Tag: adult stem cells

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Find and Replace: DNA Editing Tool Shows Gene Therapy Promise

Caption: This image represents an infection-fighting cell called a neutrophil. In this artist’s rendering,  the cell’s DNA is being “edited” to help restore its ability to fight bacterial invaders.
Credit: NIAID, NIH

For gene therapy research, the perennial challenge has been devising a reliable way to insert safely a working copy of a gene into relevant cells that can take over for a faulty one. But with the recent discovery of powerful gene editing tools, the landscape of opportunity is starting to change. Instead of threading the needle through the cell membrane with a bulky gene, researchers are starting to design ways to apply these tools in the nucleus—to edit out the disease-causing error in a gene and allow it to work correctly.

While the research is just getting under way, progress is already being made for a rare inherited immunodeficiency called chronic granulomatous disease (CGD). As published recently in Science Translational Medicine, a team of NIH researchers has shown with the help of the latest CRISPR/Cas9 gene-editing tools, they can correct a mutation in human blood-forming adult stem cells that triggers a common form of CGD. What’s more, they can do it without introducing any new and potentially disease-causing errors to the surrounding DNA sequence [1].

When those edited human cells were transplanted into mice, the cells correctly took up residence in the bone marrow and began producing fully functional white blood cells. The corrected cells persisted in the animal’s bone marrow and bloodstream for up to five months, providing proof of principle that this lifelong genetic condition and others like it could one day be cured without the risks and limitations of our current treatments.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

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First US Babies Treated in Study of Adult Stem Cell Therapy for Congenital Heart Disease

December 22, 2016

(Medical Xpress) – In a first-in-children randomized clinical study, medical researchers at the University of Maryland School of Medicine (UM SOM) and the Interdisciplinary Stem Cell Institute (ISCI) at the University of Miami Miller School of Medicine have begun testing to see whether adult stem cells derived from bone marrow benefit children with the congenital heart defect hypoplastic left heart syndrome (HLHS). UM SOM surgeons are injecting the cells into the babies’ hearts during open-heart operations at the University of Maryland Medical Center. ISCI is supplying the stem cells for the procedures.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

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Cancer Not Mainly Down to ‘Bad Luck,’ Finds Stem Cell Study

October 4, 2016

(Medical News Today) – Last year, a controversial study suggested the majority of cancer cases are down to “bad luck” – that is, random DNA mutations in adult stem cells that are not caused by lifestyle factors. A new study contradicts this claim; while bad luck does play a role cancer development, researchers find it is unlikely to be the primary contributor.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

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What is the current clinical usefulness of stem cell treatments, embryonic and iPS cells?

Human adult stem cells (iPS) have been found to be most clinically useful human cells for therapeutic purposes

Pluripotent cells (PCs) are defined as those from which cells of different tissue types can be obtained. These can be obtained either from preimplantation human embryonic cells, in which case pluripotent embryonic stem cells are obtained, or from somatic (adult) cells that can be reprogrammed to a state of pluripotentiality, called induced pluripotent cells (iPS cells).

Ad present stem cell treatments with human adult stem cells have been found to be most clinically useful human cells for therapeutic purposes, especially cells from bone marrow, peripheral blood, umbilical cord blood or other tissues. Mesenchymal cells obtained from bone marrow, placenta and the umbilical cord are particularly useful. These cells have the advantage of having low immunogenicity and the disadvantage that after being transplanted, they persist for very little time in the recipient, which poses problems for maintaining their effect over time. These cells can be obtained from the patients themselves or from external donors, which gives rise to autologous therapy (which uses cells from the patient themselves) or allogenic therapy (which uses cells from individuals other than the patient). Their use has so far given good results as regards their safety, which has been evaluated in very diverse clinical trials. However, pluripotent cells, both embryonic and iPS cells, have limited clinical applications as they have been used in little more than ocular diseases. Therefore, it is of no interest to analyse this clinical usefulness at present, following a magnificent review recently published in Nature Reviews/Molecular Cell Biology (17; 194-200, 2016).

