Archive for the ‘Gene Therapy Research’ Category
Gym Genetics – Video
Gym Genetics
If you don #39;t like to go to the gym, it may not be your fault. See how you can overcome "DNA" and stay healthy.
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Interview with Brien Foerster — Unravelling the Genetics of Elongated Skulls – Video
Interview with Brien Foerster -- Unravelling the Genetics of Elongated Skulls
In this revealing interview, Brien Foerster sheds lights on DNA testing undertaken on one of the Paracas elongated skulls. More than 300 of these elongated s...
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Gene therapy – Psychology Wiki
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Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, and hereditary diseases in particular. Gene therapy typically aims to supplement a defective mutant allele with a functional one. Although the technology is still in its infancy, it has been used with some success. Antisense therapy is not strictly a form of gene therapy, but is often lumped together with them.
In the 1980s, advances in molecular biology had already enabled human genes to be sequenced and cloned. Scientists looking for a method of easily producing proteins such as insulin, the protein deficient in diabetes mellitus type 1 investigated introducing human genes to bacterial DNA. The modified bacteria then produce the corresponding protein, which can be harvested and injected in people who cannot produce it naturally.
On September 14, 1990 researchers at the U.S. National Institutes of Health performed the first approved gene therapy procedure on four-year old Ashanti DeSilva. Born with a rare genetic disease called severe combined immunodeficiency (SCID), she lacked a healthy immune system, and was vulnerable to every passing germ. Children with this illness usually develop overwhelming infections and rarely survive to adulthood; a common childhood illness like chickenpox is life-threatening. Ashanti led a cloistered existence--avoiding contact with people outside her family, remaining in the sterile environment of her home, and battling frequent illnesses with massive amounts of antibiotics.
In Ashanti's gene therapy procedure, doctors removed white blood cells from the child's body, let the cells grow in the lab, inserted the missing gene into the cells, and then infused the genetically modified blood cells back into the patient's bloodstream. Laboratory tests have shown that the therapy strengthened Ashanti's immune system; she no longer has recurrent colds, she has been allowed to attend school, and she was immunized against whooping cough. This procedure was not a cure; the white blood cells treated genetically only work for a few months, and the process must be repeated every few months. (VII, Thompson [First] 1993).
Although this simplified explanation of a gene therapy procedure sounds like a happy ending, it is little more than an optimistic first chapter in a long story; the road to the first approved gene therapy procedure was rocky and fraught with controversy. The biology of human gene therapy is very complex, and there are many techniques that still need to be developed and diseases that need to be understood more fully before gene therapy can be used appropriately. The public policy debate surrounding the possible use of genetically engineered material in human subjects has been equally complex. Major participants in the debate have come from the fields of biology, government, law, medicine, philosophy, politics, and religion, each bringing different views to the discussion.
Scientists took the logical step of trying to introduce genes straight into human cells, focusing on diseases caused by single-gene defects, such as cystic fibrosis, hemophilia, muscular dystrophy and sickle cell anemia. However, this has been much harder than modifying simple bacteria, primarily because of the problems involved in carrying large sections of DNA and delivering it to the right site on the genome.
In most gene therapy studies, a "normal" gene is inserted into the genome to replace an "abnormal," disease-causing gene. A carrier molecule called a vector must be used to deliver the therapeutic gene to the patient's target cells. Currently, the most common vector is a virus that has been genetically altered to carry normal human DNA. Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner. Scientists have tried to take advantage of this capability and manipulate the virus genome to remove disease-causing genes and insert therapeutic genes.
Target cells such as the patient's liver or lung cells are infected with the vector. The vector then unloads its genetic material containing the therapeutic human gene into the target cell. The generation of a functional protein product from the therapeutic gene restores the target cell to a normal state.
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Gene therapy - Psychology Wiki
T Cell Therapy for ALL | The Children’s Hospital of …
CTL019 is a clinical trial ofT cell therapyfor patients with B cell cancers such as acute lymphoblastic leukemia (ALL), B cell non-Hodgkin lymphoma (NHL), and the adult disease chronic lymphocytic leukemia (CLL). At this time, The Children's Hospital of Philadelphia is the only hospital enrolling pediatric patientson this trial.
