Archive for the ‘Gene Therapy Research’ Category

Thursday 16 January 2014 11.33

Pioneering gene therapy has restored vision to two men with a rare inherited eye disease who were told to expect to go blind.

Scientists hope early intervention with the surgical treatment will halt progression of the devastating disorder, choroideremia, before patients are robbed of their sight.

It is the first time gene therapy has successfully been applied to the light-sensitive photoreceptors of the retina, the digital camera at the back of the eye.

Preliminary results from the first six patients taking part in a Phase One trial surprised and delighted the Oxford University team.

Although the trial was only designed to test safety and dosages, two men with relatively advanced disease experienced dramatic improvements to their eyesight.

The researchers are now planning a larger Phase II trial that will focus on the therapy's effectiveness.

Professor Robert MacLaren, who led the gene therapy operations at Oxford Eye Hospital, said: "We're absolutely delighted with the results so far.

"It is still too early to know if the gene therapy treatment will last indefinitely, but we can say that the vision improvements have been maintained for as long as we have been following up the patients, which is two years in one case.

"In truth, we did not expect to see such dramatic improvements in visual acuity and so we contacted both patients' home opticians to get current and historical data on their vision in former years, long before the gene therapy trial started.

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Gene therapy breakthrough in blindness research

By Thomas Moore, Health and Science Correspondent

Patients suffering from an inherited form of blindness have for the first time had their vision dramatically improved by gene therapy.

The first six patients to be given the experimental injections into the back of the eye were all able to see better in dim light.

And two of them were able to read more lines on an eye chart.

The patients were born with a genetic form of blindness called choroideremia, which affects 1 in 50,000 people, most of them boys, who start to lose their vision in late childhood.

But in a groundbreaking clinical trial, doctors at the Oxford Eye Hospital injected a harmless virus that had been engineered to carry a working copy of the gene that sufferers lack.

The trial was intended to confirm that the injections did not damage the delicate light-sensing cells in the retina.

But the gene therapy had an unexpected therapeutic benefit - and three more patients have now been treated with a higher dose.

Professor Robert MacLaren of Oxford University, who led the trial, said: "In truth we did not expect to see such dramatic improvements in visual acuity.

"It is still too early to know if the gene therapy treatment will last indefinitely, but we can say that the vision improvements have been maintained as long as we have been following up the patients, which is two years in one case."

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Gene Therapy Breakthrough Could 'Cure' Blindness

Goodie Mob - Cell Therapy (O.N.P. Instrumental) (1995) [HQ]
Producer: Organized Noize Productions Released: 1995 : LaFace Records Official Facebook Page: https://www.facebook.com/BeatJunKie82 --- Original Copyright O...

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Goodie Mob - Cell Therapy (O.N.P. Instrumental) (1995) [HQ] - Video

By MARIA CHENG AP Medical Writer

LONDON (AP) - A small, preliminary study using gene therapy to treat a rare form of blindness is promising and could trigger similar efforts for other causes of vision loss, British doctors say.

They studied just six patients. Of those, two have had dramatic improvements in their vision and none has reported any serious side effects. The study was only designed to test the treatment's safety, not its effectiveness.

Gene therapy -inserting copies of a normal gene into a patient who has a faulty or missing gene - has previously been tried for other rare types of blindness with limited success.

All of the patients in the new trial were men with choroidermia, an inherited and untreatable form of progressive blindness. The disease is the result of a protein deficiency that kills eye cells that detect light.

"We're trying to rescue cells that would otherwise have died," said Dr. Robert MacLaren of the University of Oxford, who led the research. He and colleagues injected a harmless virus carrying the missing protein into the thinnest part of the patients' retinas. The study was paid for by the Wellcome Trust and Britain's Department of Health, and was published online Thursday in the journal, Lancet.

Dr. Ian MacDonald, chairman of ophthalmology and visual sciences at the University of Alberta in Canada, who was not part of the study but is planning a similar trial, called the new work "very promising because there is really no other way to deliver this protein."

Since the paper only had data up to six months after the patients' surgeries, MacDonald said it was unclear if the treatment would last forever or if the men's eyesight would continue to worsen.

