Archive for the ‘Gene Therapy Research’ Category

Genetics of Complex Diseases with Prof Nick Martin Part 4
This is the first #39;Where Is Science Heading? #39; presentation at the University of Queensland, presented on May 14, 2013. Senior scientist Prof Nick Martin, from the Queensland Institute of Medical...

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Genetics of Complex Diseases with Prof Nick Martin Part 4 - Video

Genetics of Complex Diseases with Prof Nick Martin Part 3
This is the first #39;Where Is Science Heading? #39; presentation at the University of Queensland, presented on May 14, 2013. Senior scientist Prof Nick Martin, from the Queensland Institute of Medical...

By: Luke Sartor

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Genetics of Complex Diseases with Prof Nick Martin Part 3 - Video

Rosetrees 25th Anniversary Event - Professor Steve Jones
Professor Steve Jones - Department of Genetics, Evolution Environment UCL Nature, Nurture or Neither? What the public does understand about genetics 17 Oct...

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Rosetrees 25th Anniversary Event - Professor Steve Jones - Video

About 80 per cent of people who have asthma also have hay fever or eczema. Photo: shutterstock.com

Weve long known that allergies such as eczema, hay fever and asthma are caused by a combination of genetic and environmental factors. But weve been unclear about which genes are responsible.

This week, my international research team published a study identifying ten genetic variants that increase a persons risk of developing allergies. The study, published in Nature Genetics, showed that the more variants a person has, the higher the risk of developing an allergic condition.

Allergies occur when the immune system thinks harmless substances such as dust are a threat. Photo: shutterstock.com

In Australia and New Zealand, about 30% of children have an allergy of some type. This occurs because their immune system incorrectly believes a harmless substance such as dust or pollen is a threat. These allergy-causing substances are called allergens.

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An allergy develops when a particular type of cell from our immune system, B lymphocyte, produces antibodies against an allergen. This process often occurs at a young age and is known as sensitisation, that is, a person becomes sensitive to a particular substance in the environment.

From then on, if the same person is exposed to the same allergen, the antibodies produced by the B cells bind to the allergen. This triggers a series of reactions that culminate with inflammation appearing in the skin (eczema), nose (hay fever) or lungs (asthma). The symptoms of this inflammation are itching, sneezes and wheezes.

One in 10 Australians and New Zealanders have asthma. Photo: shutterstock.com

One in five Australians and New Zealanders will develop eczema in their lifetime; one in five will suffer with hay fever; and one in ten live with asthma, which can cause serious illness and can be fatal. About 80% of people who have asthma also have hay fever, and many also have eczema.

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Genetics of allergies is nothing to sneeze at

LONDON - Two men with HIV have been off AIDS drugs for several months after receiving stem-cell transplants for cancer that appear to have cleared the virus from their bodies, researchers reported on Wednesday.

Both patients, who were treated in Boston and had been on long-term drug therapy to control their HIV, received stem-cell transplants after developing lymphoma, a type of blood cancer.

Since the transplants, doctors have been unable to find any evidence of HIV infection, Timothy Henrich of Harvard Medical School and Brigham and Women's Hospital in Boston told an International AIDS Society conference in Kuala Lumpur.

While it is too early to say for sure that the virus has disappeared from their bodies altogether, one patient has now been off antiretroviral drug treatment for 15 weeks and the other for seven weeks.

Last July Henrich first reported that the two men had undetectable levels of HIV in their blood after their stem-cell treatment, but at that time they were still taking medicines to suppress HIV.

Using stem-cell therapy is not seen as a viable option for widespread use, since it is extremely expensive, but the latest cases could open new avenues for fighting the disease, which infects about 34 million people worldwide.

The latest cases resemble that of Timothy Ray Brown, known as "the Berlin patient", who became the first person to be cured of HIV after receiving a bone marrow transplant for leukaemia in 2007. There are, however, important differences.

While Brown's doctor used stem cells from a donor with a rare genetic mutation, known as CCR5 delta 32, which renders people virtually resistant to HIV, the two Boston patients received cells without this mutation.