In this clinical trial, immune cells called T cells are taken from a patient's own blood. These cells are genetically modified to express a protein which will recognize and bind to a target called CD19, which is found on cancerous B cells. This is how T cell therapy works:
27 patients with acute lymphoblastic leukemia (22 children and 5 adults) have been treatedusing T cell therapy.Of those patients:
Scientists at The Childrens Hospital of Philadelphia and the University of Pennsylvania are very hopeful that CTL019 could in the future be an effective therapy for patients with B cell cancers. However, because so few patients have been treated, and because those patients have been followed for a relatively shorttime,it is critical that more adult and pediatric patients are enrolled in the study to determine whether a larger group of patients with B cell cancers will have the same response, and maintain that response.
At this point CHOP's capability to enroll patients is limited because of the need to manufacture the T cell product used in this therapy. Our goal is to boost enrollment soon, by increasing our manufacturing capabilities and by broadening this study to other pediatric hospitals.
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T cell therapyis a treatment for children and adolescents with fairly advanced B cell acute lymphoblastic leukemia (ALL) and B cell lymphomas, but not other leukemias or pediatric cancers.It is an option for those patients who have very resistant ALL.
Roughly 85 percent of ALL cases are treated very successfully with standard chemotherapy. For the remaining 15 percent of cases, representing a substantial number of children in the United States, chemotherapy only works temporarily or not at all. This is not a treatment for newly diagnosed leukemia, only for patients whose leukemia is not responding to chemotherapy,and whose disease has come back after a bone marrow transplant.
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Rice SPIKE Gene Research Aims to Boost Global Food …
SPIKE gene can boost rice yields in Asia and Southeast Asia significantly and contribute to global food security in coming years, according to Dr. Tsutomu Ishimaru, Plant Breeder of International Rice Research Institute (IRRI)-Japan Collaborative Research Project.
The SPIKE gene is derived from Indonesian landrace, and helps improve yield of rice plants significantly. Dr. Tsutomu Ishimaru says he has achieved yield improvement of around 13-36% compared to IR64 and IRRI146. The SPIKE gene version of IR64 is being tested in several Asian countries including Laos, Indonesia (Java island), India (Tamil Nadu) and Japan. Tests are going on to improve yield in popular rice varieties in South Asia and Southeast Asia using the SPIKE gene. Dr. Tsutomu Ishimaru says, The SPIKE breeding is still in the process for BR11 (popular variety in Bangladesh), Swarna (popular variety in Eastern India), TDK1 (popular variety in Laos), Ciherang (popular variety in Indonesia) and PSBRc18 (popular variety in the Philippines). It will take a few years to finish the transfer of SPIKE to these five varieties.
Though it hasnt been tested yet, the taste, milling quality and cooking properties of traditional rice varieties (such as basmati fragrant rice) are likely to remain same with the introduction of the SPIKE gene through conventional breeding. Increase in yield would also translate into more profit for rice farmers and help in the improvement of the economy of South Asia and Southeast Asia.
Rice is a staple food for almost half of the global population which is growing in Southeast Asia and South Asia. Rice consumption is also increasing in Africa. It is estimated that by 2035, a 26% increase in rice production will be essential to feed the growing population, and according to the International Grains Council (IGC), global rice consumption will surpass rice production in just three years. Dr. Tsutomu Ishimaru hopes SPIKE research will spread to several rice growing countries in future and contribute to global food security.
The IRRI-Japan Collaborative Research Project is supported by the Ministry of Foreign Affairs and the Ministry of Agriculture, Forestry and Fisheries of Japan. The Japan International Research Center for Agricultural Sciences (JIRCAS) is a key partner of the Global Rice Science Partnership (the CGIAR Research Program on Rice), IRRI says.
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Tissue Culture & Genetic Engineering can revive Sugar industry in UP: MODI – Video
Tissue Culture Genetic Engineering can revive Sugar industry in UP: MODI
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Sugar industry can be revived with the help of Tissue Culture & Genetic Engineering in UP. – Video
Sugar industry can be revived with the help of Tissue Culture Genetic Engineering in UP.
02 February 2014, Shri Narendra Modi highlight the growth of cooperatives in Gujarat which along with focusing on tissue culture, genetic engineering and dri...
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Sugar industry can be revived with the help of Tissue Culture & Genetic Engineering in UP. - Video
Genetic signals affecting lipid levels used to investigate heart disease risk
New genetic evidence strengthens the case that one well-known type of cholesterol is a likely suspect in causing heart disease, but also casts further doubt on the causal role played by another type. The findings may guide the search for improved treatments for heart disease.
