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Featured post

New Stem Cell Cancer Treatment on the Horizon?

Stem Cell Research is an amazing field right now, and promises to be a powerful and potent tool to help us live longer and healthier lives. Just last month, for example, Stem Cell Therapy was used to restore sight in patients with severe retinal deterioration, allowing them to see clearer than they had in years, or even decades.

Stem Cell Therapy is the Future

Now, there is another form of Stem Cell Treatment on the horizon—this one of a very different form. Stem Cells have now been used as a mechanism to deliver medical treatment designed to eliminate cancer cells, even in hard to reach places. One issue with current cancer treatments is that, treatments that are effective at treating tumors on the surface of the brain cannot be performed safely when the tumor is deeper within the brain’s tissues.

Stem Cell Science

Stem Cells have the fantastic ability to transform into any other kind of cell within the human body, given the appropriate stimulation. As of today, most of these cells come from Embryonic Lines, but researchers are learning how to backwards engineer cells in the human body, reverting them back to their embryonic state. These cells are known as Induced Pluripotent Stem Cells.

How Does This Stem Cell Cancer Treatment Work?

Using genetic engineering, it is possible to create stem cells that are designed to release a chemical known as Pseudomonas Exotoxin, which has the ability to destroy certain tumor cells in the human brain.

What is Pseudomonas Exotoxin?

Pseudomonas Exotoxin is a compound that is naturally released by a form of bacteria known as Pseudomonas Aeruginosa. This chemical is toxic to brain tumor cells because it prevents polypeptides from growing longer, essentially preventing the polypeptides from growing and reproducing. When used in a specific manner, this toxin has the ability to destroy cancerous and malignant tissue without negatively impacting healthy tissue. In addition to its potential as a cancer treatment, there is also evidence that the therapy could be used for the treatment of Hepatitis B.

PE and Similar Toxins Have been Used Therapeutically in the Past

As of now, this chemical, which we will refer to for the rest of the article as PE, has been used as a cancer treatment before, but there are major limitations regarding the use of PE for particular cancers, not because of the risks of the treatment, but because of the lack of an effective method to deliver the medication to where it is needed.

For example, similar chemicals have been highly effective in the treatment of a large number of blood cancers, but haven’t been nearly as effective in larger, more inaccessible tumors. The chemicals break down or become metabolized before they can fully do their job.

How do Stem Cells Increase the Effectiveness of PE Cancer Treatment

Right now, PE has to be created in a laboratory before it is administered, which is not very effective for these embedded cancers. By using Stem Cells as an intermediary, it is possible to deliver the medication to deeper areas of the brain more effectively, theoretically highly increasing the efficacy of the treatment.

The leader of this Stem Cell Research is Harvard researcher Dr. Khalis Shah. His goal was to find an effective means to treat these deep brain tumors which are not easily treated by methods available today. In utilizing Stem Cells, Dr. Shah has potentially found a means by which the stem cells can constantly deliver this Cancer Toxin to the tumor area. The cells remain active and are fed by the body, which allows them to provide a steady stream of treatment that is impossible to provide via any other known method.

This research is still in its early stages, and has not yet reached human trials, but in mice, the PE Toxin worked exactly as hypothesized and was able to starve out tumors by preventing them from replicating effectively.

Perhaps this might seem a bit less complicated than it actually is. One of the major hurdles that had to be overcome was that this Toxin would normally be strong enough to kill the cell that hosted it. In order for the Stem Cells to release the cancer, they had to be able to withstand the effects of PE, themselves. Using genetic engineering, Dr. Shah and his associates were able to create a cell that is capable of both producing and withstanding the effects of the toxin.

Stem Cell delivered medical therapy is a 21st century form of medical treatment that researchers are just beginning to learn how to effectively utilize. Essentially, this treatment takes a stem cell and converts it into a unique symbiotic tool capable of feeding off of the host for energy in order to perform a potentially life-saving function. It’s really quite fascinating.

How Does PE Not Damage or Kill Brain Cells Indiscriminately?

