Friday, May 01, 2015

Scientists transform donor blood to make it safe for all


The NHS has welcomed the research at British Columbia University in Vancouver, Canada, but said clinical trials were needed.

Millions of hospital patients are set to benefit from a new medical breakthrough which turns all donated blood into a universal type.

Every day, thousands of people need donated blood. But only blood without A or B antigens, such as type O, can be given to people in need.

Type O blood is usually in short supply because less than one in two people have it and stocks are used up quickly.

Now scientists have made a major leap forward in making all blood universal. According to research in the Journal of the American Chemical Society, doctors have discovered an efficient way to transform A and B blood into a neutral type that can be given to any patient.

Currently, blood transfusions require that the blood type of the donor match that of the recipient. If they are not the same, a patient can suffer serious side effects and even die.

The exception is the universal-donor blood type O, which can be given to anyone because it doesn’t have the A or B antigens that could provoke an immune reaction. Up to now scientists have been searching for a way to convert types A and B into type O.


In new experiments, Dr Stephen Withers and a team from the University of British Columbia in Vancouver, Canada, found that some enzymes from bacteria can clip the sugars off red blood cells that give blood its “type.”

But the enzymes are not very efficient. So the research team set out to see if they could boost the activity of the enzymes. The researchers tweaked one of those enzymes and improved its ability to remove type-determining sugars by 170-fold, rendering it antigen-neutral and more likely to be accepted by patients regardless of their blood type.

In addition to blood transfusions, the researchers say their advance could potentially allow organ and tissue transplants from donors that would otherwise be mismatched. In the UK only 36 per cent of adults have blood group O+ (positive) and 11 per cent have O- (negative).

However, only four per cent of Britons are blood donors. According to latest figures, one in four people in Britain will need a blood donation at least once in their lifetime and 1.65 million units of donated blood, each of 470 millilitres, are issued to hospitals a year.

Nick Watkins, assistant director of research and development at the NHS Blood and Transplant service, said: “We welcome new developments that have the potential to improve transfusion practice. The concept of creating universal donor cells by chemical/enzymatic treatment is not new and clinical trials will be required before such a product could be used in patients. There remains a need for volunteer blood donors of all blood types for the foreseeable future."
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Saturday, July 12, 2014

Now, personalized gene therapies for vision loss

A new approach to develop personalized gene therapies for people suffering from retinitis pigmentosa (RP) has been developed by the researchers.

 The RP is one of the major causes of vision loss.
The approach uses induced pluripotent stem cell technology to transform skin cells into retinal cells. These cells are then used by the researchers as a patient-specific model for disease study and preclinical testing.

The researchers from Columbia University Medical Center (CUMC) used this approach to show that a form of RP caused by mutations to the gene MFRP (membrane frizzled-related protein) can disrupt the protein that gives retinal cells their structural integrity.

"The use of patient-specific cell lines for testing the efficacy of gene therapy to precisely correct a patient's genetic deficiency provides yet another tool for advancing the field of personalised medicine," said Stephen H Tsang, the Laszlo Z Bito Associate Professor of Ophthalmology and associate professor of pathology and cell biology.

The RP could also begin during infancy but its first symptoms typically emerge in early adulthood, causing night blindness. In later stages, the photoreceptors in the macula are destroyed by the RP. The photoreceptors are responsible for fine central vision.

"This study provides both in vitro and in vivo evidence that vision loss caused by MFRP mutations could potentially be treated through AAV gene therapy," said coauthor Dieter Egli, an assistant professor at CUMC.

The paper was published in Molecular Therapy, the official journal of the American Society for Gene & Cell Therapy.

 

 

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Saturday, January 25, 2014

Human stem cells converted to functional lung cells

Scientists have for the first time transformed human stem cells into functional lung and airway cells.
The advance, by Columbia University Medical Center (CUMC) researchers, has significant potential for modelling lung disease, screening drugs, studying human lung development, and, ultimately, generating lung tissue for transplantation.
“Researchers have had relative success in turning human stem cells into heart cells, pancreatic beta cells, intestinal cells, liver cells, and nerve cells, raising all sorts of possibilities for regenerative medicine,” said study leader Hans-Willem Snoeck.
“Now, we are finally able to make lung and airway cells. This is important because lung transplants have a particularly poor prognosis,” said Snoeck, professor of medicine (in microbiology & immunology) and affiliated with the Columbia Center for Translational Immunology and the Columbia Stem Cell Initiative.
“Although any clinical application is still many years away, we can begin thinking about making autologous lung transplants – that is, transplants that use a patient’s own skin cells to generate functional lung tissue,” Snoeck said.
The research builds on Snoeck’s 2011 discovery of a set of chemical factors that can turn human embryonic stem (ES) cells or human induced pluripotent stem (iPS) cells into anterior foregut endoderm – precursors of lung and airway cells.
In the current study, Snoeck and his colleagues found new factors that can complete the transformation of human ES or iPS cells into functional lung epithelial cells (cells that cover the lung surface).
The resultant cells were found to express markers of at least six types of lung and airway epithelial cells, particularly markers of type 2 alveolar epithelial cells.
Type 2 cells are important because they produce surfactant, a substance critical to maintain the lung alveoli, where gas exchange takes place; they also participate in repair of the lung after injury and damage.
The findings have implications for the study of a number of lung diseases, including idiopathic pulmonary fibrosis (IPF), in which type 2 alveolar epithelial cells are thought to play a central role.
“No one knows what causes the disease, and there’s no way to treat it,” said Snoeck.
“Using this technology, researchers will finally be able to create laboratory models of IPF, study the disease at the molecular level, and screen drugs for possible treatments or cures,” Snoeck said.
The study was published in the journal Nature Biotechnology. 
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