Dr. Jean Bennett of the University of Pennsylvania, who conducted earlier work for a different eye disease, called the new study results exciting and said the same approach might be useful for other causes of blindness, including macular degeneration.

But she also said scientists should be cautious in trying future gene therapy treatments. "We can do as much work as we can in the laboratory and try to sort out all the variables, but there are always surprises," she said.

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Early gene therapy trial for blindness promising - Quincy Herald-Whig | Illinois & Missouri News, Sports

Genetics How I am is How I am- E.V
Have you ever wondered why your ears are so big? Or why your nose is so long? Genetics is the answer, and when you find out how it all works, you can finally blame your dad for his ears.

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Genetics How I am is How I am- E.V - Video

Scrog; Bubba Kush genetics: plants under scrog for 17 days
Scrog @ 17 days.

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Genetics- Intro to Genetics

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Scrog Bubba Kush genetics; plants 14 days old right before the scrog
Scrog Bubba Kush.

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Table One: Genetics and Heredity
For Mr. Kennedy #39;s class. Made by: Courtney Wright, Lindsey Blizzard, Laken Hatcher, D #39;Andre Johnson, and Drake Lanier.

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Table One: Genetics and Heredity - Video

click image to zoom The future of dairy cattle breeding will be among the key topics as eight breeders from coast to coast voice their perspectives at the workshop Advancing Dairy Cattle Genetics: Genomics and Beyond in Tempe, Ariz., from February 17 to 19.

For more workshop details, go to http://www.ans.iastate.edu/events/dairygenomics/ to complete your registration.

In the meantime, here is more insight on the two breeder panels that will hone in on the future of cattle breeding:

Traits of the Future:

Dana Allen Tully, Gar-Lin Dairy Farms, Eyota, Minn. Dana manages an 1,855 cow dairy of which two-thirds of the cows are Holsteins and one-third are crossbred. A Ph.D. nutritionist by training, Dana places a strong focus on health traits such somatic cell score, daughter pregnancy rate, and productive life when making mating decisions. She also is a member of the Innovation Center for U.S. Dairys Cow of the Future Committee.

Alan Andersen, Seagull-Bay, American Falls, Idaho In recent years, the Andersens 600 cow Holstein herd has routinely placed bulls in the top 100 of the DNA or genomic-tested list. The most recognizable bull in that group is Seagull-Bay Supersire. Alan and his son, Greg, focus on high production and fitness traits. The family also has another 1,500 cow dairy at Declo, Idaho, managed by Alan's son Ben. This herd has utilized a 3-way crossbreeding system since 2005.

Alan Chittenden, Dutch Hollow Farm, Schodack Landing, N.Y. These Jersey breeders have been leaders in developing polled genetics that also rank high among all animals in the breed. Today, the herd consists of 600 Jersey cows. Their incredible track record of breeding top-notch cattle earned them the prestigious Master Breeder award from the American Jersey Cattle Association in 2012.

Jonathan Lamb, Oakfield Corners Dairy, Oakfield, N.Y. Lamb regularly uses embryo transplant and in-vitro fertilization on his 6,000 cow dairy to improve the genetics of his herds. Respected among his peers, Lamb serves as chairman of the genetic advancement committee for Holstein Association USA. Lamb was also named the Holstein USA Associations Distinguished Young Breeders in 2012.

On-farm Genomic Applications:

John Andersen, Double A Dairy, Jerome, Idaho Perhaps one of the largest users of genomic tests on a commercial scale, Andersen and his team routinely run genomic tests to sort out high-end and low-end genetic animals at their 13,000 cow dairy. Additionally, John stays involved of high-end genetics through breeding cattle under the Triple Crown Genetics prefix.

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Rethinking dairy genetics: Eight breeders to share needs in cows

A small, preliminary study using gene therapy to treat a rare form of blindness is promising and could trigger similar efforts for other causes of vision loss, British doctors say.

They studied just six patients. Of those, two have had dramatic improvements in their vision and none has reported any serious side effects. The study was only designed to test the treatment's safety, not its effectiveness.

