Stem cell and gene therapy combination could help cure genetic diseases - Cord Blood Banking

Researchers from the Salk Institute for Biological Studies have demonstrated for the first time ever that stem cell and gene therapy combination technique could work in human cells.

Previously several studies have conducted with mice but human studies were never successful. Though they did able to cure a cell not any diseased human but morning shows the day, still lot to go but if this studies really works then, if scientists could able to cure genetic problems through this combined technique the it will open up a door of limit less options.

Leader of the team Salk Professor Juan-Carlos Izpisua Belmonte and colleges, have corrected a defective gene in cells taken from patients with Fanconi anemia, a disease that can lead to bone marrow failure, leukemia and other cancers.

Researchers at first collected hair or skin cells from patients with Fanconi anemia, then the successfully corrected the gene defect and reprogram the cells into induced pluripotent stem cells cells created synthetically from skin or other cells. Finally this FA-iPS cells were indistinguishable from human embryonic stem cells and iPS cells generated from healthy donors.

They used gene therapy techniques developed in the lab of Salk Professor Inder Verma, and also collaborated with researchers at the Center of Regenerative Medicine in Barcelona, Spain, and CIEMAT in Madrid, Spain.

Keeping in mind that Fanconi anemia always help bone marrow failure as a result of the progressive decline in the numbers of functional hematopoietic stem cells , researchers tested and found that FA-iPS cells readily differentiated into hematopoietic progenitor cells primed to differentiate into healthy blood cells.

Researchers are looking forward in particularly to over come the main obstacle that is preventing the reprogrammed cells from inducing tumors.

The study has been published online on Sunday by the journal Nature, offers the first proof that the technology can work in human cells though more work remains for it to be tried in patients.

The researchers received a $6.6 million grant from California’s stem cell institute in April to continue the work. One big remaining problem is to prevent the reprogrammed cells from inducing tumors.

Source: SignOnSanDiego.com
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LONDON – Scientists at Massachusetts General Hospital in Boston and the Harvard Stem Cell Institute claim that they have isolated such embryonic stem cells from mice as are capable of differentiating into mature cardiac muscles.

This work attains significance because the cells identified by the researchers can be a powerful tool in animal models of heart disease, may pave the way for identifying the equivalent cells in humans, and one day aid human heart transplants.

While presenting the research team’s findings at a Beijing meeting, Ibrahim Domian of Massachusetts General Hospital in Boston said that they had had isolated embryonic stem cells from those mice and identified different progenitor populations that express different sets of genes.

The researcher revealed that, in culture, the cells divided for a few days before differentiating into mature cardiac muscles.

The researchers also found that cardiac myocetes, the type of heart muscle cells that are capable of coordinated contraction, derived from these progenitor cells contracted with a level of force – the defining characteristic of cardiac muscle – that was the same as neonatal cardiac myocytes.

Domian declined to discuss details, stating that the results are under review at a journal.

“This is a very interesting study,” Nature magazine quoted Ulrich Martin, a stem-cell researcher at Hannover Medical School, Germany, who was not involved with the research, as saying. (ANI)

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The 2nd embryo-deprived of the microRNA has major developmental problems

Researchers at the Rockefeller University, through their new study have rendered a great blow to the old convention that agrees about the symmetric roles of RNA that guided stem cells differentiation in a same way in all vertebrates from fish to human.

This new research have proved that microRNAs, works differently in different specise. Researchers say:
When it comes to microRNAs, what works for one animal may not work the same way in another.

Discovered in the 1990s and first named in 2001, microRNAs are single strands of RNA that put the brakes on gene expression, halting the translation process that makes proteins.

They closely observed the role of a family of microRNAs in the African clawed frog embryo and human embryonic stem cells, comparing their findings with earlier ones in fish, and found that although the genes for these microRNAs were identical across the three species, their function was different.

Researchers found that the microRNAs targeted in each species. Previous experiments had shown that this particular microRNA targets both the Nodals and the Lefties in zebra fish.

Postdoctoral associate Alessandro Rosa found that the same microRNA in both frogs and humans also targets the Lefties. But in humans, the microRNA does not directly target the Nodal protein, and in frogs, it targets only the pathways weakest elements.

The study has been published last month in Developmental Cell, bore that out.