You might be concerned about the idea of a patient having a toxin injected into the brain to cure a disease. It sounds almost like a dangerous, tribal, homeopathic remedy. In reality, the researchers have been able to harness the destructive power of the toxin and re-engineer it so that it directly targets cancer cells while having limited negative effects on healthy, non-cancerous tissue.

The toxin does its damage after it has been absorbed by a cell. By retooling the toxin so that it does not readily absorb into healthy cells, the dangers associated with having such a potentially dangerous toxin in the brain are seriously and significantly mitigated.

Beyond that, Dr. Shah and his associates have been able to take steps to effectively “turn off” PE while it is inside the host stem cell, and only activates when it has entered the cancerous tissue. Dr. Shah explains that, although this research has only been conducted in animal subjects, there is no known reason why the effectiveness and safety of the treatment would not be applicable to human patients.

In this treatment, surgeons remove as much of the tumor as possible from the brain, and insert the engineered Stem Cells submerged in a sterile gel in the area where the tumor was removed or partially still exists. Researchers found that, when they used this treatment on laboratory rats, they could tell through imaging and analysis that the modified PE toxin effectively killed the cancer cells, and that this cancer treatment effectively lengthened the life of the rat, as compared to control subjects.

What’s the Next Step?

What's Next in Healthcare?

Of course, cancer treatment is far more complex than a single treatment, no matter how effective that treatment may be. Because human cancer treatment is a comprehensive therapy approach, the end goal of this research is to create a form of therapy in which the method used in animal subjects is combined with other existing approaches, increasing and maximizing the effectiveness of the comprehensive treatment.

Featured post

New Advancement in Induced Pluripotent Stem Cell Research

A recent change in how well we understand stem cells may make it easier for scientists and researchers to gather stem cells for use in scientific research as well as medical application. A new study was released in the research publication, Cell, which was performed by representatives from the University of California San Francisco.

One of the issues which hinder the use of stem cells as a more widespread treatment or field of research is that researchers and patients have a bottleneck of available healthy stem cell lines which can be used for research. Researchers hope that this new discovery will allow future scientific discoveries and applications in the areas of creating new and healthy tissue for patients with kidney failure or any other form of organ tissue failure. The future of medical therapy lies with Stem Cell Research, but many other forms of treatment, including Hormone Replacement Therapy, are already in practice today.

Researchers have discovered that it is possible to essentially “flip a switch” in an adult cell, reverting it back to the preliminary state at which cells existed in one of the earliest stages of development—the embryonic stem cell. Medical researchers hypothesize that Stem Cell treatments could be used for a variety of medical health issues which plague the world today, including kidney failure, liver disease, and Type-1 and Type-2 Diabetes.

Use of Embryonic Stem Cells Contentious

There is an ethical issue in Stem Cell Research today. Many Pro-Life Advocates are vociferously against the use of Embryonic Stem Cells harvested from procedures such as fertility treatments designed for conception. They believe that the use of embryonic stem cells harvested from donors and couples looking to conceive is unethical.

Using current research, it may be possible to bypass this ethical quandary completely by using adult cells and converting them into embryonic stem cells. Furthermore, because these stem cells are genetic derivatives of the patient from which the adult cells were harvested, this potentially paves the way for patient-specific medical treatments using stem cells.

After adult cells have been converted back into Embryonic Stem Cells, it will be possible to convert them into any possible cell that the patient needs or would benefit from.

Hijacking the Blueprint of the Cell Allows Scientists to Revert Adult Cells to their Earliest State

Researchers have increased the capacity to produce Embryonic Stem Cells by identifying previously unrecognized biochemical processes which tell human cells how to develop. In essence, researchers have discovered how the body blueprints cells, and can change the blueprints so that a new cell is made.

By utilizing these newly recognized pathways, it is possible to create new stem cells more quickly than ever before. One of the researchers explains the implications of this research. Dr. Miguel Ramalho-Santos is an associate professor of obstetrics, medicine, and cancer research at the University of California San Francisco. Dr. Ramalho-Santos is also a member of the Broad Center of Regenerative Medicine and Stem Cell Research.

