“Latest Stem Cells News”

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.

“For the first time, we have been able to successfully deliver inhibitory RNA sequences from stem cells directly into neurons, significantly decreasing the synthesis of the abnormal huntingtin protein,” said Jan A. Nolta, principal investigator of the study and director of the UC Davis stem cell program and the UC Davis Institute for Regenerative Cures. “Our team has made a breakthrough that gives families affected by this disease hope that genetic therapy may one day become a reality.”

Huntington’s disease can be managed with medications, but currently there are no treatments for the physical, mental and behavioral decline of its victims. Nolta and other experts think the best chance to halt the disease’s progression will be to reduce or eliminate the mutant huntingtin (htt) protein found in the neurons of those with the disease. RNA interference (RNAi) technology has been shown to be highly effective at reducing htt protein levels and reversing disease symptoms in mouse models.

“Our challenge with RNA interference technology is to figure out how to deliver it into the human brain in a sustained, safe and effective manner,” said Nolta, whose lab recently received funding from the California Institute for Regenerative Medicine to develop an RNAi delivery system for Huntington’s disease. “We’re exploring how to use human stem cells to create RNAi production factories within the brain.”

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For the first time, researchers at Brigham and Women’s Hospital (BWH) have identified a human lung stem cell that is self-renewing and capable of forming and integrating multiple biological structures of the lung including bronchioles, alveoli and pulmonary vessels.  This research is published in the May 12, 2011 issue of the New England Journal of Medicine.

“This research describes, for the first time, a true human lung stem cell.  The discovery of this stem cell has the potential to offer those who suffer from chronic lung diseases a totally novel treatment option by regenerating or repairing damaged areas of the lung,” said Piero Anversa, MD, director of the Center for Regenerative Medicine at Brigham and Women’s Hospital and corresponding author.

Using lung tissue from surgical samples, researchers identified and isolated the human lung stem cell and tested the functionality of the stem cell both in vitro and in vivo.  Once the stem cell was isolated, researchers demonstrated in vitro that the cell was capable of dividing both into new stem cells and also into cells that would grow into various types of lung tissue.  Next, researchers injected the stem cell into mice with damaged lungs.  The injected stem cells differentiated into new bronchioles, alveoli and pulmonary vessel cells which not only formed new lung tissue, but also integrated structurally to the existing lung tissue in the mice.

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A notice for 8 million euros in funding for stem cell projects, a line of research that promises important results for ocular diseases, Parkinson’s, heart diseases, and the fight against tumors was announced by deputy health minister Ferruccio Fazio, who while speaking to the AGI press agency underlined “the importance of regenerative medicine, one of the great hopes for the future, as well as biotechnologies for new treatments altering molecular systems”.

The notification will expire on July 20 and is intended for universities, the National Research Council (CNR), and other private and public research groups. Three independent judges, one of whom will be from abroad, will evaluate the projects that are presented. The research topics eligible for funding include innovative strategies for experimental models (cellular and animal), risk/benefit analysis for pharmacological and non-pharmacological treatments and epidemiology research.

An allocation of three million euros for the next three years is planned to finance projects on rare diseases. No referral to embryonic stem cells, which are considered by many experts to be more promising in research because they are totipotent, or potentially able to become any type of cell, was made in the notice. “This was a choice,” specified Fazio, “that the Federal and Regional government conference made, which asked for a change to the original announcement, which was open also to stem cells.

Not mentioning them was a not a political choice by the health ministry, but a decision made by the federal and regional governments”. In any case, explained the deputy minister, “studying adult stem cells and their differentiation is more interesting to me and I believe that it is more advantageous”. There are numerous possibilities for treatments: “Not only ocular and cardiac diseases, Parkinson’s and rare diseases,” explained Fazio, “but also new possibilities to fight tumors.

Some researchers believe that tumor stem cells are the most aggressive stem cells of the tumor, and somehow determine the spread of the tumor. Tumor stem cell research could help understand the evolution of tumors and prevent their growth by striking their stem cells”. Financing, assured Fazio, “is in line with previous funding and in 2009 there will be funding for general research, open to all lines of research, therefore there will be funds available for stem cells.”

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British regulators have given Moorfields approval to begin trials using retinal cells derived from human embryonic stem cells (hESCs).

Twelve patients with Stargardt’s disease will have the cells injected into the eye. You can read more about the trial here.
Although there is great excitement about the trial, Julia knows that the initial phase will simply check safety (…)

“It would be marvellous if I could get some of my sight-loss reversed”, said Julia. “Even if it simply halts the deterioration, that would be great. And the real benefit would be for children. It could mean they don’t need to lose any sight and have normal vision.”

