Genetic engineering part 2 – Video
Genetic engineering part 2
Lol.
By: Gayatri JC
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Genetic engineering part 2 - Video
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A Third Act For Biofuels
Editors note:Ryan Clarke is a biochemistry PhD candidate with an interest in genetic engineering. He is a published scientist with a background in synthetic biology and social analytics.James Hyun is aPhD student in the life sciences with a background in molecular biology. He haspublished numerous scientific paperswheregenetically engineered microorganisms were used to produce high value therapeutic proteins.
In the midst of the lowest oil prices our nation has seen in six years, its easy to forget that the primary energy source in the world is in finite supply. Fracking and extraction from shale have enhanced the potential amount of obtainable oil (and Americas total reserves have the potential to be the highest in the world), but these means are still limited by the Earths reserves.
In fact, British Petroleum projects that we will deplete the Earths oil reservoirs in roughly 50 years. With this sobering reality looming over our heads, we can look to recent advances in the biotechnology of biofuel production as a potential solution. Moving beyond bioethanol, the unsuspecting platform of algae may be the most promising.
Our vision for a carbon neutral world is one in which the ultimate goal is replacing petroleum-based energy consumption systems with clean energy production/consumption. However, replacing fossil fuel-derived energy with renewable sources such as wind, solar or hydro is a daunting task. These electricity-producing energy sources have a lower energy density, which is measured in joules per liter or kilogram, or BTUs (British Thermal Unit):
Furthermore, solar, wind and hydro cannot be controlled with an on and off switch. Rather, the electricity generated must be used immediately or stored in batteries and is considerably more expensive than fossil fuels. On a large scale, a coal mine or oil field, for instance, yields five to 50 times more power per square meter than a solar facility, 10 to 100 times more than a wind farm, and 100 to 1,000 times more than a biomass plant like corn.
If we want to avoid hitting the brick wall of a global energy drought when we tap our final oil reserves, we must do what humans do best: solve the problems we have created ourselves. Big oil and other major players in the market are highly cognizant that current petroleum supplies are finite, so they have been heavily investing (i.e. BP has invested $4 billion since 2005) in alternative energy sources to alleviate our dependency on classic fossil fuels. A significant portion of this funding is focused on biofuels, which might be the most realistic answer to the fossil fuel issue.
Biomasses to Bioethanol.The conversion of cellulose (a prolific sugar produced in plants), such as corns and sugar cane, to ethanol through chemically catalytic procedures has been a major prospect for ridding petroleum dependency for 20 years in the U.S.
Unfortunately, it turns out our existing combustion engines can only handle 10 percent ethanol mixtures with our gasoline (denoted as E10 fuels) on average, unless the engine has been modified or produced (flex-fuel engines) to handle 85 percent ethanol (E85 fuels), which is much less common. In America, there are roughly 10 million flex-fuel vehicles out of the total 250 million, so an infrastructure turnover is required for ethanol to be a plausible alternative.
The utmost defeating point for the ethanol argument is that oil yields 50 times more energy than ethanol from corn and 10 times more than ethanol from sugar cane, according to ecological economist Cutler Cleveland.
If we were to commit to ethanol as a petroleum replacement and ethanol werethe sole source used to achieve the 2020 federal mandates for renewable fuel, then 100 percent of the corn currently available in the U.S. would be required. To meet these mandates and maintain todays 30 percent corn crop utilization would require an increase in corn harvest by 423 percent.
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A Third Act For Biofuels
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Genetics of the Porsche 918 Spyder – Video
Genetics of the Porsche 918 Spyder
http://bit.ly/PorscheAutoGallery | This is the 2015 Porsche 918 Spyder. A work of art. Sculpted like no other...for those with a passion for life, style and ...
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Genetics of the Porsche 918 Spyder - Video
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Quick Medical Genetics – Disorders of Phenylalanine and Tyrosine Metabolism – Video
Quick Medical Genetics - Disorders of Phenylalanine and Tyrosine Metabolism
This is a lecture about the disorders of phenylalanine and tyrosine metabolism, a family of genetic disorders. The lecture is intended for trainees and medical professionals. Lecture by Philip...