He explains that these stem cell discoveries have the ability to alter the way that the medical sciences can take advantage of stem cells with regard to both cancer research and regenerative medicine. Dr. Ramalho-Santos was the lead researcher for this study, and the research was largely funded by the Director of the National Institutes of Health New Innovator Award, granted to promising young researchers which are leading highly innovative and promising medical research studies.

Dr. Ramalho-Santos’ research builds off of earlier research which discovered that it was possible to take adult cells and turn them back into embryonic stem cells. These stem cells don’t have any inherent aging processes, and they can be turned into any other kind of tissue. In the process of this conversion, the adult cells lose all of their unique characteristics, leaving them in an ultimately immature and malleable state.

This earlier research was conducted by researchers from UC San Francisco in partnership with Dr. Shinya Yamanaka from Kyoto University and Gladstone Institutes. These entities all gained a piece of the Nobel Prize in Physiology or Medicine from their part in the study.

Pluripotent Stem Cells vs. Embryonic Stem Cells

Thus far, we’ve described these cells as Embryonic Stem Cells, but in fact, the more accurate term for these cells are Induced Pluripotent Stem Cells (IPS). These cells are biologically and functionally similar to Embryonic Stem Cells, but have a different name because they are sourced from adult cells. The difference between Induced Pluripotent Stem Cells and Embryonic Stem Cells is that Induced Pluripotent Stem Cells do seem to retain some of the characteristics of their previous state, which appears to limit their ability to convert into any other type of cell. This new research identifies new pathways by which it may be possible to increase the number of cells that an individual IPS Cell can turn into, perhaps allowing them to convert into any other kind of human cell.

Induced Pluripotent Stem Cells are not explicitly considered an alternative to Embryonic Stem Cells, but are considered a different approach to produce similar cells. If researchers fully uncover the mechanisms of how to reprogram these cells, it will lower many barriers to stem cell research and the availability of stem cell treatments.

As of today, researchers have figured out how to make these Induced Pluripotent Stem Cells, but the percentage of adult cells which are reverted successfully is quite low, and frequently, these cells still show some aspects of specialization, which limits their use.

How Do Scientists Make Stem Cells From Adult Cells?

There are genes within every cell which have the ability to induce pluripotency, reverting the cell to an earlier stage of specialization. The initial stage of this process is the result of activating Yamanaka Factors, specific genes that initiate this reversion process.

As of today, this process of de-maturation is not completely understood, and researchers realized from the start that the cells they created were not truly identical to Embryonic Stem Cells, because they still showed signs of their former lives, which often prevented them from being successfully reprogrammed.

The new research conducted by Dr. Ramalho-Santos appears to increase our knowledge regarding how these cells work, and how to program them more effectively. Dr. Ramalho-Santos and his team discovered more genes associated with these programming/reprogramming processes, and by manipulating them, they have increased the viability and range of particular stem cells.

It appears that these genetic impulses are constantly at play to maintain the structure and function of a cell, and that by systematically removing these safeguards, it is possible to increase the ability to alter these cells.

This research increases researchers’ ability to produce these stem cells, by increasing the ability of medical scientists to produce adequate numbers of stem cells, while also increasing the range of potential treatment options by more effectively inducing the total pluripotency which is available in Embryonic Stem Cells. This research may also help scientists treat certain forms of cancer which are the result of malfunctions of these genes.

Genetic engineering: a guide for kids by Tiki the Penguin

Genetic engineering (GE for short) is about scientists altering the ‘recipes’ for making life the genes which you find in all living things. Doing this is very clever and seems to be very useful. Back in the 1990s, many ‘Greens’ campaigned against genetic engineering and still do. They predicted disaster but that hasn’t happened. Nobody has died from eating genetically modified (GM) food. They were also worried about the private GE companies’ ownership of the recipes genes for making these new life forms. So is genetic engineering okay? My guide explains the basics but it’s up to you to make up your own mind about GE.