Gene therapy inserting copies of a normal gene into a patient who has a faulty or missing gene has previously been tried for other rare types of blindness with limited success.

All of the patients in the new trial were men with choroidermia, an inherited and untreatable form of progressive blindness. The disease is the result of a protein deficiency that kills eye cells that detect light.

"We're trying to rescue cells that would otherwise have died," said Dr. Robert MacLaren of the University of Oxford, who led the research. He and colleagues injected a harmless virus carrying the missing protein into the thinnest part of the patients' retinas. The study was paid for by the Wellcome Trust and Britain's Department of Health, and was published online Thursday in the journal, Lancet.

Dr. Ian MacDonald, chairman of ophthalmology and visual sciences at the University of Alberta in Canada, who was not part of the study but is planning a similar trial, called the new work "very promising because there is really no other way to deliver this protein."

Since the paper only had data up to six months after the patients' surgeries, MacDonald said it was unclear if the treatment would last forever or if the men's eyesight would continue to worsen.

Dr. Jean Bennett of the University of Pennsylvania, who conducted earlier work for a different eye disease, called the new study results exciting and said the same approach might be useful for other causes of blindness, including macular degeneration.

But she also said scientists should be cautious in trying future gene therapy treatments. "We can do as much work as we can in the laboratory and try to sort out all the variables, but there are always surprises," she said.

Toby Stroh, 56, had the gene therapy surgery nearly two years ago and credits it with allowing him to keep reading and playing tennis.

Original post:
Early Gene Therapy Trial for Blindness Promising

"In truth, we did not expect to see such dramatic improvements

This has huge implications for anyone with a genetic retinal disease such as age-related macular degeneration or retinitis pigmentosa because it has, for the first time, shown, that gene therapy can be applied safely before the onset of vision loss.

The trial was carried out on patients suffering from choroideremia a rare inherited cause of blindness which affects around 1 in 50,000 people.

It is caused by a defective gene which fails to produce REP-1 a protein needed to keep pigment cells in the retina healthy. Without it the cells slowly stop working, switch off and die.

The first symptom of the condition is usually poor night vision which can occur in early childhood. Later, the field of vision progressively narrows to a "tunnel" until only a small central slit remains.

The purpose of our trial is to put this missing protein back into the retinal cells and prevent further degeneration, said Prof MacLaren.

We're not talking about treatment that needs to be repeated we're talking about a single one off replacement of the gene.

What was unique and exciting is we noticed visual improvements very early, which shows us that it is working.

"If we were able to treat people early, get them in their teens or late childhood, we'd be getting the virus in before their vision is lost. If the treatment works, we would be able to prevent them from going blind."

The scientists found that the protein could be replaced in the eye by inserting it into the DNA of a harmless virus which can be injected into cells beneath the retina. As the virus infects these retinal cells the missing protein is restored.

Continued here:
Gene therapy heralds cure for blindness

3 hours ago

Oxford Eye Hospital

Dr. Robert MacLaren and colleagues at Britain's Oxford Eye Hospital perform a gene therapy experiment. Patients who took part say the experimental, approach improved their vision

An experimental therapy for a blinding eye disease showed early and surprising promise when it improved the vision of patients in an early trial that was only supposed to test its safety, doctors reported Wednesday.

The experimental gene therapy not only stopped the steady degeneration of the patients vision, but appears to have reversed some of the damage. And the effects have lasted two years in one case, British researchers report in the Lancet medical journal.

Wayne Thompson of Staffordshire in Britain saw the stars for the first time in years after being treated in April.

One night in the summer, my wife called me outside as it was a particularly starry evening. As I looked up, I was amazed that I was able to see a few stars, Thompson, 43, said in a statement.

I hadnt seen stars for a long, long time, he added.

It is still too early to know if the gene therapy treatment will last indefinitely, but we can say that the vision improvements have been maintained for as long as we have been following up the patients, which is two years in one case, says Dr. Robert MacLaren of the Nuffield Laboratory of Ophthalmology at the University of Oxford, who leads the research team.

