World’s first GM babies born

World’s first GM babies born


The world’s first genetically modified humans have been created, it was revealed last night, 27th June 2012.

The disclosure that 30 healthy babies were born after a series of experiments in the United States provoked another furious debate about ethics.

So far, two of the babies have been tested and have been found to contain genes from three ‘parents’.

Fifteen of the children were born in the past three years as a result of one experimental programme at the Institute for Reproductive Medicine and Science of St Barnabas in New Jersey.

The babies were born to women who had problems conceiving. Extra genes from a female donor were inserted into their eggs before they were fertilised in an attempt to enable them to conceive.

Genetic fingerprint tests on two one-year- old children confirm that they have inherited DNA from three adults –two women and one man.

The fact that the children have inherited the extra genes and incorporated them into their ‘germline’ means that they will, in turn, be able to pass them on to their own offspring.

Altering the human germline – in effect tinkering with the very make-up of our species – is a technique shunned by the vast majority of the world’s scientists.

Geneticists fear that one day this method could be used to create new races of humans with extra, desired characteristics such as strength or high intelligence.

Writing in the journal Human Reproduction, the researchers, led by fertility pioneer Professor Jacques Cohen, say that this ‘is the first case of human germline genetic modification resulting in normal healthy children’.

Some experts severely criticised the experiments. Lord Winston, of the Hammersmith Hospital in West London, told the BBC yesterday: ‘Regarding the treat-ment of the infertile, there is no evidence that this technique is worth doing . . . I am very surprised that it was even carried out at this stage. It would certainly not be allowed in Britain.’

John Smeaton, national director of the Society for the Protection of Unborn Children, said: ‘One has tremendous sympathy for couples who suffer infertility problems. But this seems to be a further illustration of the fact that the whole process of in vitro fertilisation as a means of conceiving babies leads to babies being regarded as objects on a production line.

‘It is a further and very worrying step down the wrong road for humanity.’ Professor Cohen and his colleagues diagnosed that the women were infertile because they had defects in tiny structures in their egg cells, called mitochondria.

They took eggs from donors and, using a fine needle, sucked some of the internal material – containing ‘healthy’ mitochondria – and injected it into eggs from the women wanting to conceive.

Because mitochondria contain genes, the babies resulting from the treatment have inherited DNA from both women. These genes can now be passed down the germline along the maternal line.

A spokesman for the Human Fertilisation and Embryology Authority (HFEA), which regulates ‘assisted reproduction’ technology in Britain, said that it would not license the technique here because it involved altering the germline.

Jacques Cohen is regarded as a brilliant but controversial scientist who has pushed the boundaries of assisted reproduction technologies.

He developed a technique which allows infertile men to have their own children, by injecting sperm DNA straight into the egg in the lab.

Prior to this, only infertile women were able to conceive using IVF. Last year, Professor Cohen said that his expertise would allow him to clone children –a prospect treated with horror by the mainstream scientific community.

‘It would be an afternoon’s work for one of my students,’ he said, adding that he had been approached by ‘at least three’ individuals wishing to create a cloned child, but had turned down their requests.





Ethical Issues and Other Problems of Gene Therapy

‘There is always something good in a bad person and something bad in a good person’ as shown from the yin-yang symbol.

Thus there are also quite a number of ethical issues about gene therapy which raises many questions and discussions on how it should be used. This includes:

  1. How can “good” and “bad” uses of gene therapy be distinguished?
  2. Who decides which traits are normal and which constitute a disability or disorder?
  3. Will the high costs of gene therapy make it available only to the wealthy?                                                            
  4. Could the widespread use of gene therapy make society less accepting of people who are different?
  5. Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability (might use eugenics)?
  6. Is religion a factor? If yes, then somatic cell therapy should be applied and the following generations would also be able to make their own decisions.
  7. Regulations might be hard to control, even with laws, there
    is the possibility of gene therapy being available on the black market. It
    could be used for any genetically linked trait such as personality, or physical enhancement. These concerns will have to be dealt with
    in time.
  8. Invasion of privacy. Insurance companies could make it mandatory to have genetic screening because of their policies. This could cause discrimination to families with genetic diseases. Or potential employers could question a person’s genetic background before passing them up for a promotion or job.
  9. Even if gene therapy have been tested many times before both on humans and mice, it might react differently with various people with a different set of genetics and thus, there might be a chance of rejection of the inserted gene or a bad reaction and worsen the illness or condition.
  10. Gene Therapy is short-lived as it has to be ensured that the therapeutic DNA into the target cells must remain functional and the target cells must be long-lived and stable. Thus in certain cases, the patient would have to undergo many roundsof gene therapy.
  11. Multigene disorders e.g. high blood pressure, diabetes, arthritis caused by combined effects of variations in many genes are very difficult to treat.
  12. Viral Vectors might cause some unwanted side effects by having the ability to cause diseases again, and might also lead to toxicity, immune and inflammatory responses.
  13. Immune responses reduce the effectiveness of Gene Therapy as they would fight against unwanted invaders. This would prevent repetition of gene theraphy die to the immune system’s enhanced response to invaders.
  14. If DNA is integrated in the wrong place in the genome, it might induce a tumor. One  X-linked severe combined immunodeficiency (X-SCID) patient died of leukemia following gene therapy treatment in 2003.