Finding your way around my GE Guide You can jump to any part that interests you from the table below. If you want to start at the beginning, click the green arrow below (forward to ‘Genes, snails and whales’).

Table of contents

Genes, snails and whales What makes you human or me a penguin? What are genes?

Tried and tested Life on Earth has been around for a long time so it’s been well tested.

Adapt or die Only the fittest life survives. Here’s how it does it.

Coils and corkscrews About that incredible stuff DNA.

Copycat: How DNA copies itself.

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Genetic engineering: a guide for kids by Tiki the Penguin

Biochemistry – Federal University of …


The scientific evolution of man science and need for science, history of science, classifications, modern scientific methods; science and mans environment; terrestrial and cosmic life; harnessing science climate and vegetation. Production, processing conversion, distribution: energy resources: solar thermal nuclear energy- fossil fuels, estimates of energy reserves in Nigeria. Key revolutions in technology: technology, electronics and computer technology, robotics and cybernetics, everyday applications: technology history of technological evolution/ practice in Nigeria, role of technology in the national economy. Education for technology past, present and future; constraints in the utilization of new technological products reliability quality control, cost effectiveness, politics and environment; effect of mechanization, consumerism; social implications of scientific advances science in the civilization of man, science and culture; society- social implications of scientific implications of scientific advances e.g population explosion, environmental pollution; social implication of technological research and advances: e.g displacement of man by machines, space travel threat of nuclear and neutron war, genetic research, energy crisis, ethics in technology; ethics, professionalism, legal aspects.

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Biochemistry – Federal University of …

American Society for Biochemistry and Molecular Biology

Grad students and postdocs: Tell us what resources you need and enter to win a $150 Amazon gift card. Take the survey. July 24: Membrane-Anchored Serine Proteases Symposium late breaking deadline. Kinases and Pseudokinases Symposium: Now accepting abstracts for oral programming consideration. JBC Thematic Minireview Series – Metals at the Host-Pathogen Interface

June 9, 2015This seventh Metals in Biology Thematic Series deals with the metal-based interactions of mammalian hosts with pathogens. Both pathogens and host have complex regulatory systems for metal homeostasis. Understanding these provides strategies for fighting pathogens, either by excluding essential metals from the microbes, by delivery of excess metals to cause toxicity, or by complexing metals in microorganisms. Intervention is possible by delivery of complexing reagents or by targeting the microbial regulatory apparatus.

Read all of the articles in this series here

May 15, 2015The study of cytoskeletal polymers has been an active area of research for more than 70 years. Yet, despite decades of pioneering work by some of the brightest scientists in biochemistry, cell biology and physiology, many central questions regarding the polymers themselves are only now starting to be answered. In this thematic series of mini-reviews, these topics are covered by some of the very same scientists who generated these recent insights, thereby providing us with an overview of the State of the Cytoskeleton in 2015.

Read all of the articles in this series here

The Howard Hughes Medical Institute (HHMI) announced 15 scientist-educators who have been named HHMI professors. Each will receive $1 million over five years “to create activities that integrate their research with student learning in ways that enhance students’ understanding of science.” Forty scientists have been named HHMI professors since the program began in 2002.

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American Society for Biochemistry and Molecular Biology

BRS Gross Anatomy (Board Review Series): 9781451193077 …

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Leslie P. Gartner PhD


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BRS Gross Anatomy (Board Review Series): 9781451193077 …

UCLA Human Genetics

The Department of Human Genetics is the youngest basic science department in the Geffen School of Medicine at UCLA. When the Department was launched just prior to the sequencing of the human genome, it was clear that the practice of genetics research would be forever changed by the infusion of massive amounts of new data. Organizing and making sense of this genomic data is one of the greatest scientific challenges ever faced by mankind. The knowledge generated will ultimately transform medicine through patient-specific treatments and prevention strategies.

The Department is dedicated to turning the mountains of raw genetic data into a detailed understanding of the molecular pathogenesis of human disease. The key to such understanding is the realization that genes not only code for specific proteins, but they also control the temporal development and maturation of every living organism through a complex web of interactions.

