Source: University of California - Irvine
Date: June 28, 2012
Summary:
An international consortium of Huntington's disease experts, including several from the Sue & Bill Gross Stem Cell Research Center at UC Irvine, has generated a human model of the deadly inherited disorder directly from the skin cells of affected patients. The re-created neurons, which live in a petri dish, will help researchers better understand what disables and kills brain cells in people with HD and let them gauge the effects of potential drug therapies on cells that are otherwise locked deep in the brain. The research is published online June 28 in the journal Cell Stem Cell.
Showing posts with label hereditary diseases. Show all posts
Showing posts with label hereditary diseases. Show all posts
Thursday, June 28, 2012
Turning Skin Cells Into Brain Cells: Huntington's Disease in a Dish
Source: Johns Hopkins Medical Institutions
Date: June 28, 2012
Summary:
Johns Hopkins researchers, working with an international consortium, say they have generated stem cells from skin cells from a person with a severe, early-onset form of Huntington's disease (HD), and turned them into neurons that degenerate just like those affected by the fatal inherited disorder.
To conduct their experiment, researchers took a skin biopsy from a patient with very early onset HD.When seen by Ross at the HD Center at Hopkins, the patient was just seven years old. She had a very severe form of the disease, which rarely appears in childhood, and of the mutation that causes it. Using cells from a patient with a more rapidly progressing form of the disease gave Ross' team the best tools with which to replicate HD in a way that is applicable to patients with all forms of HD.
Her skin cells were grown in culture and then reprogrammed in a lab into induced pluripotent stem cells. Scientists converted those cells into generic neurons and then into medium spiny neurons. What they found was that the medium spiny neurons deriving from HD cells behaved just as they expected medium spiny neurons from an HD patient would. They showed rapid degeneration when cultured in the lab using basic culture medium without extensive supporting nutrients. By contrast, control cell lines did not show neuronal degeneration.
The research, published in the journal Cell Stem Cell, is the work of a Huntington's Disease iPSC Consortium, including scientists from the Johns Hopkins University School of Medicine in Baltimore, Cedars-Sinai Medical Center in Los Angeles and the University of California, Irvine, as well as six other groups.
Date: June 28, 2012
Summary:
Johns Hopkins researchers, working with an international consortium, say they have generated stem cells from skin cells from a person with a severe, early-onset form of Huntington's disease (HD), and turned them into neurons that degenerate just like those affected by the fatal inherited disorder.
To conduct their experiment, researchers took a skin biopsy from a patient with very early onset HD.When seen by Ross at the HD Center at Hopkins, the patient was just seven years old. She had a very severe form of the disease, which rarely appears in childhood, and of the mutation that causes it. Using cells from a patient with a more rapidly progressing form of the disease gave Ross' team the best tools with which to replicate HD in a way that is applicable to patients with all forms of HD.
Her skin cells were grown in culture and then reprogrammed in a lab into induced pluripotent stem cells. Scientists converted those cells into generic neurons and then into medium spiny neurons. What they found was that the medium spiny neurons deriving from HD cells behaved just as they expected medium spiny neurons from an HD patient would. They showed rapid degeneration when cultured in the lab using basic culture medium without extensive supporting nutrients. By contrast, control cell lines did not show neuronal degeneration.
The research, published in the journal Cell Stem Cell, is the work of a Huntington's Disease iPSC Consortium, including scientists from the Johns Hopkins University School of Medicine in Baltimore, Cedars-Sinai Medical Center in Los Angeles and the University of California, Irvine, as well as six other groups.
Thursday, January 19, 2012
INVESTIGATORS ACHIEVE IMPORTANT STEP TOWARD TREATING HUNTINGTON'S DISEASE
Source: University of California - Davis
Date: January 19, 2012
Summary:
A team of researchers at the UC Davis Institute for Regenerative Cures has developed a technique for using stem cells to deliver therapy that specifically targets the genetic abnormality found in Huntington's disease, a hereditary brain disorder that causes progressive uncontrolled movements, dementia and death. The findings, now available online in the journal Molecular and Cellular Neuroscience, suggest a promising approach that might block the disease from advancing.
