�US researchers have establish a way to grow immortal cubicle strains and tissue types from pathologic patients by converting their cells into pluripotent
stem cells with the same genetic errors. The new cell lines will enable scientists to investigate ten-spot different genetic disorders like Parkinson's,
muscular dystrophy, and type 1 diabetes in the test tube instead of in the affected role, a huge step ahead compared to current methods.
The study is the work of researchers from Harvard Medical School, Massachusetts General Hospital, and the University of Washington, led by
Howard Hughes Medical Institute investigator George Q Daley who is based at Children's Hospital Boston. It is promulgated in the advanced on-line
publication of the journal Cell on August 7th.
Although scientists stimulate for years been ontogeny human cells from diseased patients in the research lab, current methods are unsatisfactory because cells
taken directly from patients have a limited life, and when these ar modified to make them "immortal", their physiology tail end change and make the
results confutative.
Induced pluripotent stem (iPS) cells are gaining ground in stem cell research because like embryonic root cells, they have the potential to turn into
almost whatsoever type of cell and body tissue paper, and the cell lines go on for ever; they don't have the limited lifespan drawback of cells taken directly from
patients, or the inherited characteristics of the tumor or pathologic tissue from which they were derived. This allows scientists to study "healthy" tissue
cultures with the genetic code of the disease as well as the diseased tissue.
In this study, Daley and colleagues produced induced pluripotent bow (iPS) cells from patients with 10 different familial diseases:
Adenosine deaminase deficiency-related austere combined immunodeficiency (ADA-SCID, a form of "boy in the gurgle disease"),
Shwachman-Bodian-Diamond syndrome (SBDS, which causes off-white marrow to fail and predisposes the patient to leukemia),
Gaucher disease (GD) type III (a metabolic disorder where fat accumulates in organs),
Duchenne powerful dystrophy (DMD),
Becker muscular dystrophy (BMD),
Parkinson disease (PD),
Huntington disease (HD),
Juvenile-onset, type 1 diabetes mellitus (JDM),
Down syndrome (DS)/trisomy 21, and
The carrier state of Lesch-Nyhan syndrome (an enzyme deficiency that builds up uric acidic in dead body fluids).
Thanks to a fund from the Harvard Stem Cell Institute, the stalk cell lines will be available to scientists planetary. It is possible to create stem cell
lines for early diseases to a fault, said the researchers.
Daley said:
"Researchers have long wanted to find a agency to move a patient's disease into the test tube, to develop cells that could be cultured into the many
tissues relevant to diseases of the stemma, the brain and the heart, for example."
"Now, we have a way to do just that -- to derive pluripotent cells from patients with disease, which way the cells can defecate any tissue and can grow
constantly. This enables us to model thousands of conditions using classical cell culture techniques," explained Daley.
To make a disease-specific iPS cellular telephone line, Daley and colleagues took cells from a diseased patient and motley them with a benign virus that introduced
what they called "reprogramming factors" to induce the cells to turn pluripotent, most like a reverse engine room process that reprograms the
cells plunk for to a pre-specialized state. And the beauty of it is, the cells also have the genetical code that reflects the type of disease the patient has
inherited.
Daley and colleagues then isolated the disease-specific iPS cells and analysed the genes to confirm that they did indeed deliver the same disease-causing defects as the original donor cells. And just to be sure, they besides confirmed they were really pluripotent and could differentiate into a range of
tissue types.
The researchers ar confident that in many cases these new iPS cell lines will mimic human diseases better than animal models. Although beast
models like mice ar close to humans in many slipway, some things are too different to make them useful, for instance Down's syndrome does not
causal agency the same symptoms in mice. New things that can be explored using iPS methods will be for case comparing how the same disease varies
among unlike people, or how drugs might impact different people with the same disease. The scientists will be able to create iPS cell lines of
unlike genetic types with the same disease defect.
In the longer term, as well as organism useful for research into disease and drug testing, it is possible that iPS methods can be used to re-engineer a
disease free version of a cell line specific to a patient then re-introduce them into the patient's dead body to substitute diseased cells.
Although Daley and colleagues are making their methods available to other scientists so they canful generate their own
disease-specific iPS cell lines, as Daley explained, it will not be easy:
"They don't grow like weeds; they're more like orchids," aforesaid Daley, "you really take to tend to them," he added.
He has offered to collaborate
with other scientists to facilitate them perfect the method for the diseases they want to investigate.
"Disease-Specific Induced Pluripotent Stem Cells."
In-Hyun Park, Natasha Arora, Hongguang Huo, Nimet Maherali, Tim Ahfeldt, Akiko Shimamura, M. William Lensch, Chad Cowan, Konrad
Hochedlinger, and George Q. Daley.
Cell advanced online issue 7th August 2008.
DOI: 10.1016/j.cell.2008.07.041
Click here for
Abstract.
Sources: Howard Hughes Medical Institute, journal abstract.
Written by: Catharine Paddock, PhD
Copyright: Medical News Today
Not to be reproduced without permission of Medical News Today
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