Also, current gene therapy research has focused on treating individuals by targeting the therapy to body cells such as bone marrow or blood cells. This type of gene therapy cannot be passed on to a person’s children. Gene therapy could be targeted to egg and sperm cells (germ cells), of which the inserted gene would be passed on to future generations. This approach is known as germline gene therapy.

But, the idea of germline gene therapy is controversial. While it could spare future generations in a family from having a particular genetic disorder, it might affect the development of a fetus in unexpected ways or have long-term side effects that are not yet known. Because people who would be affected by germline gene therapy are not yet born, they can’t choose whether to have the treatment. Because of these ethical concerns, the U.S. Government does not allow federal funds to be used for research on germline gene therapy in people. Several teenagers have undergone clinical trials of gene therapy and died or developed leukemia too.

But from the recent news, European regulators have recommended approval of the Western world’s first gene therapy drug. (3 recent news articles about this in 5.)In China, Shenzhen SiBiono GeneTech won approval for a gene therapy drug for head and neck cancer in 2003 but no products have been approved until now in Europe or the United States.



European regulators back First Gene Therapy Drug

Updated 11:05 AM Jul 22, 2012
        LONDON – European regulators have recommended approval of the Western world’s first gene therapy drug – after rejecting it on three previous occasions – in a significant advance for the novel medical technology.
More than 20 years since the first experiments with the ground-breaking method for fixing faulty genes, scientists and drug companies are still struggling to apply gene therapy in practice.
Friday’s decision by the European Medicines Agency (EMA) is a win for the drug’s maker, the small Dutch biotech company uniQure, and a potential lifeline for patients with the ultra rare genetic disorder lipoprotein lipase deficiency (LPLD).
It comes too late, however, for investors in the previous listed firm Amsterdam Molecular Therapeutics (AMT).
After the earlier rebuffs for its Glybera medicine, AMT was taken private by newly created uniQure in April because it could no longer fund itself in the public markets.
Patients with LPLD are unable to handle fat particles in their blood plasma and are afraid of eating a normal meal because it can lead to acute inflammation of the pancreas.
The disorder – estimated to affect no more than one or two people per million – can cause acute pancreatitis and death.
Winning approval for Glybera proved particularly challenging because the company was only able to test it on 27 patients in clinical trials, due to the rarity of the condition.
That thin evidence base made the European agency reluctant to approve the drug initially.
But the London-based watchdog said it now accepted there was sufficient benefit to justify a green light for the worst-affected patients, on condition that those receiving the one-off therapy continued to be followed.
“This approval unlocks the potential of gene therapy because it is a first at either the EMA or FDA for gene therapy,” uniQure’s chief executive Jorn Aldag said.
“People have been sceptical as to whether the regulators would buy into this concept, which they have now done.”
The idea of treating disease by replacing a defective gene with a working copy gained credence in 1990 with the success of the world’s first gene therapy clinical tests against a rare condition called severe combined immunodeficiency (SCID).
People with SCID – also known as “bubble boy disease” – cannot cope with infections and usually die in childhood.
The field then suffered a major setback when an Arizona teenager died in a gene therapy experiment in 1999 and two French boys with SCID developed leukaemia in 2002.
In China, Shenzhen SiBiono GeneTech won approval for a gene therapy drug for head and neck cancer in 2003 but no products have been approved until now in Europe or the United States.
More recently, some large pharmaceutical companies have also been exploring gene therapy. GlaxoSmithKline, for example, signed a deal in 2010 with Italian researchers to develop a SCID therapy. REUTERS


Hi all! 🙂

This blog is our Biology Performance Task in school about Genes and its 4 sub-topics we decided on. Gene Therapy, Genetically-Modified Organisms, Plant Hybrids and Cloning. Or you could say this is simply a ICT medium on which we display information to satisfy people’s thirst for knowledge and for others to learn more about the intriguing diverse universe of science.  It’s up to you what you view it as. Also, all the posts, pictures and videos here have been credited for, on the sources which they have come from and this includes newspaper articles from various sources such as BBC News. The information is only updated as of 14 August 2012 and is for educational purposes only. Hopefully you would be able to benefit from our fantastic blog.