Date: January 19, 2012
Summary:
A team of researchers at the UC Davis Institute for Regenerative Cures has developed a technique for using stem cells to deliver therapy that specifically targets the genetic abnormality found in Huntington's disease, a hereditary brain disorder that causes progressive uncontrolled movements, dementia and death. The findings, now available online in the journal Molecular and Cellular Neuroscience, suggest a promising approach that might block the disease from advancing.
Monday, December 12, 2011
Study finds iPS cells match embryonic stem cells in modeling human disease
Source: Stanford University School of Medicine
Date: December 12, 2011
Summary:
Stanford University School of Medicine investigators have shown that iPS cells, viewed as a possible alternative to human embryonic stem cells, can mirror the defining defects of a genetic condition — in this instance, Marfan syndrome — as well as embryonic stem cells can. An immediate implication is that iPS cells could be used to examine the molecular aspects of Marfan on a personalized basis. Embryonic stem cells, on the other hand, can’t do this because their genetic contents are those of the donated embryo, not the patient’s.
This proof-of-principle regarding the utility of induced pluripotent stem cells also has more universal significance, as it advances the credibility of an exciting approach that’s been wildly acclaimed by some and viewed through gimlet eyes by others: the prospect of using iPS cells in modeling a broad range of human diseases. These cells, unlike ESCs, are easily obtained from virtually anyone and harbor a genetic background identical to the patient from which they were derived. Moreover, they carry none of the ethical controversy associated with the necessity of destroying embryos.
The study was published online Dec. 12 in Proceedings of the National Academy of Sciences.
Date: December 12, 2011
Summary:
Stanford University School of Medicine investigators have shown that iPS cells, viewed as a possible alternative to human embryonic stem cells, can mirror the defining defects of a genetic condition — in this instance, Marfan syndrome — as well as embryonic stem cells can. An immediate implication is that iPS cells could be used to examine the molecular aspects of Marfan on a personalized basis. Embryonic stem cells, on the other hand, can’t do this because their genetic contents are those of the donated embryo, not the patient’s.
This proof-of-principle regarding the utility of induced pluripotent stem cells also has more universal significance, as it advances the credibility of an exciting approach that’s been wildly acclaimed by some and viewed through gimlet eyes by others: the prospect of using iPS cells in modeling a broad range of human diseases. These cells, unlike ESCs, are easily obtained from virtually anyone and harbor a genetic background identical to the patient from which they were derived. Moreover, they carry none of the ethical controversy associated with the necessity of destroying embryos.
The study was published online Dec. 12 in Proceedings of the National Academy of Sciences.
Wednesday, September 28, 2011
CORRECTING SICKLE CELL DISEASE WITH STEM CELLS
Source: Johns Hopkins Medical Institutions
Date: September 28, 2011
Summary:
Using a patient’s own stem cells, researchers at Johns Hopkins have corrected the genetic alteration that causes sickle cell disease (SCD), a painful, disabling inherited blood disorder that affects mostly African-Americans. The corrected stem cells were coaxed into immature red blood cells in a test tube that then turned on a normal version of the gene. The research team cautions that the work, done only in the laboratory, is years away from clinical use in patients, but should provide tools for developing gene therapies for SCD and a variety of other blood disorders.
In an article published online August 31 in Blood, the researchers say they are one step closer to developing a feasible cure or long-term treatment option for patients with SCD, which is caused by a single DNA letter change in the gene for adult hemoglobin, the principle protein in red blood cells needed to carry oxygen. People who inherited two copies — one from each parent — of the genetic alteration, the red blood cells are sickle-shaped, rather than round. The misshapen red blood cells clog blood vessels, leading to pain, fatigue, infections, organ damage and premature death.