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

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Adult stem cells could effectively substitute embryonic cells in regenerative medicine

Many technics showed that adult stem cells could effectively substitute embryonic cells in cellular therapy. One medical and ethical problem to resolve is to find adequate substitute for the use of embryonic stem cells in regenerative and reparatory medicine. The best possibility could be to use adult stem cells. Among these are stem cells from amniotic fluid. An article has now been published in the American Journal of Obstetrics and Gynecology (214; 321-327, 2016), which reviews the usefulness of amniotic fluid stem cells, highlighting that there are no ethical difficulties in using them, and that clinically they can be applied in foetal therapy, degenerative diseases and in regenerative and reparatory medicine in general.

 

 

 

La entrada Adult stem cells could effectively substitute embryonic cells in regenerative medicine aparece primero en Observatorio de Bioética, UCV.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

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Ban on embryo research survives challenge in Italy

Italy’s Constitutional Court has reaffirmed the legitimacy of a ban on human embryo research. In a decision late last month the Court declared that Article 13 of Law 40, a 2004 law on assisted reproduction, was constitutional. 

The legislation was challenged by a couple who had created several embryos in an IVF clinic. They requested that the defective ones be made available for genetic research, although this obviously clashed with Law 40.

Geneticist Bruno Dallapiccola, of the Bambino Gesù Children’s Hospital, was scathing in his comments to the L’Avvenire newspaper:

“The embryo is not simply a mass of cells, but something more which deserves to be respected. The Constitutional Court’s ruling, by maintaining the ban on using frozen embryos for research, confirms this principle.”

He also expressed scepticism about the usefulness of human embryonic stem cells, in the wake of recent developments in stem cell research:

“We have not achieved the results that many hoped. The idea that research on embryonic stem is helpful in treating serious diseases today is, in my opinion, only a slogan that is not reflected in reality. While adult stem cells have led to tangible results which are transferable to clinical work, and while induced pluripotent cells have led to the creation of experimental models of diseases, embryonic stem cells have led nowhere.”  

This article is published by Michael Cook and BioEdge under a Creative Commons licence. You may republish it or translate it free of charge with attribution for non-commercial purposes following these guidelines. If you teach at a university we ask that your department make a donation.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

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Panel on infant lives meets in Washington

Professor G. Kevin Donovan of Georgetown University testifying before the Panel

The US Senate Select Investigative Panel on Infant Lives met for the first time last week, in a heated session that saw several experts testify against the procurement of fetal tissue for research. The panel was convened following the release of several videos apparently capturing Planned Parenthood employees negotiating the sale of tissue to private research firms.

Tensions on the panel were evident even before testimony began. Democrat and Republican representatives clashed over the subpoenaing of the names of researchers, technicians and medical personnel working in a select number of abortion clinics.

Referring to a shooting late last year at a clinic in Colorado, Democratic representative Jan Schakowsky said, “Linking individual’s names to an investigation that the Republicans describe as examining the ‘harvesting of baby body parts’ and the ‘horrific practices’ of abortion providers puts people in danger”. The chair of the committee, Republican Marsha Blackburn, replied that  the panel is “entitled to the information,” and a Democratic motion to quash the three subpoenas issued thus far was defeated on a party-line vote.

Bioethicists and scientists with a variety of perspectives appeared before the panel.

Professor G. Kevin Donovan of Georgetown’s Pellegrino Center for Clinical Bioethics said that said that tissue might be harvested from spontaneous miscarriages, thus avoiding the moral implications of using aborted fetuses. “If we cannot act with moral certainty regarding the appropriate respect and dignity of the fetus, we cannot morally justify its destruction. Alternatives clearly exist that are less controversial, and moral arguments exist that support our natural abhorrence at the trafficking of human fetal parts.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

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Human-animal chimeras produced for use in regenerative medicine

The production of human-animal chimeras, i.e. animal organisms with human organs or tissues, is currently in the spotlight of ethical debates in the field of regenerative medicine. The process consists of introducing human stem cells into early animal embryos, so that the developing animal forms human organs and tissues inside its body.