By: Quick Medical Genetics
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Quick Medical Genetics - Disorders of Phenylalanine and Tyrosine Metabolism - Video
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Innovation, research and Public Health policies in Personalized Medicine. Discussion – Video
Innovation, research and Public Health policies in Personalized Medicine. Discussion
Instituto Roche Forum on Personalized Medicine: Challenges for the next decade 25th september 2014, Palacio de Congresos y Exposiciones de Galicia. Session 2...
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Innovation, research and Public Health policies in Personalized Medicine. Discussion - Video
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Autologous Stem Cell Transplant | Animation Video – Video
Autologous Stem Cell Transplant | Animation Video
What is a Autologous Stem Cell Transplant? Most stem cells are in your bone marrow. You also have some in your blood that circulate from your bone marrow. Bo...
By: Medical.Animation.Videos.Library
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Autologous Stem Cell Transplant | Animation Video - Video
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Observing stem cells maturing into blood cells in living mouse
In the bone marrow, blood stem cells give rise to a large variety of mature blood cells via progenitor cells at various stages of maturation. Scientists from the German Cancer Research Center (DKFZ) have developed a way to equip mouse blood stem cells with a fluorescent marker that can be switched on from the outside. Using this tool, they were able to observe, for the first time, how stem cells mature into blood cells under normal conditions in a living organism. With these data, they developed a mathematical model of the dynamics of hematopoiesis. The researchers have now reported in the journal Nature that the normal process of blood formation differs from what scientists had previously assumed when using data from stem cell transplantations.
Since ancient times, humankind has been aware of how important blood is to life. Naturalists speculated for thousands of years on the source of the body's blood supply. For several centuries, the liver was believed to be the site where blood forms. In 1868, however, the German pathologist Ernst Neumann discovered immature precursor cells in bone marrow, which turned out to be the actual site of blood cell formation, also known as hematopoiesis. Blood formation was the first process for which scientists formulated and proved the theory that stem cells are the common origin that gives rise to various types of mature cells.
"However, a problem with almost all research on hematopoiesis in past decades is that it has been restricted to experiments in culture or using transplantation into mice," says Professor Hans-Reimer Rodewald from the German Cancer Research Center (Deutsches Krebsforschungszentrum, DKFZ). "We have now developed the first model where we can observe the development of a stem cell into a mature blood cell in a living organism."
Dr. Katrin Busch from Rodewald's team developed genetically modified mice by introducing a protein into their blood stem cells that sends out a yellow fluorescent signal. This fluorescent marker can be turned on at any time by administering a specific reagent to the animal. Correspondingly, all daughter cells that arise from a cell containing the marker also send out a light signal.
When Busch turned on the marker in adult animals, it became visible that at least one third (approximately 5000 cells) of a mouse's hematopoietic stem cells produce differentiated progenitor cells. "This was the first surprise," says Busch. "Until now, scientists had believed that in the normal state, very few stem cells -- only about ten -- are actively involved in blood formation."
However, it takes a very long time for the fluorescent marker to spread evenly into peripheral blood cells, an amount of time that even exceeds the lifespan of a mouse. Systems biologist Prof. Thomas Hfer and colleagues (also of the DKFZ) performed mathematical analysis of these experimental data to provide additional insight into blood stem cell dynamics. Their analysis showed that, surprisingly, under normal conditions, the replenishment of blood cells is not accomplished by the stem cells themselves. Instead, they are actually supplied by first progenitor cells that develop during the following differentiation step. These cells are able to regenerate themselves for a long time -- though not quite as long as stem cells do. To make sure that the population of this cell type never runs out, blood stem cells must occasionally produce a couple of new first progenitors.
During embryonic development of mice, however, the situation is different: To build up the system, all mature blood and immune cells develop much more rapidly and almost completely from stem cells.
The investigators were also able to accelerate this process in adult animals by artificially depleting their white blood cells. Under these conditions, blood stem cells increase the formation of first progenitor cells, which then immediately start supplying new, mature blood cells. In this process, several hundred times more cells of the so-called myeloid lineage (thrombocytes, erythrocytes, granulocytes, monocytes) form than long-lived lymphocytes (T cells, B cells, natural killer cells) do.