"Dr. Henrich is charting new territory in HIV eradication research," Kevin Robert Frost, chief executive officer of the Foundation for AIDS Research, which funded the study, said in a statement.

Scientific advances since HIV was first discovered more than 30 years ago mean the virus is no longer a death sentence and the latest antiretroviral AIDS drugs can control the virus for decades.

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Stem-cell therapy wipes out HIV in 2 patients

Rochester, NY (PRWEB) July 03, 2013

A new study showed that anti-herpes medication improves the thought processes of patients with bipolar disorder. This is an important finding since people with bipolar disorder often complain and exhibit symptoms of cognitive dysfunction, particularly in the domains of attention, memory, and executive function. (1)

Another study published in the journal Biological Psychiatry in 2004 already identified a link between herpes simplex virus type 1 (HSV-1) and cognitive dysfunction in bipolar disorder. (2) Thus, researchers have begun to consider the possibility that anti-herpes medication might improve the mental disorder.

Participants in the 4-month trial were evaluated every 2 weeks for a change in mood symptoms using the MADRS and YMRS (two standard mental tests). The results came as a surprise. (1)

Dr. Jennifer L. Payne, the lead author of the study, expected that antiviral medication will decrease depression levels. However, it didnt happen. Instead, the patients showed an improvement in overall cognitive function. (1)

The idea behind this is that the [herpes] virus in the [central nervous system] could trigger mood, cognitive, or psychotic symptoms. If you treat patients you see this all the time: A patient comes under some kind of stress and becomes psychiatrically ill. One of the thoughts is that [herpes simplex virus] could underlie some of that psychopathology." (1) - Dr. Payne, director of the womens mood disorders center at Johns Hopkins University, Baltimore

She further noted that, If these findings hold up, it would indicate that as clinicians, we need to be testing for HSV-1 and treating it in our patients. (1)

Since the herpes virus has been linked to Bipolar Disorder, polyDNA recommends killing the herpes virus as soon as possible to help prevent the onset of BPD. Gene-Eden-VIR boosts the immune system and helps it attack and eliminate the latent herpes virus. A recent post marketing clinical study showed that Gene-Eden-VIR is safe and effective. Up to 70% of those studied reported a decrease in viral symptoms. (3)

Each ingredient of Gene-Eden-VIR was chosen through a scientific approach. Scientists scanned thousands of scientific and medical papers published in various medical and scientific journals around the world to identify the safest, most effective natural ingredients that target the latent form of herpes. (4)

To learn more about Gene-Eden-VIR and herpes, visit http://www.gene-eden-kill-virus.com/Herpes-Remedy.php. All orders of Gene-Eden-VIR are completely confidential, and no information is shared or sold to any third party. Privacy is assured.

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Study: Oral Herpes May Trigger Bipolar Disorder (BPD); polyDNA recommends Natural Herpes Remedy

July 3, 2013 In a discovery that sheds new light on the aggressiveness of certain breast cancers, Whitehead Institute researchers have identified a transcription factor, known as ZEB1, that is capable of converting non-aggressive basal-type cancer cells into highly malignant, tumor-forming cancer stem cells (CSCs). Intriguingly, luminal breast cancer cells, which are associated with a much better clinical prognosis, carry this gene in a state in which it seems to be permanently shut down.

The researchers, whose findings are published this week in the journal Cell, report that the ZEB1 gene is held in a poised state in basal non-CSCs, such that it can readily respond to environmental cues that consequently drive those non-CSCs into the dangerous CSC state. Basal-type breast carcinoma is a highly aggressive form of breast cancer. According to a 2011 epidemiological study, the 5-year survival rate for patients with basal breast cancer is 76%, compared with a roughly 90% 5-year survival rate among patients with other forms of breast cancer.

"We may have found a root source, maybe the root source, of what ultimately determines the destiny of breast cancer cells -- their future benign or aggressive clinical behavior," says Whitehead Founding Member Robert Weinberg, who is also a professor of biology at MIT and Director of the MIT/Ludwig Center for Molecular Oncology.