Although this technique would enable organs to be obtained for transplantation and studies to be conducted on human disease, it nonetheless raises important ethical questions that must be resolved.

The main concern is that on combining human cells with animal embryos, human brain cells or germ line cells could arise in the animal offspring, cells that are strongly associated with human identity.

As a result of these ethical difficulties, on 23rd September 2015, the National Institutes of Health in the United States reported that, for now, it will not fund this type of research.

Nevertheless, this does not mean that there is a regulation prohibiting these techniques. In fact, a study conducted by researchers at the Salk Institute for Biological Studies in La Jolla, California (USA) was recently published in scientific journal Nature, whose findings could mean a major breakthrough in this field. However in addition to the aforementioned ethical obstacles, this experimental methodology still has a considerable number of technical obstacles.

The team, led by Juan Carlos Izpisúa Belmonte, has discovered a new type of embryonic stem cells called region-selective pluripotent stem cells (rsPSCs), which have molecular and functional characteristics that give them a unique ability to generate chimeric embryos among species.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

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Abortion: “Loopholes” and Attitudes

Earlier this week, Dr. Scott Gottlieb wrote in the Wall Street Journal about “Closing the Planned Parenthood Loophole.”  In his article (which was accompanied by a photo of a 12-week fetus), Dr. Gottlieb argued that the 1993 National Institutes of Health Revitalization Act should be amended to delete the word “solely” from the requirement that the doctor performing the abortion certify that “no alteration of the timing, method, or procedures used to terminate the pregnancy was made solely for the purposes of obtaining the tissue.”  That “solely,” Dr. Gottlieb argues, allows Planned Parenthood (or another entity providing fetal tissue from abortions for the sake of research) to claim it is in accord with the letter of the law, while clearly flouting its spirit.

Well enough.  But Dr. Gottlieb also spends considerable effort to claim that the research requiring newly-acquired fetal tissue is uncommon, or can be done with embryonic stem cells, adult stem cells, or fetal cells that are already available.  (Presumably, induced pluripotent stem cells could also be used.)  “The market for fetal tissue is limited… [and] the demand for the tissue isn’t that high.”  So, toughening the laws in this regard would have “little consequence” to research that is supposedly “essential.” 

So why not just ban the use of newly-acquired fetal tissue for research, if the need is low or even non-existent?  The Journal has also recently reported on proposed state laws that would do just that.  A proposal in Wisconsin has apparently rankled the dean of the medical school there, who pronounces such a law “bizarre…it will turn the lights off on research.”

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.

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Cell therapy with IPs cells for corneal lesions

Cell therapy, repairing damaged tissues is a top therapeutic objective in current medicine. Cell therapy is one of the main medical instruments used to reach this objective, mainly using adult stem cells or IPs cells (reprogrammed cells). One of the clinical areas in which this cell therapy with IPs cells seems most promising is in the field of eye diseases.

The cornea is a transparent structure that allows us to see, but it is a structure that is easily damaged by trauma or infections, which can lead to reduced vision or even blindness.  One solution for these problems is corneal transplants, but these can be limited due to immune tolerance issues and the shortage of donors. One possible alternative is cell therapy.

In relation to this, Basu et al. (Sci Transl Medicine 6,266 ra172 (2014)) Cell therapya. These types of corneal cells could theoretically be produced from the patient’s own cells and used to prevent the blindness that corneal lesions can cause.

La entrada Cell therapy with IPs cells for corneal lesions aparece primero en Observatorio de Bioética, UCV.

The views, opinions and positions expressed by these authors and blogs are theirs and do not necessarily represent that of the Bioethics Research Library and Kennedy Institute of Ethics or Georgetown University.