Thanks for viewing it! 😉


By biologypt2012 Posted in About Tagged

Ways to Insert a Gene

A gene that is inserted into a cell directly usually does not function. Thus, a carrier molecule called a vector can be used to deliver the therapeutic gene to the patient’s target cells. Currently, the most common vector is a virus 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 would be infected with the viral 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.

These are some of the different types of viruses used as gene therapy vectors:

  • Retroviruses– A class of viruses that can create double-stranded DNA copies of their RNA genomes. These copies of its genome can be integrated into the chromosomes of host cells. Human immunodeficiency virus (HIV) is a retrovirus.
  • Adenoviruses– A class of viruses with double-stranded DNA genomes that cause respiratory, intestinal, and eye infections in humans. The virus that causes the common cold is an adenovirus.
  • Adeno-associated viruses– A class of small, single-stranded DNA viruses that can insert their genetic material at a specific site on chromosome 19.
  • Herpes simplex viruses – A class of double-stranded DNA viruses that infect a particular cell type, neurons. Herpes simplex virus type 1 is a common human pathogen that causes cold sores.

Besides virus-mediated gene-delivery systems, there are several nonviral options for gene delivery:

  • The simplest method is the direct introduction of therapeutic DNA into target cells. This approach is limited in its application because it can be used only with certain tissues and requires large amounts of DNA.
  • Creation of an artificial lipid sphere with an aqueous core. This liposome, which carries the therapeutic DNA, is capable of passing the DNA through the target cell’s membrane.
  • By chemically linking the DNA to a molecule that will bind to special cell receptors, the therapeutic DNA are engulfed by the cell membrane and passed into the interior of the target cell. This delivery system tends to be less effective than other options.
  • Researchers also are experimenting with the introduction of a 47th (artificial human) chromosome to target cells. This chromosome would exist autonomously alongside the standard 46  without affecting their functions or causing any mutations. It would be a large vector capable of carrying substantial amounts of genetic code, and scientists anticipate that, because of its  autonomy, the body’s immune systems would not attack it. A problem with this potential method is the difficulty in delivering such a large molecule to the nucleus of a target cell.

The researchers must also ensure that the genes are fully controlled by the body after being inserted.

A new gene is injected into an adenovirus vector, which is used to introduce the modified DNA into a human cell. If the treatment is successful, the new gene will make a functional protein.

A new gene is injected into an adenovirus vector, which is used to introduce the modified DNA into a human cell. If the treatment is successful, the new gene will make a functional protein.


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Gene Therapy Against Nicotine May Someday Help Smokers Quit

Bloomberg News

Gene Therapy Against Nicotine May Someday Help Smokers Quit

By  Elizabeth Lopatto on June 27, 2012

An experimental vaccine against nicotine, delivered using gene therapy, prevents the substance from reaching the brain and may make quitting easier for smokers, a study using mice indicates.

A single dose of vaccine allowed the liver to produce antibodies that stopped most of the nicotine from getting to the brain, according to a study in the journal Science Translational Medicine. The concentration of nicotine in the brains of treated mice was just 15 percent of that in untreated ones.

Of the more than 4,000 chemicals in cigarette smoke, it is nicotine that leads to addiction, the researchers wrote. Keeping the substance away from the brain might stymie nicotine’s addictive power by preventing smokers from enjoying their cigarettes, giving them no incentive to relapse, said Ronald Crystal, one of the study’s researchers.

“This looks really terrific if you’re a mouse, but the caveat is that they aren’t small humans,” said Crystal, the chairman of genetic medicine at Weill Cornell Medical College in New York, in a telephone interview.

The gene therapy delivers the vaccine to the liver using a virus engineered not to be harmful. The gene sequence for the antibodies is inserted into liver cells, which then begin to create antibodies to nicotine.

“The antibody is floating around like Pac-Man in the blood,” Crystal said. “If you give the nicotine and the anti-nicotine gobbles it up, it doesn’t reach the brain.”

Relapse Rate

The idea of vaccines against nicotine has emerged before, in the form of injections used to trigger an immune response. Those methods proved ineffective, according to the researchers. They turned to gene therapy to trigger production of antibodies.

About 20 percent of U.S. adults are smokers, and most relapse shortly after quitting.