Housed in the new Gonda Research Center, the Department serves as a focal point for genetics research on the UCLA campus, with state of the art facilities for gene expression, sequencing, genotyping, and bioinformatics. In addition to its research mission, the Department offers many exciting training opportunities for graduate students, postdoctoral fellows, and medical residents. Our faculty and staff welcome inquiries from prospective students. We also hope that a quick look at our web pages will give you a better idea of the Department’s research and educational activities.

News Highlights

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UCLA Human Genetics

Human Genetics – University of Illinois at Chicago – UIC

University of Illinois at Chicago College of Medicine DEPARTMENT of MOLECULAR GENETICS INTRODUCTION Organization of the Course Required and Optional Texts General Introduction MENDELIAN INHERITANCE General Background categories of genetic diseases population frequencies modes of inheritance Mitosis and Meiosis Meiosis and Gametogenesis Pedigree Construction Modes of Inheritance autosomal dominant inheritance heterozygous affected phenotype hallmarks Punnett square pedigree variable expressivity late onset high recurrent mutation rate incomplete penetrance autosomal recessive inheritance introduction hallmarks carrier probabilities in a pedigree effects of consanguinity X-linked dominant inheritance hallmarks Punnett square pedigree lethality in males X-linked recessive inheritance pedigree Punnett square hallmarks Bayesian probability new mutations in genetic lethals sex limited inheritance mitochondrial inheritance Imprinting CHROMOSOMAL INHERITANCE Importance Karyotype Chromosome Replication Autosomal Chromosomal Abnormalities meiotic nondisjunction mitotic nondisjunction Robertsonian translocation isochrome formation Sex Chromosome Abnormalities Lyon hypothesis Barr bodies Turner syndrome Klinefelter syndrome XYY and XXX syndromes Non-Meiotic Chromosome Abnormalities Inversions Ring Chromosomes Translocations (non-Robertsonian) Uniparental Disomy MULTIFACTORIAL INHERITANCE Importance Regression to the mean Polygenic Inheritance The Multifactorial Model Concordance Threshold Model of Disease Degree of Relationship and Genes in Common Two Threshold Diseases Severity of Disease and Recurrence Risk Multiple Affected Offspring and Recurrence Risk Consanguinity Hallmarks of Multifactorial Inheritance LINKAGE AND MAPPING Introduction X-linkage Autosomal Linkage POPULATION GENETICS Introduction Gene and Genotype Frequencies Codominant Alleles Hardy-Weinberg Equilibrium Assumptions Calculating frequencies Evidence that it applies to humans Exceptions to Hardy-Weinberg Assumptions Effect of recurrent mutation Effect of selection against the recessive phenotype Balance between selection and recurrent mutation Balanced polymorphism Non-random mating Small populations X-linked loci

uizzes on the five major topics listed above are available on-line at our secure Mallard site. Click here and the UIC WWW Identification Service will ask for your netid and then your password (these are the same as those you use for email.)

Once the Mallard page loads you can access the quizzes by clicking on the Lessons Page link (also the third icon from the top of the navigation bar) or the Current Lesson link (also the fourth icon from the top of the navigation bar).

Contact Dr. Robert Tissot with questions about the content of these pages.

Contact Dr. Elliot Kaufman, Course Director with questions about the functionality of these pages.

Go to Department of Molecular Genetics homepage.

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Human Genetics – University of Illinois at Chicago – UIC

Cellular and Molecular Medicine, Department and PhD …

Welcome to the Department of Cellular and Molecular Medicine at the University of Arizona. Department faculty pursue excellence through teaching in the medical curriculum and graduate and undergraduate courses, through the pursuit of leading edge biomedical research, and through service to the University, local community and the nation.

Department faculty run active research programs in modern molecular and cellular biology which include areas of developmental biology, neuroscience, parasitology, immunology, cancer biology, and cellular structure and function. Our PhD graduate program attracts outstanding students from all parts of the US and the world. Graduate students from the interdisciplinary programs of Cancer Biology, Genetics, Molecular & Cellular Biology and Biochemistry, Neuroscience, and Physiological Sciences also receive training in CMM faculty laboratories.