"When we transplanted our labeled blood stem cells from the bone marrow into other mice, only a few stem cells were active in the recipients, and many stem cells were lost," Rodewald explains. "Our new data therefore show that the findings obtained up until now using transplanted stem cells can surely not be reflective of normal hematopoiesis. On the contrary, transplantation is an exception [to the rule]. This shows how important it is that we actually follow hematopoiesis under normal conditions in a living organism."
The scientists in Rodewald's department, working together with Thomas Hfer, now also plan to use the new model to investigate the impact of pathogenic challenges to blood formation: for example, in cancer, cachexia or infection. This method would also enable them to follow potential aging processes that occur in blood stem cells in detail as they occur naturally in a living organism.
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Observing stem cells maturing into blood cells in living mouse
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Stem cells offer promising key to new malaria drugs: US research
NEW YORK (Thomson Reuters Foundation) - Human stem cells engineered to produce renewable sources of mature, liver-like cells can be grown and infected with malaria to test potentially life-saving new drugs, according to researchers at the Massachusetts Institute of Technology.
The advance comes at a time when the parasitic mosquito-borne disease, which kills nearly 600,000 people every year, is showing increased resistance to current treatment, especially in Southeast Asia, according to the World Health Organization.
The liver-like cells, or hepatocytes, in the MIT study were manufactured from stem cells derived from donated skin and blood samples.
The resulting cells provide a potentially replenishable platform for testing drugs that target the early stage of malaria, when parasites may linger and multiply in the liver for weeks before spreading into the bloodstream.
Sangeeta Bhatia, a biomedical engineer and senior author of the MIT report, told the Thomson Reuters Foundation that the breakthrough study not only showed that these liver-like cells could host a malaria infection but also described a way to mature the young cells so that an adult-like metabolism, necessary for drug development, could be established.
The study is published in the Feb. 5 online issue of Stem Cell Reports.
Stem cells retain the genetic makeup of their donors, affording researchers the potential to test drugs against a large variety of genetic types and a variety of diseases.
"This allows us to explore in depth how different diseases affect different people, in this case malaria," Bob Palay, chairman and CEO of Cellular Dynamics International (CDI), told the Thomson Reuters Foundation.
"This allows you to study it in a dish and find new drugs," he added, noting that CDI uses blood samples for its stem cells.
Before this development, researchers tested new drugs using human liver cells from cadavers and cancerous liver cells.
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Stem cells offer promising key to new malaria drugs: US research
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Theres a lot to love at The Body Shop
DROPS of Youth Bouncy Sleeping Mask is designed to be left on overnight without washing off. The lightweight, pliable mask molds itself like a second skin, making skin look younger and fresher.
The Body Shop introduces its Drops of Youth, Red Musk Fragrance, and Limited-Edition Forbidden Flower collections.
Drops of Youth, made from edelweiss flower stem cells sourced from the Alps, replenishes your skin in the most natural way. Left on overnight, the lightweight mask is like a second skin. In the morning, skin feels smooth and hydrated, and looks younger and fresher.
Red Musk, The Body Shops most unconventional scent to date, turns up the heat.
The Limited-Edition Forbidden Flower Collection is a body care and fragrance line inspired by the poppy flower.
DROPS of YouthWonderblur is a skin smoother that reduce fine lines and pores for an even, flawless finish.
Known for its thrust in protecting the planet, The Body Shop never tests its products on animals. The line has a Community Fair Trade program, where high-quality natural ingredients are sourced in different parts of the world where small stakeholders and artisans can benefit.
The Drops of Youth and Red Musk collections are available at The Body Shop stores nationwide, while Forbidden Flower Collection is available at selected The Body Shop branches. SM Advantage Card members can now earn and redeem points in all The Body Shop stores.
THE RED Musk Fragrance Collection. I wanted to create a fragrance that wasnt the typical girly girl scent. I wanted to change the rules of fragrance. Instead, I used the sensuality and the warmth of spices blended withmusk to approach femininity differently, says Corinne Cachen, master perfumer.