Source: ScienceDaily

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Researchers from Los Angeles Children Hospital claimed that autologous stem cell transplant along with radiation and chemotherapy help improve the survival rates of the children with average and high-risk Medulloblastoma (MB).

Medulloblastoma is a relatively rare brain tumor in children and has a grave prognosis.

The treatment of Medulloblastoma brain tumor in children usually consists of maximum surgery followed by chemotherapy and radiation therapy.

The major problems with this intensive therapy approach is brain damage (leukoencephalopathy), which is thought to be predominantly due to radiation. Because of the risk of leukoencephalopathy, many children with MB are often treated initially with chemotherapy.

A recent multicenter trial has reported that children with average and high-risk MB have improved survivals following post-operative risk-adapted radiotherapy and four cycles of high-dose cyclophosphamide and cisplatin with peripheral blood stem cell support.

The details of this study appeared in an early online publication in Cancer on April 28, 2009.

The current study involved 19 patients, where six patients had not received prior irradiation and 13 patients had received prior radiation. All received high-dose thiotepa-based therapy with autologous stem cell support.

The three-year post-transplant event-free survival was 83% for patients not exposed to radiation therapy and 20% for patients who had received prior radiation therapy.

In the 13 patients who had received prior irradiation, four died of toxicity and five of tumor recurrence. There was a high rate of toxic death in patients transplanted within one year of radiotherapy.

Survivors following radiotherapy had a long interval from radiation to transplant and had chemoresponsive disease.

Source:Cancer Consultant

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Eight year old girl Sophie Edward who was suffering from leukaemia and underwent stem cell treatment three months ago is progressing positively.

Leukaemia is a cancer of the blood and is the most common cancer in children, accounting for one third of cases.

In leukaemia, normal control mechanisms in the blood break down and the bone marrow starts to produce large numbers of abnormal white blood cells, disrupting production of normal blood cells and affecting the vital functions that these blood cells carry out.

Leukaemia can be classified as either lymphoid or myeloid, denoting the type of white blood cell affected. It is also categorised as either acute or chronic, reflecting the speed of progression.

Sophie was diagnosed with a rare form of leukaemia, acute lymphoblastic leukaemia in February last year and tried bone marrow transplant earlier, but eventually her bone marrow transplant failed and she and her family had no other way left but to relay on stem cell transplant.

The original transplant that eventually failed, took place at St James’s Hospital, in Leeds, in October.

Three months ago Sophie underwent a special type of stem cell treatment that was the first of its kind in Leeds.

Doctors used part of the original bone marrow left over from the previous transplant, but as Sophie was too ill there wasn’t time for it to be treated, so instead it was transplanted unprepared and chemotherapy used to get rid of the cells she didn’t need.

According to Sophie’s mother Emma Edwards, of Newsome, Huddersfield, they practically evaluated each day after the eight-year-old underwent the stem cell treatment three months ago, certainly they were very much worried as the previous bone marrow transplant failed. But after three months they are now much relieved as so far all the signs are good and she is much better.

Almost all childhood leukaemias are of the acute form, meaning that they progress rapidly.

Acute lymphoblastic (lymphoid) leukaemia (ALL) accounts for more than 80% of cases of childhood leukaemia. It is the only form of leukaemia and one of the few forms of cancer that is more common in children than in adults.

Acute myeloid leukaemia (AML) accounts for most of the remaining cases.

Chronic leukaemias, which progress slowly, are very rare in childhood.

Chronic myeloid leukaemia (CML) accounts for less than 3% of childhood leukaemias (less than 15 children per year in the UK).

Chronic lymphoblastic leukaemia is unheard of in children.

Source: Yorkshire Post

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The Madison company, Cellular Dynamics, which was founded by stem cell pioneer and University of Wisconsin-Madison scientist, James Thomson in 2004, has an exclusive license agreement with a New York medical school to turn stem cells into heart cells.

The license will allow Cellular Dynamics to manufacture cardiac cells from stem cells and provide them to pharmaceutical and biotech companies that test the toxicity of drugs.

According to Cellular Dynamics the license will cover a key patent portfolio that will allow the company to make cardiac cells such as cardiomyocytes, endothelial cells and vascular smooth muscle cells and could make available those cells to pharmaceutical and biotech companies working on testing the live saving cardiac drugs.

The University of Wisconsin-Madison scientist are famous for important stem cell discoveries, they were the first to isolate human embryonic stem cells in 1998.