The Department has an extensive seminar and student research seminar program with guest speakers from around the country and abroad. The Department provides a stimulating academic environment and we welcome you to explore the scope of these activities as you navigate through this website.

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Medical School

What would happen if both doctors and patients knew how much healthcare costs?

There has been a movement toward price transparency and consumerism in healthcare over the last decade or so. If people can get an accurate cost prior to receiving healthcare, the assumption is theyll use healthcare more intelligently and cost-effectively. If they only knew that getting their hip replaced at a specialized hip replacement center was $54,000 versus $103,000 at a large academic hospital, they would choose the most bang for their buck.

But lets analyze the actual process of healthcare delivery and see how the knowledge of cost applies to something as common as headaches from sinus pressure thats been plaguing you for the last month.

So now its Monday. Your head hurts and youre afraid theres something really wrong. If youre not that savvy with how to use the healthcare system, you might just make an appointment with your primary care doctor or go to an urgent care center. But those places dont really have the deep expertise nor the time to truly evaluate your sinuses, so youll be referred to a specialist. If youre super savvy, you know that you need an Ear/Nose/Throat doctor. Then, you need one close to your home or work who also takes your insurance. So whats the typical next step?

Great, one of your Facebook friends said she had a sinus problem and recommended this ENT, Dr. Blewitt, who happens to be relatively close to your work. He had an ok personality but really fixed her up about 3 years ago. You google this doctor and see that his Yelp rating is 4 stars with many one star and five star reviews. Seems ok. You give his office a call and ask if they take your insurance. They do! And you try to make an appointment and are told its going to be 3 weeks from now. This guy must be good, so you make the appointment. Hes relatively close. Hes recommended by a friend and the internet doesnt seem to hate him. He takes your insurance. Hes associated with the academic medical center with the best reputation in your area. And, hes busy and in-demand.

But what if you knew that it would cost you a baseline $300 for an office-visit with him but only $260 with a visit from another doctor you found via your insurance companys website who was a few more miles from your home, did not come with a recommendation from a friend, had a Yelp rating of 3.5 peppered with interviews like he was fine, and graduated from residency last year.

This $300 only includes the actual visit fee. It doesnt include any tests Dr. Blewitt may do in the office or orders he might make. Depending on what happens in the office, you could walk away with a simple $300 fee or the opposite could happen. Because of your unique situation and your story, Dr. Blewitt is concerned and wants to throw a full battery of tests at you to take a really good look at your sinuses. He whips out his endoscope and sticks it up your nose to look around (this is a diagnostic procedure and he later bills you $505 for it). He cant get a perfect look, so he says I need to order a CT scan of your sinuses to really understand whats going on up there. In your mind, Dr. Blewitt has a great personality, really seems to know what hes doing, and hes being very complete, covering all bases. Hes truly gained your trust.

He pulls out his pen. He orders you a CT scan of your sinuses. You dont know this, but this is whats happening here: Dr. Blewitt always refers CT scans to the in-house radiology group because thats what hes always done, he trusts their results and their state-of-the-art scanners, he knows he can give the radiologist a call on his mobile to ask any questions about the findings, and he knows they will turn this test around in no time. Plus, the radiologist is his golfing buddy every single summer Saturday morning. He also has the paper requisition forms pre-printed in his office that he fills out and faxes over to his favorite radiology group. He orders radiology tests in the same way, every day, 5 to 10 times a day. Youve already decided to trust Dr. Blewitt, so you assume hes acting in your best interest. The problem here is Dr. Blewitt has no idea how much youll be charged for the CT scan. And, frankly, hes too busy to care. At the point of his decision-making, he has absolutely no idea about how much his orders cost his patients. For him to find out how much youll be charged, hed have to personally call the insurance company for you. The insurance company could tell him how much they typically reimburse for that test. But they couldnt tell Dr. Blewitt how much the radiology group actually bills them because the test hasnt been done. And youre only on the hook for the difference between what the radiology group bills and what your insurance company pays. He doesnt have time to do that for every single one of his patients. And hes simply doing whats medically indicated for you because hes trying to do the best thing for you and hes also trying to cover his butt and do the things that will protect him in court should there be some sort of bad outcome for you. Price is honestly not even on his radar because its not his problem. And he doesnt think it should be his problem. Its too complicated and his job is to do whats medically right for you. Cost be damned. But the radiology group gets you in for the test right away (another reason why Dr. Blewitt loves them!) and bills your insurance company $935. Your insurance company only pays $300 for the test, so it costs you $635.