FORBIDDEN Flower Body Butter gives your skin the pleasure of pure potent moisture.
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Theres a lot to love at The Body Shop
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FAQ Part 1: MEsenchymal Stem cell therapy for CAnadian MS patients (MESCAMS) – Video
FAQ Part 1: MEsenchymal Stem cell therapy for CAnadian MS patients (MESCAMS)
The Multiple Sclerosis Society of Canada and the Multiple Sclerosis Scientific Research Foundation have announced a $4.2 million grant in support of the MEse...
By: MSSocietyCanada
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FAQ Part 1: MEsenchymal Stem cell therapy for CAnadian MS patients (MESCAMS) - Video
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Creation Of Rejuvenated Cell By Stem Cell Therapy – The Line Clinic – Video
Creation Of Rejuvenated Cell By Stem Cell Therapy - The Line Clinic
Stem cell therapy has become reality which was just possibilities and thoughts of science few days before. This amazing innovation makes life more secured an...
By: Nicky Lee
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Creation Of Rejuvenated Cell By Stem Cell Therapy - The Line Clinic - Video
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Comment: T cell therapy for children with aggressive leukaemia – Video
Comment: T cell therapy for children with aggressive leukaemia
Visit http://www.ecancer.org for more. Dr Bollard (George Washington University, Washington, USA) discusses key abstracts showcased at ASH 2014 with ecancert...
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Comment: T cell therapy for children with aggressive leukaemia - Video
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terapia celular para cinomose – distemper stem cell therapy – Video
terapia celular para cinomose - distemper stem cell therapy
Caso de cinomose tratado com terapia celular - unesp - botucatu Stem cell therapy for distemper in a dog.
By: Jean Joaquim
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terapia celular para cinomose - distemper stem cell therapy - Video
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MPN Research Foundation initiates exploratory gene editing project
Chicago, IL (PRWEB) February 12, 2015
One of the primary goals of the MPN Research Foundation is to catalyze new avenues of research into the cause(s) and potential treatments for the blood cancer group polycythemia vera, essential thrombocythemia and myelofibrosis. As an increasing number of new approaches to cancer research are emerging, the Foundation has a new project which hopefully will ensure any applicable technology is tested in MPNs.
One such approach to cancer treatment is CRISPR-cas9, an acronym for Clustered Regularly Interspaced Short Palindromic Repeats. CRISPR-cas9 allows for the targeting of specific mutations. This approach is currently being tested in cystic fibrosis, sickle cell anemia, and some cancers. It is believed to be particularly effective for diseases associated with single point genetic mutations, which suggests that it may be applicable to MPNs, for which there are several known genetic mutations suck as JAK2V617F and CALR.
Currently MPNs are under-represented among the conditions being studied by CRISPR researchers. In addition, not enough is known about the potential impact of this treatment on the people living with MPNs. To ensure that MPNs are sufficiently explored by the CRISPR research world the MPN Research Foundation will be taking action on two fronts:
As a cautionary note, this technology, although exciting and currently the subject of much scientific and commercial activity, is in its preliminary stages. The long-term costs and benefits are not yet clear. But given its potential to fundamentally alter the course of MPNs and other diseases, the Foundation believes that further study is critical. The RFP is scheduled to go out in the end of February. Check http://www.mpnresearchfoundation.org for further details.
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MPN Research Foundation initiates exploratory gene editing project
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Mutated gene causes 70% cancer risk
Health officials hit back at e-cig claims
Health professionals say more research is needed to prove using e-cigarettes is a good way to quit smoking.
According to new health figures, Australian women are far less likely to survive a heart attack than men.
Research says high factor sunscreen can't be relied on to protect against the deadliest skin cancer form.
A British study using skin electrodes has found men experience greater levels of emotion than women.
High protein diets may protect against stroke, especially if they contain a lot of fish, scientists say.
Driving too much is bad for your health, according to a study of 40-thousand middle-aged Australians.
Researchers say the financial crisis may have led to thousands of suicides in Europe and North America.
Biologists have devised a new weapon against malaria by genetically engineering mosquitoes.