Most of us have heard of "good cholesterol" and "bad cholesterol" coursing through our bloodstream. In the conventional health wisdom of the past 30 years, having more of the "good" variety (high-density lipoprotein, or HDL) lowers your risk of heart disease, while more of the bad one (low-density lipoprotein, LDL) increases your risk. Indeed, over the years, clinical trials and other studies have found that drugs that lower LDL also lower your probability of heart disease.
On the other hand, drug trials have not shown heart-health benefits to increasing HDL or to lowering triglycerides, a third type of blood lipid. Now a new study co-led by scientists at The Children's Hospital of Philadelphia and Penn Medicine sheds light on the role of genes and blood lipid levels in cardiovascular health. Newer tools for gene analysis show how variations in DNA are underlying actors affecting heart disease -- a major worldwide cause of death and disability.
"Now we are able to pinpoint gene signals that actually cause some of these conditions," says geneticist Brendan J. Keating, D. Phil., of The Center for Applied Genomics at The Children's Hospital of Philadelphia. "Unraveling how genetic variants that influence lipid traits are related to heart disease risk is a step toward designing treatments." Keating and his colleagues, working in large international collaborative groups, are wielding advanced gene-analysis tools to uncover important clues to heart disease.
Keating collaborated with clinical epidemiologist Michael V. Holmes, M.D., Ph.D., of the Perelman School of Medicine at the University of Pennsylvania, in a blood lipid study published online Jan. 27 in the European Heart Journal. Research co-authors were from six countries and various centers, including the University College London in the U.K.
The study team used a recently developed epidemiology tool called Mendelian randomization (MR). MR analyzes genetic variations using a method that identifies genes responsible for particular diseases, independent of confounding factors such as differences in behavior or environmental influences that often limit the conclusions of epidemiology research. This was one of the largest studies to date using MR, as well as the largest to use an allele-score method, described below.
The researchers analyzed DNA data from 17 studies including over 60,000 individuals, of whom more than 12,000 had experienced coronary heart disease, including heart attacks. Because previous studies had found signals from nearly 200 genes to be associated with blood lipid levels, the study team aggregated data into composite groups, called allele scores, for each of three blood lipids: LDL, HDL and triglycerides, then calculated their relationship to coronary heart disease.
As expected, the current study confirmed that higher levels of LDL, the "bad cholesterol," were more likely to cause heart disease. But there were new results: high levels of triglyceride also caused higher risk of heart disease. At the same time, there was little evidence that higher levels of HDL, the "good cholesterol," had a protective effect.
The novelty of their approach, say the authors, lies in their use of a gene score MR analysis using individual participant data. These results build on previous findings and help clarify in further detail the separate roles of triglycerides and HDL in risk for coronary heart disease.
Previous genetic studies, including by Keating and others, found associations among gene variations (single nucleotide polymorphisms, or SNPs) and heart disease, but did not indicate causality, as found in the current study. Holmes said, "These findings are important in understanding which blood lipids cause heart disease, and will enable clinicians to better target those lipids with drugs to reduce the risk of heart disease."
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Genetic signals affecting lipid levels used to investigate heart disease risk
Gene screen eyes mainstream
Genome sequencing holds great potential for diagnosing diseases, finding treatments and ultimately cutting medical costs, experts say, but insurance companies are leery of covering the still-new procedure, preventing it so far from becoming a routine part of medical care.
Boston-based Partners HealthCare is one of just two systems in the country to offer full genome sequencing for clinical patients. The out-of-pocket cost of unlocking your full genetic code, though, is steep: $9,000.
Cost is a barrier, said Heidi Rehm, chief laboratory director at the Partners Center for Personalized Genetic Medicine in Cambridge.
The lab started offering full genome sequencing last August using blood samples to extract information from DNA but it has done the complex analysis for fewer than half a dozen patients since then. Insurance companies didnt cover the costs for any of those patients, Rehm said.
For patients suffering from a range of diseases, from cancer to hearing loss, sequencing can help identify the gene causing the problem and help doctors determine which treatments will be most effective. Genetic sequencing can also tell patients if theyre at risk of developing certain conditions later in life.
The challenge for scientists like Rehm is to prove that this kind of analysis is useful not just for sick patients but for healthy ones.
Can I say every patient should get their genome sequenced? We just dont have the collective evidence and the studies to prove that today, Rehm said. So the insurers are not going to cover everything today.
