Wednesday September 19, 2018

Australian university research holds out hope for thalassemia patients

UNSW is home to more than 52,000 students from nearly 130 countries

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There are 29 types of blood groups in reality.
There are 29 types of blood groups in reality.
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Researchers at the University of New South Wales (UNSW) in Sydney, Australia, have used CRISPR gene editing technology to introduce beneficial natural mutations into blood cells to boost production of foetal haemoglobin.

The method could lead to new therapies for sickle cell anaemia and other blood disorders, says the university. The research solves a 50-year-old mystery about how these mutations — which are naturally carried by a small percentage of people — operate and alter the expression of human genes.

A total of 100 men had serum levels indicative of hyponatremia. Wikimedia Commons
People with thalassemia have defective adult haemoglobin. Wikimedia Commons

The details of the study, carried out by an international team led by UNSW scientist Professor Merlin Crossley, is published in the journal Nature Genetics. Genome editing or gene editing give scientists the ability to change an organism’s DNA. These technologies allow genetic material to be added, removed or altered at particular locations in the genome.

“Our new approach can be seen as a forerunner to ‘organic gene therapy’ for a range of common inherited blood disorders including beta thalassemia and sickle cell anaemia,” said Professor Crossley, who is also UNSW Deputy Vice-Chancellor Academic.

“It is organic because no new DNA is introduced into the cells. Rather, we engineer in naturally occurring, benign mutations that are known to be beneficial to people with these conditions. It should prove to be a safe and effective therapy, although more research would be needed to scale the processes up into effective treatments,” he added.

Also Read: iPhone app measured blood flow better in cardiac assessment

People with thalassemia or sickle-cell anaemia have defective adult haemoglobin — the vital molecule that picks up oxygen in the lungs and transports it around the body — and require life-long treatment with blood transfusions and medications.

According to UNSW, it has engaged in a series of initiatives with the Indian government, higher education institutions, and corporations for sharing and transfer of its vast pool of tech expertise. This sets UNSW apart from host of other institutions that see India as a one-way street to train Indian students. UNSW is home to more than 52,000 students from nearly 130 countries. IANS

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CRISPR Gene Editing can Cause Risky Collateral DNA Damage: Study

The work has implications for how CRISPR/Cas9 is used therapeutically and is likely to re-spark researchers' interest in finding alternatives to the standard CRISPR/Cas9 method for gene editing

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Genetic variants on the X chromosome explain virtually identical amounts of variation in men and women. Pixabay

The much celebrated CRISPR/Cas9 gene editing technique can cause greater genetic damage in cells than was previously thought, scientists have warned.

CRISPR/Cas9 is a type of molecular scissor technology that can alter sections of DNA in cells by cutting at specific points and introducing changes at that location.

Besides extensive use in scientific research, CRISPR/Cas9 has also been seen as a promising way to create potential genome editing treatments for diseases such as HIV, cancer or sickle cell disease.

But the new research, reported in the journal Nature Biotechnology, revealed that CRISPR/Cas9 frequently caused extensive mutations, though at a distance from the target site.

Many of the cells, in both mice and humans, had large genetic rearrangements such as DNA deletions and insertions.

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CRISPR/Cas9 frequently caused extensive mutations, though at a distance from the target site.. Pixabay

These could lead to important genes being switched on or off, which could have major implications for CRISPR/Cas9 use in therapies.

In addition, some of these changes were too far away from the target site to be seen with standard genotyping methods, the researchers said.

“This is the first systematic assessment of unexpected events resulting from CRISPR/Cas9 editing in therapeutically relevant cells, and we found that changes in the DNA have been seriously underestimated before now,” said Allan Bradley, Professor at the Wellcome Sanger Institute in London.

Also Read: New Link Found Between Alcohol, Genes And Heart Failure

“It is important that anyone thinking of using this technology for gene therapy proceeds with caution, and looks very carefully to check for possible harmful effects,” Bradley added

The work has implications for how CRISPR/Cas9 is used therapeutically and is likely to re-spark researchers’ interest in finding alternatives to the standard CRISPR/Cas9 method for gene editing.

“While it is not known if genomic sites in other cell lines will be affected in the same way, this study shows that further research and specific testing is needed before CRISPR/Cas9 is used clinically,” the researchers said. (IANS)