Stomach-shrinking bariatric surgery beats other forms of treatment in bringing about remission of diabetes.
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Mutated gene causes 70% cancer risk
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Studies Find More Genetic Links to Obesity
Latest Diet & Weight Management News
WEDNESDAY, Feb. 11, 2015 (HealthDay News) -- New research offers more evidence that genes play a significant role in obesity.
The findings may help explain why some people are more likely to put on extra pounds and develop obesity-linked conditions, the investigators said.
The researchers analyzed genetic samples from more than 300,000 people and identified more than 140 locations across their sets of DNA that play a role in obesity. They also pinpointed new biological pathways that play important roles in body weight and fat distribution.
The findings appear in two companion papers published Feb. 11 in the journal Nature.
This is the first step toward identifying individual genes involved in body shape and size, the researchers said. The proteins produced by the genes could offer targets for the development of new drugs to fight obesity.
One of the papers focused on genes that affect where fat is stored in the body, which affects health risk. For example, people with more belly fat are more likely to have metabolic conditions such as type 2 diabetes and cardiovascular disease than those with more fat in the hips or distributed throughout the body.
"We need to know these genetic locations because different fat deposits pose different health risks," senior author Karen Mohlke, a professor of genetics at the University of North Carolina School of Medicine, said in a University of Michigan Health System news release.
"If we can figure out which genes influence where fat is deposited, it could help us understand the biology that leads to various health conditions, such as insulin resistance/diabetes, metabolic syndrome and heart disease," she explained.
The other paper looked at the link between genes and body mass index (BMI), an estimate of body fat based on height and weight. The researchers said they found 97 genetic associations linked to BMI. They also found that genetic locations associated with BMI are linked to areas that control factors such as appetite and energy use.
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Studies Find More Genetic Links to Obesity
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More genetic links to obesity discovered
New research offers more evidence that genes play a significant role in obesity.
The findings may help explain why some people are more likely to put on extra pounds and develop obesity-linked conditions, the investigators said.
The researchers analyzed genetic samples from more than 300,000 people and identified more than 140 locations across their sets of DNA that play a role in obesity. They also pinpointed new biological pathways that play important roles in body weight and fat distribution.
The findings appear in two companion papers published Feb. 11 in the journal Nature.
Play Video
There is new concern over another possible effect of antibiotics: obesity. Dr. Martin Blaser talks to the "CBS This Morning" co-hosts about the l...
This is the first step toward identifying individual genes involved in body shape and size, the researchers said. The proteins produced by the genes could offer targets for the development of new drugs to fight obesity.
One of the papers focused on genes that affect where fat is stored in the body, which affects health risk. For example, people with more belly fat are more likely to have metabolic conditions such as type 2 diabetes and cardiovascular disease than those with more fat in the hips or distributed throughout the body.
"We need to know these genetic locations because different fat deposits pose different health risks," senior author Karen Mohlke, a professor of genetics at the University of North Carolina School of Medicine, said in a University of Michigan Health System news release.
"If we can figure out which genes influence where fat is deposited, it could help us understand the biology that leads to various health conditions, such as insulin resistance/diabetes, metabolic syndrome and heart disease," she explained.
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More genetic links to obesity discovered
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New option for Jewish genetic testing
Image via shutterstock.com
When a Jewish couple is planning their wedding or anticipating starting a family, they probably arent thinking much about rare genetic conditions. But JScreen, an educational and screening program, urges couples to add genetic testing to their to-do list. And by offering home-based testing, JScreen hopes to eliminate any obstacles to this process.
Based at Emory Universitys Department of Human Genetics, JScreen (jscreen.org) provides a Web-based portal for individuals to request a genetic-screening kit. Participants provide a saliva sample most genetic tests involve a blood draw and mail it back for analysis. Before receiving the kit, participants must view an educational video and enter health information that is reviewed by an Emory genetic counselor.
We all carry [recessive genes for] various genetic diseases. We just dont know what they are, said Karen Grinzaid, a genetic counselor and instructor at Emory University School of Medicine and the senior director of outreach initiatives for JScreen.
