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Stem cell gene therapy for sickle cell disease advances toward clinical trials

Posted: Published on July 3rd, 2013

July 1, 2013 Researchers at UCLA's Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research have successfully established the foundation for using hematopoietic (blood-producing) stem cells (HSC) from the bone marrow of patients with sickle cell disease (SCD) to treat the disease. The study was led by Dr. Donald Kohn, professor of pediatrics and microbiology, immunology and molecular genetics in the life sciences. Kohn introduced an anti-sickling gene into the HSC to capitalize on the self-renewing potential of stem cells and create a continual source of healthy red blood cells that do not sickle. The breakthrough gene therapy technique for sickle cell disease is scheduled to begin clinical trials by early 2014. The study was published online in the Journal of Clinical Investigation. Gene Therapy Kohn's gene therapy approach using HSC from patient's own blood is a revolutionary alternative to current SCD treatments as it creates a self-renewing normal blood cell by inserting a gene that has anti-sickling properties into HSC. This approach also does not rely on the identification of a matched donor, thus avoiding the risk of rejection of donor cells. The anti-sickling HSC will be transplanted back into the patient's bone marrow and multiplies … Continue reading

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Stem Cell Transplants Clear HIV in Two Patients in Study

Posted: Published on July 3rd, 2013

Two HIV patients in Boston who received stem-cell transplants for cancer had no trace of the AIDS-causing virus after the procedure, suggesting they may have been cured. The two patients, treated at Brigham and Womens Hospital, stopped HIV treatment after the transplants, which in other patients has opened the door for the virus to come roaring back. In one patient there was no sign of the virus 15 weeks after stopping treatment, while the other has gone seven weeks without HIV rebounding, according to results presented today at the International AIDS Societys meeting in Kuala Lumpur. The researchers led by Timothy Henrich of Harvard Medical School and Brigham and Womens Hospital said its too early to conclude the two men have been cured and the virus may be lingering in their brains or gut. Still, their cases are similar to that of Timothy Brown, the so-called Berlin patient, who was the first person to be cured of HIV after getting a bone marrow transplant for leukemia in 2007. While stem-cell transplantation is not a viable option for people with HIV on a broad scale because of its costs and complexity, these new cases could lead us to new approaches to … Continue reading

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Nerve cells can work in different ways with same result

Posted: Published on July 2nd, 2013

Public release date: 1-Jul-2013 [ | E-mail | Share ] Contact: Tim Wall walltj@missouri.edu 573-882-3346 University of Missouri-Columbia Epilepsy, irregular heartbeats and other conditions caused by malfunctions in the body's nerve cells, also known as neurons, can be difficult to treat. The problem is that one medicine may help some patients but not others. Doctors' ability to predict which drugs will work with individual patients may be influenced by recent University of Missouri research that found seemingly identical neurons can behave the same even though they are built differently under the surface. "To paraphrase Leo Tolstoy, 'every unhappy nervous system is unhappy in its own way,' especially for individuals with epilepsy and other diseases," said David Schulz, associate professor of biological sciences in MU's College of Arts and Science. "Our study suggests that each patient's neurons may be altered in different ways, although the resulting disease is the same. This could be a major reason why doctors have difficulty predicting which medicines will be effective with specific individuals. The same problem could affect treatment of heart arrhythmia, depression and many other neurological conditions." It turns out, even happy neurons may be happy in their own way. Neurons have a natural … Continue reading

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Texas Children’s Epilepsy Center Recognized as a Level 4 Epilepsy Center by the National Association of Epilepsy …

Posted: Published on July 2nd, 2013

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AUTISM THERAPY: The Son-Rise Program® – Social Curriculum That Works – Video

Posted: Published on July 2nd, 2013

AUTISM THERAPY: The Son-Rise Program - Social Curriculum That Works http://www.autismtreatment.org/ - Sabiha's son, Adam, had such severe autism, ADHD and learning difficulties that he did not even know who his parents were! ... By: autismtreatment … Continue reading

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Autism Therapy: The Son-Rise Program® – My Son Is HAPPY and Speaking For The First Time! – Video

Posted: Published on July 2nd, 2013

Autism Therapy: The Son-Rise Program - My Son Is HAPPY and Speaking For The First Time! http://www.autismtreatment.org/ - Before The Son-Rise Program, her son was non-verbal and had no interaction beyond a couple of seconds. The Autism Treatmen... By: autismtreatment … Continue reading

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Slaying of boy with severe autism leaves community searching for answers

