindness. In two small studies, the patients' ability to sense light improved, and two can now read several lines of an eye chart. All are still legally blind, but the same treatment could potentially prevent this type of blindness in babies. The four patients have a disease called Leber...

Closure? The death of Jolee Mohr, shown here with her family, was unrelated to the gene-therapy treatment she received, the study's sponsor says.Credit: Mohr Family/AP Images Federal regulators have given a green light to a gene-therapy arthritis trial that was halted last summer after a patient died. New tests indicate that the therapy played no role in the death. The decision comes as a relief to gene-therapy researchers who had worried about a potential new setback for their field. The trial conducted by Targeted Genetics Corp. was shut down after the 24 July death of 36-year-old Jolee Mohr of Taylorville,...

Heart attacks are the leading cause of death in both men and women, with over 450,000 deaths in the United States each year. Improving the treatments available to patients who have survived a heart attack is therefore imperative. Researchers from the National Center for Regenerative Medicine (NCRM) have identified two innovative and distinctly different methods to help treat patients recovering from a heart attack.  The first study showed that cell-based gene therapy to regenerate damaged cardiac muscle tissue and improve mechanical cardiac function was feasible using skeletal muscle stem cells modified to express a stem cell honing signal. The second...

A new discovery in stem cell research may mean big things for cancer patients in the future. Gary Van Zant, Ph.D., and a research team at the University of Kentucky published their findings today in Nature Genetics, an international scientific journal. The researchers genetically mapped a stem cell gene and its protein product, Laxetin, and building on that effort, carried the investigation all the way through to the identification of the gene itself. This is the first time such a complete study on a stem cell gene has been carried out. This particular gene is important because it helps regulate...

Scientists have discovered a new gene that makes mice happy, a finding that suggests another avenue of drugs for improving depression in humans. The research represents the first time that depression has been eliminated genetically in any organism, said Guy Debonnel, a psychiatrist and professor at McGill University. Debonnel and his colleagues achieved this effect by creating and breeding mice lacking a gene also found in humans that affects the transmission of the mood-modulating chemical serotinin. Mice without the gene, called TREK-1, acted as if they had been treated with anti-depressants for at least three weeks, he said. By removing...

07/26/04 -- Researchers have found a delivery method for gene therapy that reaches all the voluntary muscles of a mouse - including heart, diaphragm and limbs ? and reverses the process of muscle-wasting found in muscular dystrophy. "We have a clear 'proof of principle' that it is possible to deliver new genes body-wide to all the striated muscles of an adult animal. Finding a delivery method for the whole body has been a major obstacle limiting the development of gene therapy for the muscular dystrophies. Our new work identifies for the first time a method where a new dystrophin gene...

Its history is marred by failures, false hopes, and even death, but for a number of the most horrendous human diseases, gene therapy still holds the promise of a cure. Now, for the first time, there is reason to believe that it is actually working. Part I: By the late 1960s, molecular biologists had erected an overarching explanation of how genes work--their substance, their structure, their replication, their expression, their regulation or control. Or at least they had done so in outline, for prokaryotes, the simplest single-celled organisms (which include bacteria), and for the viruses, called bacteriophages, that prey upon...

A top American scientist dubbed "the father of gene therapy" is facing two decades in prison after been convicted of molesting a colleague's daughter. William French Anderson, known to many in the competitive world of genetic research as "French", entered the annals of medicine in 1990 when he led a team that successfully implanted missing genes into the blood of a 3-year-old girl, enabling her recovery from a rare, hereditary disease. But yesterday the 69-year-old was found guilty of four counts of abuse towards the 10-year-old daughter of a research scientist who worked in his laboratory at the University of...

LOS ANGELES A jury convicted world-renowned geneticist William French Anderson on Wednesday of molesting the daughter of a colleague. Anderson, 69, is widely credited as the "father of gene therapy," a promising but controversial experimental medical treatment that involves injecting healthy genes into sick patients. He was the first person to successfully treat a patient this way in 1990, launching the field. The white-haired Anderson sat stoically, staring straight ahead, his head held high. He showed no reaction as the verdicts were read. His wife, a renowned surgeon, sat in the front row of the spectator section behind him with...

Researchers at Rush University Medical Center, Chicago, and Ceregene Inc., San Diego, have successfully used gene therapy to preserve motor function and stop the anatomic, cellular changes that occur in the brains of mice with Huntington’s disease (HD). This is the first study to demonstrate that, using this delivery method, symptom onset might be prevented in HD mice with this treatment. Results of the study were published in the Proceedings of the National Academy of Sciencesof the United States, June 13, 2006. “This could be an important step toward a disease modifying therapy,” says co-author Jeffrey H. Kordower, Ph.D., director...

Is the cloning of human babies' tissue an insult to god? Posted at: 22:01 A proposal to create babies that are both cloned and genetically altered to prevent serious hereditary disease has been outlined by the leader of the team that created Dolly the sheep, re-igniting the debate on the moral implications of cloning human beings. Ever since news that Dolly had been cloned from an adult cell made headlines around the world, Prof Ian Wilmut has repeatedly said he is "implacably opposed" to cloning a human being. But in his forthcoming book After Dolly, serialised in The Daily Telegraph,...