Insurance companies do pay for some genetic tests those that test specifically for a patients risk of developing breast cancer, for example but theyre still evaluating the benefit of full genome sequencing, which involves much more data and analysis.
We dont have a lot of information yet to make sweeping decisions, said Dr. Neil Minkoff, medical director for the Massachusetts Association of Health Plans. We tend to look at the individual patient or individual physicians request. Its still early in our experience with it.
The states three largest insurers, Blue Cross Blue Shield, Harvard Pilgrim Health Care and Tufts Health Plan, did not respond to requests for comment.
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Gene screen eyes mainstream
Unit 8 – Genetics – 2 – Meiosis – Video
Unit 8 - Genetics - 2 - Meiosis
Meiosis Main Ideas 1) Two cellular divisions 2) Gamete production 3) Increased Genetic Diversity.
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Mendel & Basic Genetics – Video
Mendel Basic Genetics
Unit 8 - Section 1: Introduction to Gregor Mendel #39;s work, rules, and laws that govern patterns of inheritance.
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Genetics – What are the Karyogram and Ideogram? Part 4 of 6 – Video
Genetics - What are the Karyogram and Ideogram? Part 4 of 6
http://www.sicklecellanaemia.org Resource part 4 of 6. Part of the UK Open Education Programme supporting the sharing of educational materials and expertise in sick...
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Central Dogma of Molecular Genetics, with Russian – Video
Central Dogma of Molecular Genetics, with Russian
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Scientific Evidence for Creation CSE BIBLE FORUM Origins 1110 Creation Genetics – Video
Scientific Evidence for Creation CSE BIBLE FORUM Origins 1110 Creation Genetics
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True Genetics – Video
True Genetics
Today i go to Whistler and do a short interview with Phil the owner of True Genetics , a med user who started a seed company in order to breed his own cannab...
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UC Irvine International Imaging Genetics Conference – SOLAR Workshop 2014 part 1 – Video
UC Irvine International Imaging Genetics Conference - SOLAR Workshop 2014 part 1
Genetic Analysis of Quantitative Traits * Using SOLAR-Eclipse for genetic, metagenetic and megagenetic analyses - Tenth International Imaging Genetics Prog...
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BLASTED genetics lush led tent – Video
BLASTED genetics lush led tent
via YouTube Capture.
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BLASTED genetics lush led tent - Video
Attack of the B-Team Advanced Genetics Mod Tutorial Ep.1 – Video
Attack of the B-Team Advanced Genetics Mod Tutorial Ep.1
Attack of the B-Team Advanced Genetics Mod Tutorial: In this very first episode of the new mod pack call Attack of the B-Team i go over most of the machines from the Advanced Genetics mod....
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Why is the a taboo on marrying close relatives? Icest taboo. Genetics explanation. – Video
Why is the a taboo on marrying close relatives? Icest taboo. Genetics explanation.
An incest taboo is any cultural rule or norm that prohibits acts of sexual relations between relatives. Many cultures allow sexual and marital relations betw...
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FTB Monster S01E13 – Huuuge Quarry & Advanced Genetics! – Video
FTB Monster S01E13 - Huuuge Quarry Advanced Genetics!
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Genetics Test Key – Video
Genetics Test Key
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Jeana Gilbert- Genetics Review – Video
Jeana Gilbert- Genetics Review
This video screencast was created with Doceri on an iPad. Doceri is free in the iTunes app store. Learn more at http://www.doceri.com.
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Regenerative Procedures Los Angeles | Dr Martin 323-285-5300 – Video
Regenerative Procedures Los Angeles | Dr Martin 323-285-5300
MetroMD Institute of Regenerative Medicine 7080 Hollywood Blvd Suite 804 Los Angeles, CA 90028 p: (323) 285-5300 http://metromd.net/ Dr. Martin specializes i...
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Gene Mutations In Rock Pigeon Pigmentation Have Implications For Human Medical Research
February 7, 2014
Image Caption: These English trumpeter pigeons -- blue-black on the left and red on the right -- display some of the great diversity of colors among some 350 breeds of rock pigeons. University of Utah biologists discovered three major genes explain color variations in rock pigeons. In the blue-black pigeon, none of the genes have mutations. The red bird is that color because it has a mutant version of a gene named Sox10. The same genes are involved in making some people susceptible to skin cancer and others develop albinism, or a lack of pigment. Credit: Photo courtesy of Sydney Stringham from University of Utah
Brett Smith for redOrbit.com Your Universe Online
A team of American researchers has discovered mutations in three genes that determine feather color in domestic rock pigeons, according to a new study in Current Biology. The same genes direct the pigmentation of human skin meaning the findings may have implications for medical research.