Posted: Published on July 2nd, 2013

CHICAGO - On a Saturday afternoon in March, Alex Spourdalakis, a 14-year-old boy with severe autism, sat in a darkened room at Loyola University Medical Center with his mother and godmother nearby. In a hushed voice, Dorothy Spourdalakis told an autism care advocate who had come to offer help that she had been at her son's bedside around the clock for weeks. The only time she stepped out of the room, she said, was when he slept - in short catnaps. "She was very calm," said Mary Kay Betz, executive director of the Autism Society of Illinois. "She seemed like a very caring, loving mother who wanted what was appropriate with her son." Three months after that quiet visit, Spourdalakis' relatives arrived at the family's River Grove apartment to discover a grisly sight: Alex had been stabbed to death in his bedroom. His mother and godmother lay nearby after swallowing multiple pills in an apparent suicide attempt, according to officials. The women survived and now face charges of first-degree murder. Though no one condones the crime, experts across the country say the case serves as a shocking example of the toll autism can take on an entire family. While the … Continue reading

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Ask Dr. K: Full stroke recovery possible with early treatment Early treatment best for recovery from stroke

Posted: Published on July 2nd, 2013

Anthony L. Komaroff, M.D. Ask Dr. K Dear Dr. K: My father just had a lacunar stroke. I've never even heard of this. What can you tell me about it? Dear Reader: The most common kind of strokes, called ischemic (iss-KEE-mick) strokes, occur when an artery supplying oxygen-rich blood to a part of the brain is blocked. This leads to the death of some brain cells. Many strokes are caused by blockages of the largest arteries in the brain. A lacunar stroke involves smaller arteries deep in the brain that branch off the large arteries. Because the arteries are smaller, the amount of brain tissue they feed is smaller than the amount fed by the large arteries. Still, lacunar strokes can cause significant disability. (I've put an illustration of the areas of the brain affected by lacunar stroke on my website, AskDoctorK.com.) The smaller arteries deep in the brain are vulnerable because they branch directly off a high-pressure main artery. As a result, high blood pressure can directly damage the walls of these arteries. High blood pressure also can damage the walls of larger arteries and help stimulate the growth of plaques of atherosclerosis, which can block blood flow. The … Continue reading

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Study examines out-of hospital stroke policy at Chicago hospitals

Posted: Published on July 2nd, 2013

Public release date: 1-Jul-2013 [ | E-mail | Share ] Contact: Erin White ewhite@northwestern.edu 847-491-4888 The JAMA Network Journals Implementing an out-of hospital stroke policy in some Chicago hospitals was associated with significant improvements in emergency medical services use and increased intravenous tissue plasminogen activator (tPA) use at primary stroke centers, according to a study published by JAMA Neurology. The study evaluated the relationship between a citywide policy recommending pre-hospital triage of patients with suspected stroke to transport them to the nearest primary stroke center and use of intravenous tPA use. The therapy is used to restore blood flow through blocked arteries in acute ischemic stroke (IS). The study by Shyam Prabhakaran, M.D., M.S., of Northwestern University, Chicago, and colleagues included all admitted patients with stroke and transient ischemic attack (also known as a "mini-stroke" or "warning stroke,") at 10 primary stroke center hospitals in Chicago. The study was conducted from September 2010 to August 2011, which was six months before and six months after the intervention began March 1, 2011. There were 1,075 admissions for stroke and transient ischemic attack in the pre-triage periods and 1,172 admissions in the post-triage period. Compared with the pre-triage period, use of emergency … Continue reading

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New stroke treatment saves Gary woman

Posted: Published on July 2nd, 2013

MERRILLVILLE | After walking upstairs from the laundry room one day in March, Harriet Miles stood in her kitchen, and her whole side grew weak and numb. I remember falling to the floor, she said. A day or two later, she woke up in a hospital room, paralyzed on her right side. The 31-year-old Gary resident did not recognize anyone and did not know how to write or walk. I barely knew how to talk, she said. Unbeknownst to her, emergency responders had taken Miles to Methodist Hospitals Northlake campus in Gary, where doctors suspected she was having a stroke. She did not respond to the standard treatment called IV tPA. Because her boyfriend called 911 so quickly after she fell, Miles was still within the six- to eight-hour window in which doctors could treat her with new technology. She was transferred to the Southlake campus in Merrillville, where Dr. Mayumi Oka, a neurointerventional radiologist, used a tool called Trevo to pull the blood clot from her brain. It was FDA approved in August, and Methodist Hospitals acquired the Trevo over the winter, she said. Her stroke scale score went down to almost zero in one day, Oka said. It … Continue reading

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