Transcription factors are genes that control the expression of other genes, and therefore have a significant impact on cell activities. In the case of ZEB1, it has an important role in the so-called epithelial-to-mesenchymal transition (EMT), during which epithelial cells acquire the traits of mesenchymal cells. Unlike the tightly-packed epithelial cells that stick to one another, mesenchymal cells are loose and free to move around a tissue. Previous work in the Weinberg lab showed that adult cancer cells passing through an EMT are able to self-renew and to seed new tumors with high efficiency, hallmark traits of CSCs.

Other earlier work led by Christine Chaffer, a postdoctoral researcher in the Weinberg lab, demonstrated that cancer cells are able to spontaneously become CSCs. Now Chaffer and Nemanja Marjanovic have pinpointed ZEB1, a key player in the EMT, as a gene critical for this conversion in breast cancer cells.

Breast cancers are categorized into at least five different subgroups based on their molecular profiles. More broadly these groups can be subdivided into the less aggressive 'luminal' subgroup or more aggressive 'basal' subgroup. The aggressive basal-type breast cancers often metastasize, seeding new tumors in distant parts of the body. Patients with basal breast cancer generally have a poorer prognosis than those with the less aggressive luminal-type breast cancer.

Chaffer and Marjanovic, a former research assistant in the Weinberg lab, studied non-CSCs from luminal- and basal-type cancers and determined that cells from basal cancers are able to switch relatively easily into CSC state, unlike luminal breast cancer cells, which tend to remain in the non-CSC state.

The scientists determined that the difference in ZEB1's effects is due to the way the gene is marked in the two types of cancers. In luminal breast cancer cells, the ZEB1 gene is occupied with modifications that shut it down. But in basal breast cancer cells, ZEB1's state is more tenuous, with repressing and activating markers coexisting on the gene. When these cells are exposed to certain signals, including those from TGF, the repressive marks are removed and ZEB1 is expressed, thereby converting the basal non-CSCs into CSCs.

So what does this new insight mean for treating basal breast cancer?

"Well, we know that these basal breast cancer cells are very plastic and we need to incorporate that kind of thinking into treatment regimes," says Chaffer. "As well as targeting cancer stem cells, we also need to think about how we can prevent the non-cancer stem cells from continually replenishing the pool of cancer stem cells. For example, adjuvant therapies that inhibit this type of cell plasticity may be a very effective way to keep metastasis at bay."

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Gene that controls aggressiveness in breast cancer cells identified

July 3, 2013 Whitehead Institute researchers have determined that the transcription factor Nanog, which plays a critical role in the self-renewal of embryonic stem cells, is expressed in a manner similar to other pluripotency markers. This finding contradicts the field's presumptions about this important gene and its role in the differentiation of embryonic stem cells.

A large body of research has reported that Nanog is allelically regulated -- that is, only one copy of the gene is expressed at any given time -- and fluctuations in its expression are responsible for the differences seen in individual embryonic stem (ES) cells' predilection to differentiate into more specialized cells. These studies relied on cells that had a genetic marker or reporter inserted in the DNA upstream of the Nanog gene. This latest research, published in this week's edition of the journal Cell Stem Cell, suggests that results from studies based on this approach could be called into question.

To quantify the variations in Nanog expression, Dina Faddah, a graduate student in the lab of Whitehead Institute Founding Member Rudolf Jaenisch, looked at hundreds of individual mouse ES cells with reporters inserted immediately downstream of the Nanog gene. One Nanog allele had a green reporter, while the other had a red reporter, allowing Faddah to determine which of the two alleles was being expressed.

After analyzing the results and comparing them to the expression of a "housekeeping" gene and other pluripotency factors, Faddah concluded that, regardless of the cells' growing environment, most ES cells express both Nanog alleles and the variability of this expression corresponds to that of the other genes.

When Faddah tested the established method of inserting a reporter upstream of Nanog, her results reflected the earlier studies' conclusions. However, when she checked the results with other forms of gene expression analysis, she found that the method was not a faithful indicator of Nanog's expression.

"The way the reporter was inserted into the DNA seems to disrupt the regulation of the alleles, so that when the reporter says Nanog isn't being expressed, it actually is," says Faddah.