“We don’t have very effective therapies,” Crystal said.“The problem is even with the drugs we have now, 70 percent of people go back to smoking within 6 months of trying to quit.”

The virus vector has been previously given to children with a different disease and appears to be safe, he said.

His group plans to continue studying the vaccine in rats and non-human primates, and has talked to pharmaceutical companies about testing, he said. The gene-therapy vaccine method may work in other addictions as well, Crystal said.

The study was funded by the National Institutes of Health, the National Foundation for Cancer Research and the Malcolm Hewitt Wiener Foundation.


Colon Cancer Genomic Mapping May Point Toward Therapies

Bloomberg News

Colon Cancer Genomic Mapping May Point Toward Therapies

By  Ryan Flinn on July 19, 2012

Companies Mentioned

  • BMY

    Bristol-Myers Squibb Co

    • $35.42 USD
    • -0.73
    • -2.06%
  • PFE

    Pfizer Inc

    • $23.7 USD
    • -0.10
    • -0.4%

The largest study of colon cancer tumors to date found new mutations that may cause malignancy in various organs, bolstering research that links the disease more to specific genetic changes than its location in the body.

Researchers sequenced the genomes of 276 tumors, then compared the results with normal cells from the same patients. They found some mutations previously linked to other types of cancer, and new variants that may be targeted by therapies currently in development, said Raju Kucherlapati, a professor at Harvard Medical School and an author of the study.

“It turns out there were a number of novel and very interesting things that tell us about the biology of the tumors, and also point to directions in which therapies can go,”Kucherlapati said in a telephone interview. “Some of the same genes are modified or mutated in many different cancers.”

The research published yesterday by the journal Nature is among recent studies showing how medicines designed to target gene mutations in one cancer may be applied to other malignancies with the same abnormality. These results have spurred drugmakers such as Bristol-Myers Squibb Co. (BMY) (BMY), Pfizer Inc. (PFE) (PFE)and Roche Holding AG (ROG), the world’s biggest maker of cancer medicines, to focus their cancer research on so-called targeted therapies.

About 5 percent of patients in the study had tumors with mutations to the ERBB2 gene, which also has been found in breast and gastric cancers. Herceptin, a $6 billion breast cancer drug from Basel, Switzerland-based Roche, targets that mutation, Kucherlapati said. Herceptin was one of the first cancer medicines aimed at patients whose tumors have this genetic abnormality.

Gene Target

Another potential target for drugmakers is a mutation that caused 22 percent of patients in the study to have high levels or over-expression of the insulin-like growth factor-2 gene. Companies currently working on inhibitors for this gene, include London-based AstraZeneca’s Medimmune unit, which is testing its therapy, MEDI 573, in the first of three trials typically required for regulatory approval.

Other drugmakers are expanding testing of their cancer therapies that focus on specific gene variants. In May, New York-based Pfizer found that Xalkori, its drug that targets adult lung cancer caused by a gene defect, also eradicates the malignancy in some children with rare tumors of the nerves, blood and soft tissue. Bristol-Myers, based in New York, also announced in May it would expand testing of its leukemia drug Sprycel in patients with lung cancer because it appeared to help one patient with a particular mutation.

The data released yesterday are part of a broader project by the National Institutes of Health called the Cancer Genome Atlas, which is analyzing tumors and blood samples from 20 types of cancer.

One of the project’s earlier studies of ovarian cancer patients with a genetic mutation best known for its ties to breast cancer have found they have a higher survival rate than those without the mutation.


Gene Therapy Treatment Extends Lives of Mice with Fatal Disease, MU Study Finds

Gene Therapy Treatment Extends Lives of Mice with Fatal Disease, MU Study Finds

Spinal Muscular Atrophy affects one in 6,000 children; no known cure

July 16, 2012

COLUMBIA, Mo. — A team of University of Missouri researchers has found that introducing a missing gene into the central nervous system could help extend the lives of patients with Spinal Muscular Atrophy (SMA) – the leading genetic cause of infantile death in the world.

SMA is a rare genetic disease that is inherited by one in 6,000 children who often die young because there is no cure. Children who inherit SMA are missing a gene that produces a protein which directs nerves in the spine to give commands to muscles.

The MU team, led by Christian Lorson, professor in the Department of Veterinary Pathobiology and the Department of Molecular Microbiology and Immunology, introduced the missing gene into mice born with SMA through two different methods: intravenously and directly into the mice’s central nervous systems. While both methods were effective in extending the lives of the mice, Lorson found that introducing the missing gene directly into the central nervous system extended the lives of the mice longer.