But doctors are urging caution. They are aware that there has been a lot of hype surrounding human embryonic stem cells, because of their potential to turn into any of the 200 or so cell types in the body (…)

It is always worth pointing out that other forms of stem cell treatments have been used successfully for many years.
Bone marrow transplantation is a well-established and proven technology.
More recently, stem cells from a patient’s own bone marrow have been used to help create them a new windpipe.

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Stanford University’s Faculty Senate today approved the creation of what officials believe is the first PhD program devoted solely to stem cell science in the nation and, perhaps, the world. The new doctoral program in stem cell biology and regenerative medicine is also the first interdisciplinary doctoral program created by the School of Medicine in recent years.

School officials say the fact that the university is taking the rare step of creating a new doctoral program acknowledges the growing importance of stem cell research in the realm of biomedical science. The senate’s initial approval of the program extends for five years.

“Stem cell biology is a distinct discipline that requires unique skills and includes a scope of knowledge and a skill set that is not covered by other disciplines,” said Renee Reijo Pera, PhD, professor of obstetrics and gynecology and director of the new PhD program.

Program leaders note that Stanford is among a small number of U.S. universities that have the necessary ingredients to create a doctoral program teaching the full range of stem cell science. They add that although a few other schools have recently established PhD programs involving stem cell biology, Stanford is the first to create a free-standing doctoral program dedicated solely to stem cell biology and regenerative medicine.

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Is there a future for stem cell therapies that don’t use embryonic stem cells? An international study involving EPFL has raised doubts, by showing that “reprogramming” adult stem cells leads to genetic aberrations.

It’s a discordant note in the symphony of good news that usually accompanies stem cell research announcements. Stem cells hold enormous promise in regenerative medicine, thanks to their ability to regenerate diseased or damaged tissues. They have made it possible to markedly improve the effectiveness of many medical treatments – muscle regeneration in cases of dystrophy, skin grafts for treating burn victims, and the treatment of leukemia via bone marrow transplants.

The problem is obtaining them. Those that are the true source of life, in the first days of embryonic development, are of course the most highly sought after; still undifferentiated, they are “pluripotent,” meaning they can evolve into liver, muscle, eye – any kind of cell. But the issue of how to obtain them clearly raises insurmountable ethical questions.

“In this regard, the recent discovery of the “reprogramming” phenomenon, by which somatic cells can be induced to convert to a pluripotent state simply by forcing the expression of a few genes, opens a phenomenal number of possibilities in regenerative medicine,” says Didier Trono, Dean of the EPFL School of Life Sciences.

“Imagine, for example, collecting a few cells from the hair follicle of a hemophiliac patient, reprogramming them to the pluripotentiality of their embryonic precursor, correcting the mutation responsible for the coagulation disorder that plagues the patient, and then re-administering them, genetically “cured,” after having orchestrated a differentiation into fully functional progeny.”

Increased risks for cancer?

But a study that has just been published in the journal Cell Death and Differentiation, to be followed by two articles in the journal Nature, is dampening those hopes. Conducted by the Department of Biochemistry at the University of Geneva and the European Institute of Oncology in Milan, with the participation of Trono’s laboratory, it concludes that these reprogrammed cells exhibit a “genomic instability” that appears to be caused by the process used to return the cells to their embryonic state.

Even more serious, the genetic mutations observed resemble mutations that are found in cancer cells. The scientists draw the conclusion that reprogrammed stem cells need to be extensively investigated before they can even be considered for use in regenerative medicine.
The experiments were done using mouse mammary and fibroblast cells. The researchers used three different processes for reprogramming the cells to a “stem,” or embryonic, state. The first method was developed expressly for this study, and the others have already been well documented.

Yet all the processes led to the same, implacable conclusion: the genetic anomalies multiplied, in a manner that seems to indicate that they are inherent to the reprogramming process itself, which typically makes use of oncogenes. “Interestingly, oncogenes have the potential to induce genomic instability,” the authors explain.

These results underline the necessity of conducting further studies. First, to see if the genetic anomalies are serious enough to compromise the function and stability of cells regenerated using the reprogrammed cells; and second, to “refine the methods used for generating induced pluripotent cells, in order to avoid this problem. These results will thus motivate scientists to come up with a solution,” concludes Trono.

from http://goo.gl/elaDN

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