For Jaenisch, this is an instructional tale that should be heeded by all geneticists.

"Clearly, the conclusions for this particular gene need to be reconsidered," says Jaenisch, who is also a professor of biology at MIT. "And it raises the question for other genes. For some genes, there might be similar issues. For other genes, they might be more resistant to this type of disturbances caused by a reporter."

This work is supported Vertex Scholars Program, the National Science Foundation (NSF), Jerome and Florence Brill Fellowship, Croucher and Ludwig Research Fellowship, the National Institutes of Health (NIH) (1 F32 GM099153-01A1, HD 045022 and R37CA084198).

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Long-held assumption of gene expression in embryonic stem cells challenged

Public release date: 3-Jul-2013 [ | E-mail | Share ]

Contact: Vanessa Wasta wasta@jhmi.edu 410-614-2914 Johns Hopkins Medicine

Johns Hopkins cancer scientists have discovered that a little-described gene known as FAM190A plays a subtle but critical role in regulating the normal cell division process known as mitosis, and the scientists' research suggests that mutations in the gene may contribute to commonly found chromosomal instability in cancer.

In laboratory studies of cells, investigators found that knocking down expression of FAM190A disrupts mitosis. In three pancreatic cancer-cell lines and a standard human-cell line engineered to be deficient in FAM190A, researchers observed that cells often had difficulty separating at the end of mitosis, creating cells with two or more nuclei. The American Journal of Pathology published a description of the work online May 17, which comes nearly a century after German scientist Theodor Boveri linked abnormal mitosis to cancer. Until now, there had been no common gene alteration identified as the culprit for cancer-linked mitosis.

"These cells try to divide, and it looks like they succeed, except they wind up with a strand that connects them," explains Scott Kern, M.D., professor of oncology and pathology at Johns Hopkins University School of Medicine and its Kimmel Cancer Center. "The next time they try to divide, all the nuclei come together, and they try to make four cells instead of two. Subsequently, they try to make eight cells, and so on." Movies of the process taken by Kern's laboratory are available on the journal Web site.

Kern's group previously reported that deletions in the FAM190A gene could be found in nearly 40 percent of human cancers. That report, published in 2011 in the journal Oncotarget, and the current one are believed to be the only published papers focused solely on FAM190A, which is frequently altered in human cancers but whose function has been unknown. Alterations in FAM190A messages may be the third most common in human cancers after those for the more well-known genes p53 and p16, Kern says.

"We don't think that a species can exist without FAM190, but we don't think severe defects in FAM190A readily survive among cancers," Kern says. "The mutations seen here are very special they don't take out the whole gene but instead remove an internal portion and leave what we call the reading frame. We think we're finding a more subtle defect in human cancers, in which mitosis defects can occur episodically, and we propose it may happen in about 40 percent of human cancers."

Abnormalities in FAM190A may cause chromosomal imbalances seen so commonly in cancers, Kern says. Multipolar mitosis is one of the most common functional defects reported in human cancers, and more than 90 percent of human cancers have abnormal numbers of chromosomes.

Kern says he plans to study FAM190A further by creating lab models of the subtle defects akin to what actually is tolerated by human cancer cells.

###

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Cancer-linked FAM190A gene found to regulate cell division

Public release date: 3-Jul-2013 [ | E-mail | Share ]

Contact: Nicole Rura rura@wi.mit.edu 617-258-6851 Whitehead Institute for Biomedical Research

CAMBRIDGE, Mass. (July 3, 2013) Whitehead Institute researchers have determined that the transcription factor Nanog, which plays a critical role in the self-renewal of embryonic stem cells, is expressed in a manner similar to other pluripotency markers. This finding contradicts the field's presumptions about this important gene and its role in the differentiation of embryonic stem cells.

A large body of research has reported that Nanog is allelically regulatedthat is, only one copy of the gene is expressed at any given timeand fluctuations in its expression are responsible for the differences seen in individual embryonic stem (ES) cells' predilection to differentiate into more specialized cells. These studies relied on cells that had a genetic marker or reporter inserted in the DNA upstream of the Nanog gene. This latest research, published in this week's edition of the journal Cell Stem Cell, suggests that results from studies based on this approach could be called into question.