But, the private radiology group a few blocks away from the hospital offers the same service, the same quality equipment and only bills your insurance company $400 for the CT scan, leaving you to hypothetically pay $100 for the CT scan.

What if you had access to all of this price information? How would this change the process of healthcare delivery and the behaviors of all the players?

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Medical School

What is genetic engineering? – Definition from

Genetic engineering is the deliberate, controlled manipulation of the genes in an organism with the intent of making that organism better in some way. This is usually done independently of the natural reproductive process. The result is a so-called genetically modified organism (GMO). To date, most of the effort in genetic engineering has been focused on agriculture.

Proponents of genetic engineering claim that it has numerous benefits, including the production of food-bearing plants that are resistant to extreme weather and adverse climates, insect infestations, disease, molds, and fungi. In addition, it may be possible to reduce the amount of plowing necessary in the farming process, thereby saving energy and minimizing soil erosion. A major motivation is the hope of producing abundant food at low cost to reduce world hunger, both directly (by feeding GMOs to human beings) and indirectly (by feeding GMOs to livestock and fish, which can in turn be fed to humans).

Genetic engineering carries potential dangers, such as the creation of new allergens and toxins, the evolution of new weeds and other noxious vegetation, harm to wildlife, and the creation of environments favorable to the proliferation of molds and fungi (ironically, in light of the purported advantage in that respect). Some scientists have expressed concern that new disease organisms and increased antibiotic resistance could result from the use of GMOs in the food chain.

The darkest aspect of genetic engineering is the possibility that a government or institution might undertake to enhance human beings by means of genetic engineering. Some see the possibility of using this technology to create biological weapons.

Genetic engineering is also known as genetic modification.

This was last updated in May 2007

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Challenges in Gene Therapy – Learn Genetics


Gene Therapy

Challenges in Gene Therapy?

Gene therapy is not a new field; it has been evolving for decades. Despite the best efforts of researchers around the world, however, gene therapy has seen only limited success. Why?

Gene therapy poses one of the greatest technical challenges in modern medicine. It is very hard to introduce new genes into cells of the body and keep them working. And there are financial concerns: Can a company profit from developing a gene therapy to treat a rare disorder? If not, who will develop and pay for these life-saving treatments?

Let’s look at some of the main challenges in gene therapy.

For some disorders, gene therapy will work only if we can deliver a normal gene to a large number of cellssay several millionin a tissue. And they have to the correct cells, in the correct tissue. Once the gene reaches its destination, it must be activated, or turned on, to make the protein it encodes. And once it’s turned on, it must remain on; cells have a habit of shutting down genes that are too active or exhibiting other unusual behaviors.

Introducing changes into the wrong cells Targeting a gene to the correct cells is crucial to the success of any gene therapy treatment. Just as important, though, is making sure that the gene is not incorporated into the wrong cells. Delivering a gene to the wrong tissue would be inefficient, and it could cause health problems for the patient.

For example, improper targeting could incorporate the therapeutic gene into a patient’s germline, or reproductive cells, which ultimately produce sperm and eggs. Should this happen, the patient would pass the introduced gene to his or her children. The consequences would vary, depending on the gene.

Our immune systems are very good at fighting off intruders such as bacteria and viruses. Gene-delivery vectors must be able to avoid the body’s natural surveillance system. An unwelcome immune response could cause serious illness or even death.

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Challenges in Gene Therapy – Learn Genetics

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