“Typically, mice born with SMA only live five or six days, but by introducing the missing SMN gene into the mice’s central nervous systems, we were able to extend their lives 10-25 days longer than SMA mice who go untreated,” said Lorson, who works in the MU Bond Life Sciences Center and the College of Veterinary Medicine. “While this system is still not perfect, what our study did show is that the direct administration of the missing gene into the central nervous system provides some degree of rescue and a profound extension of survival.”

There are several different types of SMA that appear in humans, depending on the age that symptoms begin to appear. Lorson believes that introducing the missing gene through the central nervous system is a way to potentially treat humans regardless of what SMA type they have.

“This is a treatment method that is very close to being a reality for human patients,” Lorson said. “Clinical trials of SMA treatment using gene therapy are likely to begin in next 12-18 months, barring any unforeseen problems.”

The study, “Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of Spinal Muscular Atrophy”, was published in Biochemical and Biophysical Research Communications. Co-authors of the study include Jacqueline Glascock and Monir Shababi from MU College of Veterinary Medicine.


Superhuman Games for Genetically Enhanced Athletes

Forget the Olympics – Here come the superhuman games for genetically enhanced athletes

  • GM athletes could get their own events
  • Scientists say they could be treated ‘like racing cars’
  • Power running, swimming and climbing could be first disciplines

PUBLISHED: 18:01 GMT, 19 July 2012 | UPDATED: 18:01 GMT, 19 July 2012

Superhuman athletes created by gene therapy and biomechanical engineering will one day be competing at the Olympics – but will need their own events, predict scientists.

Performance-enhancing technologies will advance to a point where they will not only extend human limits – but demand a events all of their own, similar to the Formula One version of car racing.

Professor Hugh Herr of the prestigious Massachusetts Institute of Technology said: ‘For each one there will be a new sport – power running and power swimming and power climbing.

‘Just like the invention of the bicycle led to the sport of cycling. What we’ll see is the emergence of all kinds of new sports.’

Mechanical prosthetics will become much more proficient than the ‘cheetah-style’ legs used by amputees including Oscar Pistorius from South Africa.

oscarThe first superhuman athlete? Oscar Pistorius trains at the track in South Africa.

The Paralympic gold medallist has now been approved to run in the London 2012 Olympics even though his prosthetics lack the stiffness of a human ankle and can’t generate the same forces.

Prof Herr’s lab at the Massachusetts Institute of Technology is currently working on a bionic running leg.

The biomechanical engineer told Nature: ‘Stepping decades into the future, I think one day the field will produce a bionic limb that’s so sophisticated it truly emulates biological limb function.

‘That technology will be the Olympic sanctioned limb. Without any such human-like constraints the Paralympics limb will become the basis of this human-machine sport like race car driving.’

If all restrictions were lifted science could push human performance to new extremes – although drugs are not the only answer, say researchers.

Some say enhancers have become so prevalent the only solution is to allow anything – as long as it is safe.

oscarOscar Pistorius, far left, is helping Team GB hopeful Jonnie Peacock in his quest to qualify for London.

But surgery and, ultimately, technological augmentations could also help athletes towards the podium.

Baseball pitchers who have undergone surgery to replace a damaged elbow ligament with tissue from a hamstring or forearm tendon claim that they can throw harder after the two-year rehabilitation process.

Dr Andy Miah, a bioethecist at West of Scotland University in Ayr, sees potential in more imaginative surgical enhancement.

He said: ‘Consider using skin grafts to increase webbing between fingers and toes to improve swimming capacity.

‘These kinds of tweaks to our biology are likely ways people would try to gain an edge over others.’

Advances in gene therapy could make it possible for any athlete to enhance their DNA.

In experiments aimed at treating muscular dystrophy in the elderly researchers at Pennsylvania University introduced a gene to cause over-expression of the growth hormone IGF1 in mice.

The treatment boosted muscle strength of young adult mice by 14 percent – earning the rodents the nickname ‘mighty mice.’

Another frontier is nanotechnology with researchers already experimenting with blood supplements based on oxygen-carrying particles for use in emergency situations.

Dr Miah said: ‘There is a lot of discussion about the possibility of biologically infused nanodevices that could perpetually maintain certain thresholds of performance.’

Future Olympic Games could allow handicaps and gene therapy for people who naturally lack genes linked to athleticism as evidence grows that world-class athletes carry a minimum set of particular ‘performance-enhancing’ genes.

Science authors Dr Juan Enriquez and Dr Steve Gullans, of Excel Venture Management, Boston, said: ‘Genetically enhanced Olympics are coming.’