The problem occurs when both parents are carriers of the same disease. In that case, each of their offspring has a 25 percent chance of manifesting the condition.
According to Emorys Department of Human Genetics, about one in five Ashkenazi Jews in the U.S. carries a genetic disease. However, most dont have a family history of the disease and are unaware of their status of carriers. In fact, 80 percent of babies with genetic diseases are born to parents with no known family history of that disease.
The only way to know if you are a carrier for a Jewish genetic disease is to have an affected child or be screened, Grinzaid said. For the vast majority of couples, genetic screening gives couples reassurance that theyre not at risk.
Saliva samples returned to Emorys lab are tested for 40 diseases prevalent in the Jewish community. Nineteen of them are more common in Ashkenazi populations, and 21 of them are common in Jews of Sephardic or Mizrahi (Middle Eastern) origins. Testing for an additional 47 diseases found in the general population is available at no extra cost. Many of the diseases included in the tests are fatal, and all impact the individuals quality of life.
Results take less than four weeks. If they are negative, individuals are notified via email. Those who are identified as carriers speak via phone or videoconference to an Emory University genetic counselor about their results and options. They might also be referred to a local genetic counselor for more extensive counseling. Grinzaid said that about 2 percent of couples will be found to be carriers of the same disease.
If both members of a couple carry the same genetic disease, they have several options. One is to undergo in-vitro fertilization using pre-implantation genetic diagnosis. This technology allows embryos to be tested for the affected gene before being implanted. Other options include using a donor egg or sperm, or pursuing adoption.
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New option for Jewish genetic testing
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Lecture 2 Developmental Genetics – Video
Lecture 2 Developmental Genetics
By: UVUProfessor
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Lecture 2 Developmental Genetics - Video
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The Origin of Nations Pt 2 – ‘GENETICS’ Nat Stock – Video
The Origin of Nations Pt 2 - #39;GENETICS #39; Nat Stock
Other Channels we recommend https://www.youtube.com/BibleProphecyHD https://www.youtube.com/ChristadelphianVideo Bible Truth about Prophecy, - Welcome to our channel run by the ...
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The Origin of Nations Pt 2 - 'GENETICS' Nat Stock - Video
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Quick Medical Genetics – Pseudohypoparathyroidism, Albright hereditary osteodystrophy – Video
Quick Medical Genetics - Pseudohypoparathyroidism, Albright hereditary osteodystrophy
This is a lecture about the genetic diseases pseudohypoparathyroidism (PHP), pseudopseudohypoparathyroidism (PPHP), and Albright hereditary osteodystrophy (A...
By: Quick Medical Genetics
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Quick Medical Genetics - Pseudohypoparathyroidism, Albright hereditary osteodystrophy - Video
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Mendelian Genetics 2 – Video
Mendelian Genetics 2
Table of Contents: 00:16 - Independent assortment 01:08 - Incomplete dominance 01:09 - Independent assortment 01:27 - Incomplete dominance 03:05 - Codominanc...
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Mendelian Genetics 2 - Video
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Choice Genetics Drives Pork Producer Productivity. – Video
Choice Genetics Drives Pork Producer Productivity.
Dr Derek Petry of Choice Genetics provides an overview of the focus of the company on how they help pork producers achieve better results. For more information visit Choice Genetics at http://www....
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Choice Genetics Drives Pork Producer Productivity. - Video
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LYSOGENE, UMM and AU Collaborate To Develop IND-supporting Preclinical Studies In GM1-gangliosidosis
Collaboration entails development of gene therapy treatment for severe neurodegenerative disease GM1-gangliosidosis
LYSOGENE, a leading, clinical stage gene therapy biotechnology company committed to the development and commercialization of breakthrough treatments for severe orphan pathologies affecting the central nervous system (CNS), recently announced that it has entered into a strategic collaboration with the University of Massachusetts Medical School (UMMS) in Worcester, Massachusetts, and Auburn University (AU) in Auburn, Alabama. Through the collaboration, LYSOGENE, UMMS and AU will develop IND-supporting preclinical studies in GM1-gangliosidosis, a rare, inherited disorder characterized by severe neurological impairment, using adeno-associated virus (AAV) gene therapy technology.