To quantify the variations in Nanog expression, Dina Faddah, a graduate student in the lab of Whitehead Institute Founding Member Rudolf Jaenisch, looked at hundreds of individual mouse ES cells with reporters inserted immediately downstream of the Nanog gene. One Nanog allele had a green reporter, while the other had a red reporter, allowing Faddah to determine which of the two alleles was being expressed.

After analyzing the results and comparing them to the expression of a "housekeeping" gene and other pluripotency factors, Faddah concluded that, regardless of the cells' growing environment, most ES cells express both Nanog alleles and the variability of this expression corresponds to that of the other genes.

When Faddah tested the established method of inserting a reporter upstream of Nanog, her results reflected the earlier studies' conclusions. However, when she checked the results with other forms of gene expression analysis, she found that the method was not a faithful indicator of Nanog's expression.

"The way the reporter was inserted into the DNA seems to disrupt the regulation of the alleles, so that when the reporter says Nanog isn't being expressed, it actually is," says Faddah.

For Jaenisch, this is an instructional tale that should be heeded by all geneticists.

"Clearly, the conclusions for this particular gene need to be reconsidered," says Jaenisch, who is also a professor of biology at MIT. "And it raises the question for other genes. For some genes, there might be similar issues. For other genes, they might be more resistant to this type of disturbances caused by a reporter."

Continued here:
Study challenges long-held assumption of gene expression in embryonic stem cells

Tribune-Herald

Actress Roseanne Barr criticized genetic engineering before the Hawaii County Council on Tuesday.

Barr, who owns a farm in Hamakua, spoke in favor of a bill to limit the use of genetically modified crops on the Big Island, saying people need to listen to independent research on the biotech industry.

When science is applied and scientific analysis is applied, a correct answer is harvested, and thats what needs to happen, she said.

Barr also addressed papaya farmers, many of whom grow virus-resistant transgenic varieties.

You know, the Hawaiians, everybody here is very giving and they would probably bend over backwards and help you burn those papayas and grow something decent, she said.

Several papaya growers spoke against the bill, referring to it as a threat to their livelihood.

The star of the 1990s sitcom, Roseanne, also filmed a reality television show about life on her 40-acre macadamia nut and livestock farm.

Titled Roseannes Nuts, the show ran for one season in 2011 on the Lifetime network.

Barr ventured into politics last year, seeking the 2012 presidential nomination for the Green Party.

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Actress speaks out against GMO on island

Eric Lander on Recent, and Surprising, Advances in Genetic Medicine
Eric Lander, Professor of Biology at the Massachusetts Institute of Technology, at the 2013 Aspen Ideas Festival.

By: AspenInstitute

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Eric Lander on Recent, and Surprising, Advances in Genetic Medicine - Video

Public release date: 3-Jul-2013 [ | E-mail | Share ]

Contact: Sue McGreevey smcgreevey@partners.org 617-724-2764 Massachusetts General Hospital

An international research team has used a novel approach to identify genetic factors that appear to influence susceptibility to cholera. The findings by investigators from Massachusetts General Hospital (MGH), the Broad Institute and the International Center for Diarrhal Disease Research, Bangladesh (ICDDR,B) indicate the importance of pathways involved in regulating water loss in intestinal cells and of the innate immune system in the body's response to the bacteria that causes cholera, which affects from 3 to 5 million people each year and causes more than 100,000 deaths.

"We sought to understand cholera by studying the genetics of a population that has been affected by the disease for centuries people in the Ganges River Delta of Bangladesh," says Regina LaRocque, MD, of the MGH division of Infectious Diseases, a co-senior author of the report receiving online publication in Science Translational Medicine. "Our findings are just a first step, but they demonstrate how combining ancient history with the current impact of an infectious disease can be a powerful way of identifying human genes that are important to disease outcome."