Date: September 28, 2011
Summary:
Using a patient’s own stem cells, researchers at Johns Hopkins have corrected the genetic alteration that causes sickle cell disease (SCD), a painful, disabling inherited blood disorder that affects mostly African-Americans. The corrected stem cells were coaxed into immature red blood cells in a test tube that then turned on a normal version of the gene. The research team cautions that the work, done only in the laboratory, is years away from clinical use in patients, but should provide tools for developing gene therapies for SCD and a variety of other blood disorders.
In an article published online August 31 in Blood, the researchers say they are one step closer to developing a feasible cure or long-term treatment option for patients with SCD, which is caused by a single DNA letter change in the gene for adult hemoglobin, the principle protein in red blood cells needed to carry oxygen. People who inherited two copies — one from each parent — of the genetic alteration, the red blood cells are sickle-shaped, rather than round. The misshapen red blood cells clog blood vessels, leading to pain, fatigue, infections, organ damage and premature death.
Sunday, May 22, 2011
Study of stem cell diseases advanced by new Stanford technique
Source: Stanford University Medical Center
Date: May 22, 2011
Summary:
STANFORD, Calif. — A rare genetic disease called dyskeratosis congenita, caused by the rapid shortening of telomeres (protective caps on the ends of chromosomes), can be mimicked through the study of undifferentiated induced pluripotent stem cells, according to new findings from the Stanford University School of Medicine. Although dyskeratosis affects only about one in a million people, the scientists' findings could greatly facilitate research into this and other diseases caused by stem cell malfunctions, including some bone marrow failure syndromes and, perhaps, pulmonary fibrosis.
The study, which used iPS cells created from the cells of patients with dyskeratosis, explains why sufferers experience a wide variety in the types and severity of symptoms, ranging from abnormal skin pigmentation and nail growth to lung scarring, bone marrow failure and cancer. The key lies in the activity of telomerase, an enzyme critical to aging and cell renewal. The study will be published online May 22 in Nature.
Date: May 22, 2011
Summary:
STANFORD, Calif. — A rare genetic disease called dyskeratosis congenita, caused by the rapid shortening of telomeres (protective caps on the ends of chromosomes), can be mimicked through the study of undifferentiated induced pluripotent stem cells, according to new findings from the Stanford University School of Medicine. Although dyskeratosis affects only about one in a million people, the scientists' findings could greatly facilitate research into this and other diseases caused by stem cell malfunctions, including some bone marrow failure syndromes and, perhaps, pulmonary fibrosis.
The study, which used iPS cells created from the cells of patients with dyskeratosis, explains why sufferers experience a wide variety in the types and severity of symptoms, ranging from abnormal skin pigmentation and nail growth to lung scarring, bone marrow failure and cancer. The key lies in the activity of telomerase, an enzyme critical to aging and cell renewal. The study will be published online May 22 in Nature.
Tuesday, June 03, 2008
Stem Cells Correct Defect in Child’s Fatal Skin Disease
Source: Columbia University Medical Center
Date: June 3, 2008
Summary;
Researchers and clinicians have paved the way toward a cure for a young boy's genetic and fatal skin disease, recessive dystrophic epidermolysis bullosa (RDEB), by using a cord blood and bone marrow transplant.
Date: June 3, 2008
Summary;
Researchers and clinicians have paved the way toward a cure for a young boy's genetic and fatal skin disease, recessive dystrophic epidermolysis bullosa (RDEB), by using a cord blood and bone marrow transplant.
Friday, December 16, 2005
Stem-cell therapies make a comeback
Source: New Scientist
16 December 2005
Summary:
Stem cells that migrate from bone marrow and fuse with cells in other tissues could one day be used to repair cells in people with genetic diseases.
16 December 2005
Summary:
Stem cells that migrate from bone marrow and fuse with cells in other tissues could one day be used to repair cells in people with genetic diseases.
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