The collaboration will combine LYSOGENEs outstanding translational and clinical expertise in gene therapy for CNS disorders with the unique preclinical expertise and infrastructure of UMMS and AU to design and test innovative AAV-based gene therapy approaches to treat GM1-gangliosidosis.
The development of a potential treatment for GM1-gangliosidosis using AAV gene therapy was initiated in 2005 by Miguel Sena-Esteves, PhD, associate professor in the Neurology Department and the Gene Therapy Center at UMMS, and Douglas R. Martin, PhD, associate professor in the Scott-Ritchey Research Center and Department of Anatomy, Physiology & Pharmacology at AU. The approach developed by the investigators uses AAV vectors to treat the entire brain and spinal cord after injection of only a few intracranial sites. Preclinical studies demonstrated a remarkable extension in lifespan from 8 months in untreated GM1 cats to greater than 4.5 years in AAV-treated cats, with dramatic improvements in quality of life. Results were published in Science Translational Medicine in 2014 (McCurdy, V.J., et al., Sustained normalization of neurological disease after intracranial gene therapy in a feline model. Science Translational Medicine, 2014. 6(231): p. 231ra48).
We are thrilled by our collaboration with University of Massachusetts Medical School and Auburn University, which constitutes a significant step towards the development of a treatment for patients affected with GM1-gangliosidosis, a severely debilitating disease. For each of these patients and their families, there is currently no option and an urgent need for a safe and effective therapy, said Karen Aiach, founding president and CEO of LYSOGENE. AAV-based therapies are particularly suitable for inherited disorders of the CNS. In this new program, LYSOGENE will leverage its unique capacity to develop these therapies and bring them to patients with unmet needs. We will also reinforce our scientific and technology base through our collaboration with leaders in the field.
Collaborating with LYSOGENE will allow us to leverage their clinical and translational expertise and advance the development of a gene transfer therapy for treating patients affected with GM1-gangliosidosis, said Sena-Esteves. In our minds, what ultimately matters is the ability to deliver a potential treatment to the children suffering from this horrible disease. Ultimately, thats what drives us all.
About Gangliosidosis with GM1 GM1-gangliosidosis is a rare inherited neurodegenerative disorder characterized by severe cognitive and motor developmental delays resulting in death of most patients at a very young age.
It is caused by mutations in the GLB1 gene, which encodes an enzyme called beta-galactosidase necessary for recycling of a molecule (GM1-ganglioside) in neurons. This brain lipid is indispensable for normal function, but its overabundance causes neurodegeneration, resulting in the severe neurological symptoms of GM1-gangliosidosis.
GM1 affects 1 in 100,000 - 200,000 newborns and is inherited in an autosomal recessive pattern. GM1-gangliosidosis can be classified into three major clinical phenotypes according to the age of onset and severity of symptoms: Type I (infantile), Type II (late infantile/juvenile) and Type III (adult). There is currently no treatment for this disease.
About LYSOGENE LYSOGENE is a clinical stage biotechnology company committed to the development and commercialization of innovative therapies for patients affected with rare disorders and high unmet medical needs. LYSOGENEs team translated its rAAVrh10 lead product for Sanfilippo from bench to bedside in an unprecedented fashion over the last years. Its lead product is for Sanfilippo syndrome, a neurodegenerative lysosomal storage disorder considered to be a perfect model for gene therapy. LYSOGENE is currently expanding its pipeline to additional diseases with high unmet medical needs. Lysogene was launched in 2009. It completed a Series A financing in May 2014 with leading life sciences investors Sofinnova Partners, BPI Innobio and Novo AIS.
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LYSOGENE, UMM and AU Collaborate To Develop IND-supporting Preclinical Studies In GM1-gangliosidosis
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Spinal Cord Injury Program at Children’s Specialized Hospital – Video
Spinal Cord Injury Program at Children #39;s Specialized Hospital
Details description of the Spinal Cord Injury program at Children #39;s Specialized Hospital.
By: Children #39;s Specialized Hospital
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Spinal Cord Injury Program at Children's Specialized Hospital - Video
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