Mutations in these genes can be responsible for skin diseases and conditions such as melanoma and albinism, said study author Michael Shapiro, associate professor of biology at the University of Utah.
In humans, mutations of these genes often are considered bad because they can cause albinism or make cells more susceptible to UV (ultraviolet sunlight) damage and melanoma because the protective pigment is absent or low, said study author Eric Domyan, a biology postdoctoral fellow at the University of Utah. In pigeons, mutations of these same genes cause different feather colors, and to pigeon hobbyists that is a very good thing.
The study team learned that coding and regulatory distinctions in the interactions among the genes Tyrp1, Sox10 and Slc45a2 affect multiple color phenotypes, or appearances, in pigeons. In one instance, scientists learned that a reddish mutation in Tyrp1 arose just once and was spread all through the species by selective mating. Different forms of Tyrp1 make pigeons blue-gray, red or brown.
Variations of Sox10 make pigeons red, regardless of what form Tyrp1 takes, the researchers found. Also, Slc45a2 makes the pigeons colors either very strong or look washed out.
Our work provides new insights about how mutations in these genes affect their functions and how the genes work together, Shapiro said. Many traits in animals, including susceptibility to diseases such as cancer, are controlled by more than one gene. To understand how these genes work together to produce a trait, we often have to move beyond studies of humans. Its difficult to study interactions among the genes in people.
Both Tyrp1 and Sox10 are potential targets for treatment of melanoma, he added. Mutations in Slc45a2 in humans can lead to changes in skin color, including albinism (lack of skin color).
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Gene Mutations In Rock Pigeon Pigmentation Have Implications For Human Medical Research
Pilot program offers genomic testing to certain patients …
FEB. 10, 2014
BY SARA WYKES
Euan Ashley
A small group of patients at Stanford Hospital & Clinics and Lucile Packard Children's Hospital Stanford now can have their DNA deciphered as part of a new pilot program.
The goal of the program, the Clinical Genomics Service, is to help doctors better diagnose and treat genetic conditions. In the pilot phase, genomic testing will be limited to patients with "mystery" diseases (typically children), patients with unexplained hereditary cancer risk, patients with inherited cardiovascular or neurological disease, and those with severe, unexplained drug reactions. Potential participants must be referred by a physician, and the clinical genomics team will then determine whether patient cases are suitable for sequencing.
"I am very excited to bring the pioneering work of Stanford genomic scientists directly to the bedside of our patients," said Euan Ashley, MCRP, DPhil, associate professor of medicine and of genetics and co-director of the new Clinical Genomics Service. "Because of the foresight and support of our leadership, we have a remarkable opportunity to bring world-leading Stanford science to Stanford patients fast and first."
The service will use an integrated approach that includes professional genetic counseling, the most advanced genome sequencing technology available and expert interpretation by molecular genetic pathologists and other physicians with expertise in this emerging and complex field. It will be closely integrated with a broad range of other diagnostic genetic testing now being offered by pathology services at the adult and children's hospitals.
"Stanford has a special wealth of information and analysts," said Jason Merker, MD, PhD, assistant professor of pathology, the service's other co-director. "We involved physicians, other health-care providers, bioethicists, bioinformaticians and other researchers, inviting everyone to voice their thoughts for the broadest, deepest discussions possible on how to apply these new methods and knowledge to clinical care."
Michael Snyder, PhD, director of the Stanford Center for Genomics and Personalized Medicine and chair of genetics, as well as other members of the center, have played a pivotal role in the design and implementation of the service. Also, included in those discussions were Carlos Bustamante, PhD, a professor of genetics who was named a 2010 MacArthur Fellow for his work in genetic sequencing, and Michael Cherry, PhD, associate professor of genetics and principal investigator in several genome database projects.
"This new service can represent the best definition of the term personalized medicine," said Amir Dan Rubin, president and CEO of Stanford Hospital & Clinics. "The collaboration of our world-class experts in patient care and scientific research will advance the leading edge of knowledge in genome sequencing, bringing greater value, in the most responsible way, to what we offer our patients. Our goal is to use this new technology for early and accurate diagnosis and treatment for patients now and to learn and share that knowledge with medicine's new future."
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Pilot program offers genomic testing to certain patients ...