In truth, we did not expect to see such dramatic improvements in visual acuity and so we contacted both patients home opticians to get current and historical data on their vision in former years, long before the gene therapy trial started. These readings confirmed exactly what we had seen, he added in a statement.

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In surprise result,gene therapy reverses blinding eye disease

Published:Tuesday, January 14, 2014

Updated:Tuesday, January 14, 2014 18:01

A new tool that could help facilitate future stem cell therapy has recently been identified by a UVM professor and his colleagues, according to UVMs College of Medicine.

The development of this tool could potentially help more than 700,000 Americans who suffer a heart attack each year.

Because stem cells have the potential to develop into a variety of cell types in the body, they may offer a renewable source of replacement cells to treat diseases, conditions and disabilities, and even regenerate damaged tissue and organs.

However, the field of regenerative medicine has struggled to successfully graft cells from culture back into injured tissue.

UVM Associate Professor of Medicine Jeffrey Spees, Ph.D., collaborated with the Center for Gene Therapy at Tulane University. His research team recently set out to develop ways to enhance graft success.

Dr. Spees and his team focused on a type of bone marrow-derived progenitor cell or biological cell that forms stromal cells or connective tissue cells.

They found that the medium contained Connective Tissue Growth Factor (CTGF) and the hormone insulin, and together, they have a synergistic effect, Spees said to UVMs College of Medicine.

The group found that the protective ligands resulted in improved graft success, breaking the record for engraftment.

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New tool assists stem cell therapy

PUBLIC RELEASE DATE:

14-Jan-2014

Contact: Beverly Clark beverly.clark@emory.edu 404-712-8780 Emory Health Sciences

A unique study of the white-throated sparrow has identified a biological pathway connecting variation in the birds' aggression and parenting behaviors in the wild to variation in their genome.

The Proceedings of the National Academy of Sciences (PNAS) is publishing the results of the experiments, conducted by the lab of neuroscientist Donna Maney in Emory University's Department of Psychology.

The research, which comprised behavioral observations of the study subjects in the field and laboratory analyses of their gene expression in the brain, showed that variation in the expression of the estrogen receptor alpha (ER-alpha) gene strongly predicts the birds' behavior.

"We believe this is the most comprehensive study yet of how the rearrangement of a chromosome affects social behavior in a vertebrate," says Brent Horton, a post-doctoral fellow in the Maney lab and lead author of the study. "So much of the process of genetic discovery is restricted behind closed doors in a laboratory. But our study began in the woods, where we first observed the social behaviors of the actual subjects of our experiments in their natural setting. The results provide valuable insight into the mechanistic basis of aggression and parenting in all vertebrates, including humans."

Such integrated studies "are exceedingly rare," Horton adds, "because they require such a variety of resources, expertise and well-balanced collaboration."

In addition to Horton and Maney, the principal investigators included Eric Ortlund, a biochemist and an expert in the ER-alpha gene at the Emory School of Medicine; and James Thomas, a human geneticist who was formerly with Emory and now works at the National Institutes of Health. Co-authors include William Hudson, a graduate fellow in Ortland's lab; Wendy Zinzow-Kramer, a post-doc in the Maney lab; Sandra Shirk, a research associate; and Emily Young, an undergraduate student of biology at Georgia Tech.

The white-throated sparrow is considered a good model organism for the genetic basis of behavior due to a genetic event that has divided the species into two distinct forms that differ in their behavior. These two forms, the white-striped morph and the tan-striped morph, are easily distinguished by their plumage markings.

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Wild sparrow study traces social behaviors in the field to specific gene

Jan. 14, 2014 The presence of a gene variant in people with mild cognitive impairment (MCI) is associated with accelerated rates of brain atrophy, according to a new study published online in the journal Radiology.

The study focused on the gene apolipoprotein E (APOE), the most important genetic factor known in non-familial Alzheimer's disease (AD). APOE has different alleles, or gene variations, said the study's senior author, Jeffrey R. Petrella, M.D., associate professor of radiology at Duke University School of Medicine in Durham, N.C.