Cholera is contracted by consuming water or food contaminated with the bacteria Vibrio cholerae, which releases a toxic protein upon reaching the small intestine. This toxin binds to the intestinal surface, causing severe diarrhea and potentially death from dehydration. Cholera or a cholera-like illness has been reported in the Ganges Delta for centuries, and most recent global outbreaks of the disease originated in that region. A potential fingerprint of cholera's genetic impact could be the relative rarity in the region of blood type O, which also confers an increased risk of severe cholera symptoms. The persistence of cholera in the Ganges Delta would be expected to exert an evolutionary force on the population, since individuals with gene variants that reduce their susceptibility to the disease would be more likely to survive and pass those variants along to their children.

To search for genomic regions that affect cholera susceptibility, the team employed a new two-step approach. The first step used a method called Composite of Multiple Signals (CMS) developed by the Broad team led by co-senior author Pardis Sabeti, MD, DPhil to scan the genomes of 126 individuals from the Ganges Delta for patterns that signal a long-term increase in the prevalence of particular DNA segments, indicating the effects of natural selection. That scan identified 305 regions under selective pressure, many of which are involved in two important biologic functions: regulation of the passage of water through intestinal cells via structures called potassium channels and a signaling pathway involved in both the innate immune system and the maintenance of the intestinal lining.

The second step directly tested the potential impact of these selected regions on cholera susceptibility by comparing the genomes of 105 cholera patients from the region with those of 167 individuals who did not contract the disease despite being exposed to it in their homes. That comparison found that the genomic region most strongly associated with cholera susceptibility in this population was one that the CMS scan had indicated as being under strong selection pressure. Genes in this region relate to an innate immune signaling pathway. LaRocque's team had previously shown this pathway to be activated by exposure to cholera toxin, and the current study identified the potential involvement of several additional genes in that pathway.

"An exciting feature of this project was the way it brought together the expertise in population genetics and natural selection of our collaborators at the Broad Institute with the leadership in addressing the health problems of the developing world of the ICDDR,B team led by Firdausi Qadri, with whom we have a longstanding collaboration," says LaRocque, an assistant professor of Medicine at Harvard Medical School. "Understanding the basic biology of a disease is fundamental to making clinically relevant advances in treatment. Our laboratory is now working on further studies of the innate immune response to cholera, and we believe this work will be highly relevant to developing improved vaccines."

###

Lead and co-corresponding author of the Science Translational Medicine report is Elinor Karlsson of the Broad Institute. In addition to Firdausi Qadri, PhD, of ICDDR,B, who is a co-senior author along with LaRocque and Sabeti, the paper's co-authors are Jason Harris, Ok Sarah Shin, Crystal Ellis, Stephen Calderwood and Edward Ryan, MGH Infectious Diseases; Christine Becker and Lynda Stuart, MGH Developmental Immunology; Shervin Tabrizi, Ilya Shylakhter, Nick Patterson, Colm O'Dushlaine and Stephen F. Schaffner, Broad Institute; Atiqur Rahman, Fahima Chowdhury and Alaullah Sheikh, ICDDR,B; and Sameer Gupta, Harvard Medical School. Support for the study includes National Institutes of Health grants TW007409, AI058935, AI079198 and NIH Innovator Award DP2-OD006514-01; grants from the Howard Hughes Medical Institute, the American Cancer Society and the Packard Foundation; and an MGH Claflin Distinguished Scholar Award.

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Genetic signals reflect the evolutionary impact of cholera

Association for Molecular Pathology v. Myriad Genetics Timeline
Made for the course US Patent Litigation Practices at NCTU.

By: Tsai-fang Chen

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Association for Molecular Pathology v. Myriad Genetics Timeline - Video

Breast Cancer genetics in PR: Population specific aspects
University of Puerto Rico, Medical Sciences Campus Cancer Genetics Course A 5-day intensive course in the genetics of cancer for upper level undergraduates, ...

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Breast Cancer genetics in PR: Population specific aspects - Video

Instant Results With Nutrition Customized Fat Loss Metabolism Overcome Your Genetics
Access To This Instant Results! Click here: http://x.vu/georgecustomizedfatloss Surprising nutrition truths that will empower you with new fat burnning knowl...

