Category Archives: Genetic Therapy

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GROVES Scooting around on her hands and knees 16-month-old Daphne Landry moved from one colorful toy to another.

The babe then maneuvered to her crib, carefully pulling her little body to a standing position as mom Heather watched from nearby. The series of movements, which are part of developmental steps and growth, are cause for celebration in the Landry household and show how strength, determination and faith can help beat the odds.

At the age of six months, Daphne was diagnosed with tuberous sclerosis complex, a rare genetic disorder that causes tumors to form in many different organs. She requires frequent trips for treatment at Childrens Memorial Hermann Hospital in Houston.

A benefit to help the family with medical expenses will be held at noon on Saturday, June 16 at Action Restoration, 5215 N. Twin City Hwy., Port Arthur. There will be barbecue dinners for sale, raffle tickets, a car wash, flea market, bake sale and kids activity tent.

Sporting a blue Little Mermaid shirt with white shorts and her hair pulled into a ponytail on top of her head, Daphne moved back across the floor to her mothers waiting arms.

She saw a neuropsychologist this week and he was amazed with how advanced she is with all the tumors going on. Shes a trooper, Heather Landry said. At the end of the month we will talk to the neurosurgeon and epileptologist about possible brain surgery.

Daphne currently has tumors in her brain, eyes and heart. The tumors are causing the child to have seizures and she is currently on three different medications, which are costly.

When she was about 6 1/2 months old she started having some weird movements and we thought, well, shes a baby and shes learned something new, the mother said of seeing Daphne lie flat then curl her extremities together. We learned she was having infantile spasm seizures.

Heather and her husband Derrick Landry first brought the child to Texas Childrens Hospital in Houston and although the hospital was great, she said, they felt not enough was being done to fight for her childs life. More seizures passed, some of which were more severe than usual, and with lots of prayer the family found a new treatment center at Childrens Memorial Hermann Hospital in Houston with Dr. Gretchen Von Allmen.

But hospital visits, medication and surgery are taking a toll on the one-income family. Heather was forced to quit her full-time job in January in order to care for her daughter.

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Saving Daphne; benefit to help local child with rare genetic disorder

NEW YORK, June 15, 2012 /PRNewswire/ -- A recent study at Oxford University in England and published in Annals of Neurology, has identified a gene that causes vitamin D deficiency and may also be the cause of multiple sclerosis. The study was partly funded by the National Multiple Sclerosis Society, The Wellcome Trust and the support of science investor, Jeffrey Epstein and The Jeffrey Epstein VI Foundation.

Multiple sclerosis is a neurological disease caused by the decay of myelin, the fatty sheath that protects the axons around the brain and spinal cord. Myelin is an essential part of neural communication because it not only protects the nerve circuits but promotes efficient conductivity. Every year, approximately 400,000 people in the United States develop the disease and about 2.5 million people worldwide. Symptoms vary widely, ranging from mild tingling to blindness and paralysis.

The cause of myelin damage is still hotly debated: some believe it to be an autoimmune disease while others cite viruses or the environment as the culprit. There is growing evidence however of a correlation between multiple sclerosis and vitamin D deficiency. Epidemiological studies also show that populations closer to the equator and the sun, have far fewer case of multiple sclerosis than populations closer to the north or south poles. Researchers at Oxford University have now taken this premise a step further by showing that vitamin D deficiency and therefore multiple sclerosis could have a genetic cause.

The study examined the DNA of a group of people with multiple sclerosis who also have a large number of family members with the disease. All the DNA samples showed a distortion of the CYP27B1 gene which controls vitamin D levels in the body. And in a few rare cases where the DNA showed two copies of the distorted gene, the person was found to have a genetic form of rickets caused by vitamin D deficiency as well as multiple sclerosis.

Despite this pivotal link, not all people with vitamin D deficiency develop multiple sclerosis. More research is needed to fully understand why only some people develop multiple sclerosis from vitamin D deficiency and why others don't. However, a distortion of the CYP27B1 gene is increasingly apparent in MS cases and it's possible that the gene generates other, yet undetected, complications that lead to the diseasesuch as genetically caused rickets.

"Although vitamin D deficiency doesn't always cause MS, it unveiled a critical genetic source that could be causing other problems that lead to MS," Jeffrey Epstein asserted, whose foundation, advances science and medical research across the United States. "Even if we don't understand all of the implications of that gene's distortion, research can focus on gene therapy, and that will accelerate a cure."

The National Multiple Sclerosis Society which also helped fund the Oxford study provides more than 325 research grants worldwide and training fellowships on a broad range of topics from immunology, nerve tissue repair and myelin biology, clinical trials, rehabilitation, psychosocial issues and health care delivery.

http://www.jeffreyepsteinfoundation.com

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A Genetic Cause For Multiple Sclerosis Is Identified And Funded By Science Patron, Jeffrey Epstein

Newswise Targeted cancer cell therapies using man-made proteins dramatically shrink many tumors in the first few months of treatment, but new research from Johns Hopkins scientists finds why the cells all too often become resistant, the treatment stops working, and the disease returns.

In a study of 28 advanced colon cancer patients treated with the monoclonal antibody panitumumab, the Johns Hopkins Kimmel Cancer Center team reports that drug-resistance tumor cell mutations appear in the blood of patients five to seven months later, and that low levels of these mutations exist in nearly all tumors before the therapy begins, making the cancers predestined to recur.

These resistance mutations develop by chance as cancer cells divide so that tumors always contain thousands of resistance cells, says Luis Diaz, M.D., associate professor of oncology and director of the Swim Across America laboratory at Johns Hopkins, who says the findings likely apply to any targeted cancer therapy.