China, with lower regulatory hurdles, is racing to a lead in gene therapy. Once a week, Hashmukh Patel, a 62-year-old retired semiconductor engineer from Silicon Valley, travels with his wife, Bena, from their Beijing hotel to Beijing-Haidian Hospital. They ride the crowded elevator to the ninth floor, enter a pleasant, sun-filled ward with private rooms, and Patel gets an injection that he hopes will save his life. Suffering from late-stage cancer of the esophagus, he has come to Beijing for a Chinese gene-therapy drug called Gendicine that's supposed to kill tumor cells. Patel tried just about everything before coming to...

In a study to be published in the January 2006 issue of Nature Biotechnology, researchers led by a team of scientists at Memorial Sloan-Kettering Cancer Center have devised a novel strategy that uses stem cell-based gene therapy and RNA interference to genetically reverse sickle cell disease (SCD) in human cells. This research is the first to demonstrate a way to genetically correct this debilitating blood disease using RNA interference technology. To prevent the production of the abnormal hemoglobin that causes sickle cell disease, a viral vector was introduced in cell cultures of patients who have the disease. The vector carried...

Genes help immunity disease boy Mustaf now attends nursery Gene therapy has been successfully used to treat a four-year-old boy with no immune system, a team of British scientists has revealed.The boy, named only as Mustaf, had the rare life-threatening immune disorder known as ada-SCID. Doctors at Great Ormond Street Hospital used a harmless virus to deliver stem cells containing a corrective gene. It is the first successful treatment of ada-SCID in the UK. Five children have been treated in Milan, Italy. Children with ada-SCID have no working immune system. They are susceptible to even the most minor infection....

LOS ANGELES (AP) - A renowned University of Southern California gene therapy scientist accused of molesting two children over the past 20 years posted bond and was released from jail, authorities said Monday. William French Anderson, 68, was released shortly after 5 a.m. on bond, according to the county Sheriff's Department Web site. No other information was immediately released. Anderson is accused of molesting a girl he instructed in martial arts between 1997 and 2001. He has pleaded not guilty to a count of continuous sexual abuse of a child under age 14 and five counts of committing a lewd...

Controversial theorist Aubrey de Grey insists that we are within reach of an engineered cure for aging. Are you prepared to live forever? On this glorious spring day in Cambridge, England, the heraldic flags are flying from the stone towers, and I feel like I could be in the 17th century—or, as I pop into the Eagle Pub to meet University of Cambridge longevity theorist Aubrey de Grey, the 1950s. It was in this pub, after all, that James Watson and Francis Crick met regularly for lunch while they were divining the structure of DNA and where, in February 1953,...

<p>Gene injections in rats can double muscle strength and speed, researchers have found, raising concerns that the virtually undetectable technology could be used illegally to build super athletes. A University of Pennsylvania researcher seeking ways to treat illness said that studies in rats show that muscle mass, strength and endurance can be increased by injections of a gene-manipulated virus that goes to muscle tissue and causes a rapid growth of cells.</p>

Parkinson's Gene Therapy Study Begins By MALCOLM RITTER AP Science Writer NEW YORK (AP) -- Researchers slipped billions of copies of a gene into the brain of a Parkinson's disease patient Monday, marking the first attempt to test gene therapy in a person with that disease. The patient, 55-year-old Nathan Klein of Port Washington, N.Y., said he was "feeling fine" in a telephone interview a few hours after the surgery. Klein said the disease gave him a tremor in his right arm, and made him shuffle and have to catch himself sometimes from falling. Medicines offer some relief, he said,...

Does human history have a future? In his latest work, Aliens in America, political philosopher Peter Augustine Lawler argues that the advent of the new biotechnology—cloning, gene therapy, Prozac, Ritalin, and the like—means that we must consider anew the possibility that Americans are lving near the end of history, a time when full equality will be achieved through the elimination of al that is distinctively human about us: the ability to passionately love and hate, to strive nobly for truth and wisdom, to search for God. For the elimination of human distinctiveness is now being systematically pursued through the biochemical...

NEW YORK - Scientists have successfully inserted a foreign gene into the DNA of mice that was later incorporated into growing hair shafts. "This will allow us to search for genes that will give a cosmetic benefit as well as potential therapeutic benefits when inserted into the hair follicle," said the study's lead author Dr. Robert M. Hoffman of AntiCancer Inc., a San Diego, California-based biotechnology company. While the green fluorescent protein (GFP) gene, which comes from jellyfish, has no medical significance, the main goal was to determine if the researchers could make transgenic mouse hair. The mouse genome did...

WASHINGTON (AP) _ A single injection of genetically modified stem cells is all it took to cure two children of a complex form of an inherited immune system disorder often referred to as the ``bubble boy disease,'' researchers report. An experimental technique that altered genes in bone marrow stem cells restored the immune systems of the children, researchers from Italy and Israel said in a study appearing in the journal Science. The children were born with what experts said was the most complex form of severe combined immunodeficiency disorder, or SCID. ``Both children have been cured but ... both will...