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Instant Results With Nutrition Customized Fat Loss Metabolism Overcome Your Genetics - Video

CENTER FOR GENETICS AND SOCIETY - Marcy Darnovsky on Initiative Radio with Angela McKenzie
Marcy Darnovsky, PhD is the Executive Director at the Center for Genetics and Society. She speaks and writes widely on the politics of human biotechnology, f...

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CENTER FOR GENETICS AND SOCIETY - Marcy Darnovsky on Initiative Radio with Angela McKenzie - Video

Zebrafish Genetics exhibit at the Royal Society Summer Science Exhibition 2013
Zebrafish Genetics exhibit at the Royal Society Summer Science Exhibition 2013. Find out more about this exhibit here: http://sse.royalsociety.org/2013/exhib...

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Zebrafish Genetics exhibit at the Royal Society Summer Science Exhibition 2013 - Video

Introduction to Gene Therapy
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Introduction to Gene Therapy - Video

Conditions for Gene Therapy
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Worlds First 3 Parent Babies Coming Soon
The UK is paving the way for a special in vitro fertilization process that gives one baby DNA from three parents. It #39;s an incredible advancement that can eli...

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Worlds First 3 Parent Babies Coming Soon - Video

July 2, 2013 A single session of a gene therapy developed by the Universitat Autnoma de Barcelona (UAB) cures Sanfilippo Syndrome A in animal models. This syndrome is a neurodegenerative disease that affects between 1 and 9 out of every 100,000 children, and causes the death of the child on reaching adolescence.

The study has been published in The Journal of Clinical Investigation.

Sanfilippo Syndrome type A, or Mucopolysaccharidosis type IIIA (MPSIIIA), is a neurodegenerative disease caused by mutations in the gene that encodes the enzyme sulfamidase. Mutations in this gene lead to deficiencies in the production of the enzyme, which is essential for the breakdown of substances known as glycosaminoglicans. If these substances are not broken down, they accumulate in the cells and cause neuroinflammation and organ dysfunction, mainly in the brain, but also in other parts of the body. Children born with this mutation are diagnosed from the age of 4 or 5. They suffer neurodegeneration, causing mental retardation, aggressiveness, hyperactivity, sleep alterations, loss of speech and motor coordination, and they die in adolescence.

A team of researchers headed by the director of the UAB's Centre for Animal Biotechnology and Gene Therapy (CBATEG), Ftima Bosch, has developed a gene therapy treatment that cures this disease in animal models, with pre-clinical studies in mice and dogs. The treatment consists of a single surgical intervention in which an adenoassociated viral vector is injected into the cerebrospinal fluid, the liquid that surrounds the brain and the spinal cord. The virus, which is completely harmless, genetically modifies the cells of the brain and the spinal cord so that they produce sulfamidase, and then spreads to other parts of the body, like the liver, where it continues to induce production of the enzyme.

Once the enzyme's activity is restored, glycosaminoglican levels return to normal for life, their accumulation in cells disappears, along with the neuroinflammation and dysfunctions of the brain and other affected organs, and the animal's behaviour and its life expectancy return to normal. While mice with the disease lived only up to 14 months, those given the treatment survived as long as healthy ones.

This is a joint project between the UAB and the pharmaceutical company Esteve. The study has been published in the online edition of The Journal of Clinical Investigation.

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Gene therapy cures a severe pediatric neurodegenerative disease in animal models

Giving hope: The promise of stem cell therapy
MANILA - As stem cell treatment begins to gain traction among Filipinos, a surgeon believes the country is now entering a new phase in medicine.

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Giving hope: The promise of stem cell therapy - Video

#39;Bebot #39; Villar: Stem cell therapy made me weaker
MANILA, Philippines - A government official on Thursday said he is no believer in stem cell therapy after undergoing the procedure but getting zero health be...

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STEM CELL THERAPY discussed in ABS-CBN, UMAGANG KAY GANDA, PUNTO por PUNTO
Please like the official Facebook page of Philippine Medical Association https://www.facebook.com/PhilippineMedicalAssociation Anthony Taberna discusses Lega...

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STEM CELL THERAPY discussed in ABS-CBN, UMAGANG KAY GANDA, PUNTO por PUNTO - Video