"We all carry two APOE alleles, and most people have at least one copy of the APOE epsilon 3 (3) variant, which is considered neutral with respect to Alzheimer's risk," Dr. Petrella said.

The less common epsilon 4 (4) allele, in contrast, is associated with a higher risk for development of AD, earlier age of onset, and faster progression in those affected, as compared with the other APOE alleles.

Dr. Petrella and colleagues recently analyzed data from the Alzheimer's Disease Neuroimaging Initiative (ADNI) involving 237 patients, mean age 79.9, with MCI, a slight but noticeable decline in cognitive ability that is tied to a higher risk of AD. The researchers used MRI to measure brain atrophy rates in these patients over a 12- to 48-month period.

The 4 carriers in the study group exhibited markedly greater atrophy rates than 3 carriers in 13 of 15 brain regions hypothesized to be key components of the cognitive networks disrupted in AD.

"The results showed atrophy in brain regions we know are affected by AD, in a population of patients who do not have AD, but are at risk for it," Dr. Petrella said. "This suggests the possibility of a genotype-specific network of related brain regions that undergo faster atrophy in MCI and potentially underlies the observed cognitive decline."

The researchers did not explore why APOE 4 might accelerate atrophy, but the affect is likely due to a combination of factors, noted Dr. Petrella.

"The protein has a broad role in the transport and normal metabolism of lipids and a protective function on behalf of brain cells, including its role in the breakdown of beta-amyloid, one of the proteins implicated in the pathophysiology of AD," he said.

With MRI playing an increasingly prominent role in MCI research, Dr. Petrella predicted that increased knowledge about the effects of APOE will improve the design and execution of future clinical trials. For instance, researchers could enrich their samples with 4 patients in MCI prevention trials to better determine potential treatment effects on brain regions vulnerable to degeneration.

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Gene variation associated with brain atrophy in mild cognitive impairment

AAP A new system bought by Sydney's Garvan Institute can map the genetic makeup of 350 people a week.

Australian medicine has taken a leap into the future with the purchase of a system that can quickly and cheaply map a person's genetic makeup.

The new system bought by Sydney's Garvan Institute can map 350 people a week at a cost of $1000 each.

This means doctors will receive quick feedback on the best way to treat cancer patients and scientists will have massive power to build an Australian genetic database.

The system differs from current genetic testing in that it maps the entire genome rather than specific gene mutations such as BRCA1 and BRCA2 that cause breast and ovarian cancer.

The first whole human genome was mapped more than a decade ago by an international team of scientists at a cost $1 billion.

Garvan is one of a few organisations in the world to buy the HiSeq X Ten Sequencing System, according to an announcement in San Diego on Tuesday (Wednesday AEDT).

"Over the next few years, we have an opportunity to learn as much about the genetics of human disease as we have learned in the history of medicine," said Garvan executive director Professor John Mattick.

"We have reached a tipping point where genome sequencing has become achievable on a broad scale."

He expected genomic sequencing to become widely available to the general public in the next few years.

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Australia takes genetic medicine leap

AAP A new system bought by Sydney's Garvan Institute can map the genetic makeup of 350 people a week.

Australian medicine has taken a leap into the future with the purchase of a system that can quickly and cheaply map a person's genetic makeup.

The new system bought by Sydney's Garvan Institute can map 350 people a week at a cost of $1000 each.

This means doctors will receive quick feedback on the best way to treat cancer patients and scientists will have massive power to build an Australian genetic database.

The system differs from current genetic testing in that it maps the entire genome rather than specific gene mutations such as BRCA1 and BRCA2 that cause breast and ovarian cancer.

The first whole human genome was mapped more than a decade ago by an international team of scientists at a cost $1 billion.

Garvan is one of a few organisations in the world to buy the HiSeq X Ten Sequencing System, according to an announcement in San Diego on Tuesday (Wednesday AEDT).

"Over the next few years, we have an opportunity to learn as much about the genetics of human disease as we have learned in the history of medicine," said Garvan executive director Professor John Mattick.

"We have reached a tipping point where genome sequencing has become achievable on a broad scale."