Cellular Dynamics, known as CDI, initially is involved in selling stem cell-derived heart cells to Roche and other pharmaceutical companies to help them test the toxicity of drugs.

Now they are looking forward to industrialize production of human cell types for research and create a bio bank in which people could store stem cells engineered from their DNA for use in personalized therapies or in testing reactions to drugs.

Source: Capital Times

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WASHINGTON – For the first time, researchers at the University at Buffalo have shown that it is possible to repair cardiac tissue and, in turn, reverse heart failure by injecting adult bone marrow stem cells into skeletal muscle.

The researchers used an animal model to demonstrate that the non-invasive procedure could increase myocytes, or heart cells, by two-fold and reduce cardiac tissue injury by 60 percent.

In addition, the therapy improved function of the left ventricle-the primary pumping chamber of the heart-by 40 percent.

It even reduced fibrosis-the hardening of the heart lining that impairs its ability to contract-by up to 50 percent.

“This work demonstrates a novel non-invasive mesenchymal stem cell (MSC) therapeutic regimen for heart failure based on an intramuscular delivery route,” said Dr. Techung Lee, UB associate professor of biochemistry and senior author on the paper.

Mesenchymal stem cells are found in the bone marrow, and can differentiate into a variety of cell types.

Lee said: “Injecting MSCs or factors released by MSCs improved ventricular function, promoted myocardial regeneration, lessened apoptosis (cell death) and fibrotic remodeling, recruited bone marrow progenitor cells and induced myocardial expression of multiple growth factor genes.

“These findings highlight the critical ‘cross-talks’ between the injected MSCs and host tissues, culminating in effective cardiac repair for the failing heart.

“An important feature of MSCs is their ability to produce a plethora of tissue healing effects, known as “tropic factors,” which can be harnessed for stem cell therapy for heart failure.

The multiple trophic factors produced by MSCs have already been shown to be capable of reducing tissue injury, inhibiting fibrosis, promoting angiogenesis, stimulating recruitment and proliferation of tissue stem cells, and reducing inflammatory oxidative stress, a common cause of cardiovascular disease and heart failure.

Lee added: “Since skeletal muscle is the most abundant tissue in the body and can withstand repeated injection of large number of stem cells, we thought it would be a good method to deliver MSCs. We hypothesized that MSCs, via secretion of these functionally synergistic trophic factors, would be able to rescue the failing heart even when delivered away from the myocardium.

“This study proves our hypothesis. We’ve demonstrated that injecting MSCs, or trophic factors released by MSCs, into skeletal muscle improved ventricular function, promoted regeneration of heart tissue, decreased cell death and improved other factors that cause heart failure.

“This non-invasive stem cell administration regimen, if validated clinically, is expected to facilitate future stem cell therapy for heart failure.”

The development has been reported in a paper appearing online in the Articles-in-Press section of the American Journal of Physiology-Heart Circulation Physiology. (ANI)

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MELBOURNE – In a world-first breakthrough, Australian scientists have used contact lenses coated in stem cells to restore a person’s sight.

Medical researchers from University of New South Wales used the technique to treat the damaged corneas of three patients. The patients’ vision improved within weeks of the groundbreaking procedure.

The results are published in the journal Transplant.

In the procedure, stem cells were harvested from the eyes of each patient. Then, they were cultured inside a contact lens, which was then stuck onto a damaged cornea in a “transplant” of regenerative cells.

The three patients treated had very poor vision caused by corneal disease – the fourth most common form of blindness, affecting around 10million worldwide.

“The procedure is totally simple and cheap,” News.com.au quoted university’s Dr Nick Di Girolamo, as saying.

“Unlike other techniques … there’s no suturing, there is no major operation, all that’s involved is harvesting a minute amount – less than a millimetre – of tissue from the ocular surface,” the expert added.

The lens stayed on for 10 days allowing stem cells to change their form, colonise and repair the cornea.

Girolamo said that in the two cases the stem cells were taken from their healthy eye – but the third patient posed an additional challenge because of a congenital disorder which affected both eyes.

“We took them from another part of the eye altogether – the conjunctiva which also harbours stem cells,” Di Girolamo said.

“The stem cells were able to change from the conjunctival phenotype to a corneal phenotype after we put them onto the cornea … that’s the beauty of stem cells,” the expert added. (ANI)

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