The best chance for a cure is when a tumor is very small, but when the cancer is advanced, our research quantifies the probability that we can achieve cures with single-agent targeted therapies, says Bert Vogelstein, M.D., professor and co-director of the Ludwig Center at Johns Hopkins and, Howard Hughes Medical Institute investigator. Long-term remissions of advanced cancers will be nearly impossible with single targeted agents, he adds.

The Johns Hopkins scientists analyzed blood samples taken from 28 patients with advanced colorectal cancers. These patients were enrolled in a clinical trial of panitumumab, one of a new and growing class of monoclonal antibodies, or synthetic proteins that homes in on cancer cells vital growth pathways. In the case of panitumumab, the agent targets a growth-factor receptor called EGFR. Patients most likely to respond to the drug also have normal copies of the KRAS gene in their tumors.

Twenty-four of the 28 patients in the study had normal KRAS gene copies in their tumors, and four had mutations in KRAS, serving as a control group. Blood samples were taken before beginning the therapy and at four-week intervals during the therapy, for a total of 169 combined blood draws.

Virtually all cancers shed DNA material into the blood, according to the researchers, and provide an easy route to collecting molecular evidence from lesions typically inaccessible for surgical biopsy. The amount of tumor DNA found in the blood is akin to tests used to determine HIV viral load, says Diaz.

In their analysis, reported online June 13 in the journal Nature, the scientists found that nine of the 24 patients with normal KRAS genes (38 percent) exhibited KRAS mutations detectable in the blood within five to seven months of beginning therapy. KRAS mutations were detected in three patients before imaging scans showed metastatic tumor growth. Then, working with Martin Nowak, Ph.D., and his team from Harvard University, the investigators used mathematical models to calculate when KRAS mutations likely originated. Nowak and colleagues determined that KRAS mutations were present prior to the initiation of treatment with panitumumab.

The probability that the mutations were absent at the beginning of treatment is exceedingly low, says Vogelstein, leading the team to conclude that the development of drug-resistance is a fait accompli. The time it takes for cancers to recur is determined simply by how long it takes cancer cells with mutant genes to multiply, he adds.

The research team says that combination therapies are the best chance for longer remissions. The good news is that there is a limited number of pathways that go awry in cancer, so it should be possible to develop a small number of agents that can be used in a large number of patients, says Vogelstein. However, I hope this research will help stimulate the testing of new drugs as combination therapies much earlier in the drug approval process than the current norm.

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Advanced Cancers Destined to Recur After Treatment with Single Drugs That "Target" Tumor Cells' Genetic Pathways

Stuart Rabin was 6 or 7 years old when his mother, Anne, first witnessed the positive effects that music therapy could have on her son. Stuart, who will soon turn 27, has a genetic disorder called Angelman Syndrome, which caused him to have significant developmental and cognitive disabilities. And 20 years ago, he was still having trouble sitting upright, Anne Rabin said.

Music therapy was introduced to Stuart back then as part of his early intervention care, and one of the first changes his parents noticed during their sons therapy sessions was his ability to sit on a bench next to the music therapist.

That might seem like a minor issue to some, but it is a primary skill that Stuart didnt have, said Anne Rabin of Pleasant Prairie.

At the time, Rabin also noticed how the music in her sons therapy sessions caught his attention, and seemed to calm him down. People with Angelman Syndrome tend to engage in a lot of repetitive patterns of behavior, which can limit their ability to have interest in other activities, she said.

Music therapy increases Stuarts opportunities to express himself, and he can even play a little bit, she said.

Always looking for ways to improve their sons quality of life, Stuarts parents have found that music therapy has increased his ability to focus on things and to engage in the world around him. His work with a music therapist has also made him better able to tolerate loud sounds something that in the past would overstimulate him to the point of hysterical reactions, Rabin said.

It has given him great opportunities, and he enjoys it, she said.

Neurologicmusic therapy

Angelman Syndrome is one of many brain disorders, illnesses and conditions that music therapy is used to treat. Recent research has shown that people of all ages, with everything from autism spectrum disorders to Parkinsons disease to brain injuries, can benefit from music therapy. And while the concept of using music to heal the mind and body dates back to ancient times, the development of new brain imaging and recording technologies in the last 20 years has led to a new scientific model of therapeutic techniques known as neurologic music therapy.

Researchers such as Michael Thaut, director of the Center for Biomedical Research in Music at Colorado State University, encourage the use of neurologic music therapy in rehabilitative care for patients ranging from those recovering from a stroke to those with language or cognitive difficulties. Such research is something that local certified music therapist Lisa Friedrich says her work is based on and which has brought results for clients in her private practice, Harmonious Beginnings.

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Music therapy makes a difference

Critics caution against opening a 'Pandora's box' which could lead to a trend towards 'designer babies'

By Fiona Macrae

PUBLISHED: 19:56 EST, 11 June 2012 | UPDATED: 19:56 EST, 11 June 2012

The creation of babies with three genetic parents would be an amazing opportunity for families whose lives have been blighted by incurable diseases, say an eminent group of experts on ethics in science.

The influential Nuffield Council on Bioethics conceded that while those with religious views might view the advance as an abomination, there is no ethical reason to stop it, provided it is proved to be safe and effective.

The approval comes as pressure builds on the Government to amend the law to allow the genetic engineering of eggs and embryos, creating babies free of devastating genetic diseases.

Three parents? Ethicists have decided that mixing DNA from more than two parents is acceptable if it is used to cure hereditary diseases

The children would effectively have two mothers and one father. Those in favour say it would give couples who have endured the heartbreak of miscarriages and stillbirths, and children who have died while still young, the option of having a healthy family.

But critics say the science is too risky and the safety of the baby should take precedence over a womans desire to be a mother.

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Experts on ethics back creation of babies with three parents saying it is 'amazing opportunity' for families blighted ...