He expected genomic sequencing to become widely available to the general public in the next few years.

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Major leap for Aust genetic medicine

Durham, NC (PRWEB) January 14, 2014

A new study released in STEM CELLS Translational Medicine indicates that stem cells can be effective in treating a debilitating and sometimes lethal genetic disorder called brittle bone disease.

Brittle bone disease, or osteogenesis imperfecta (OI), is characterized by fragile bones causing some patients to suffer hundreds of fractures over the course of a lifetime. In addition, according to the OI Foundation, other symptoms include muscle weakness, hearing loss, fatigue, joint laxity, curved bones, scoliosis, brittle teeth and short stature. Restrictive pulmonary disease occurs in the more severe cases. Currently there is no cure.

OI can be detected prenatally by ultrasound. In the study reported on in STEM CELLS Translational Medicine, an international team of researchers treated two patients for the disease using mesenchymal stem cells (MSCs) while the infants were still in the womb, followed by stem cell boosts after they were born.

We had previously reported on the prenatal transplantation for the patient with OI type III, which is the most severe form in children who survive the neonatal period, said Cecilia Gtherstrm, Ph.D., of the Karolinska Institutet and Karolinska University Hospital, Stockholm, Sweden. She and Jerry Chan, M.D., Ph.D., of the Yong Loo Lin School of Medicine and National University of Singapore, and KK Womens and Childrens Hospital, led the study that also included colleagues from the United States, Canada, Taiwan and Australia.

The first eight years after the prenatal transplant, our patient did well and grew at an acceptable rate. However, she then began to experience multiple complications, including fractures, scoliosis and reduction in growth, so the decision was made to give her another MSC infusion. In the two years since, she has not suffered any more fractures and improved her growth.

She was even able to start dance classes, increase her participation in gymnastics at school and play modified indoor hockey, Dr. Gtherstrm added.

The second child, which was experiencing a milder form of OI, received a stem cell transfusion 31 weeks into gestation and did not suffer any new fractures for the remainder of the pregnancy or during infancy. She followed her normal growth pattern just under the third percentile in height until 13 months of age, when she stopped growing. Six months later, the doctors gave her another infusion of stem cells and she resumed growing at her previous rate.

Our findings suggest that prenatal transplantation of autologous stem cells in OI appears safe and is of likely clinical benefit and that re-transplantation with same-donor cells is feasible. However, the limited experience to date means that it is not possible to be conclusive, for which further studies are required, Dr. Chan said.

Although the findings are preliminary, this report is encouraging in suggesting that prenatal transplantation may be a safe and effective treatment for this condition, said Anthony Atala, M.D., editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.

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Stem Cells Could Prove Effective in Treating Brittle Bone Disease

Genetics of Sickle Cell Disease
Sickle cell disease is an inherited genetic disorder and is a recessive trait. This video describes how the genetic mutation causing sickle cell disease is p...

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How to analyze and solve genetics problems
Solving Genetic Problems What is a Genetic Problem? A genetic problem is a type examination question that involves both a knowledge of Mendel #39;s experiments, ...

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How to analyze and solve genetics problems - Video

Mutants Genetics Gladiators: Omega Boss Neo Paris | Medusas lvl 33 and Dracus nobilis.
en este video muestro como matar al dracus nobilis de omega aunk me sobro un poco de suerte XD ya k este si fue algo dificil MI FB: https://www.facebook.com/...

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Mutants Genetics Gladiators: Omega Boss Neo Paris | Medusas lvl 33 and Dracus nobilis. - Video

Minecraft Mod Spotlight - Advanced Genetics
Now that Tom (A.K.A.) has defeated the Ender Dragon he has now begun searching for cool mods made by the community! Get Advanced Genetics: http://www.minecra...

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Minecraft Mod Spotlight - Advanced Genetics - Video

MineCraft Modded Survival Grouer Adventure #5 Advanced genetics

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MineCraft Modded Survival Grouer Adventure #5 Advanced genetics - Video

Lets play the sims3 perfect genetics challenge pt9

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Lets play the sims3 perfect genetics challenge pt9 - Video