Thursday November 15, 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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Novel Synthetic DNA Vaccines Safe To Use Against Ebola: Scientists

While there are no licensed treatments available for Ebola virus disease yet, multiple experimental therapies are being developed.

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Ebola, UNICEF. congo, DNA
A Congolese health worker administers Ebola vaccine to a woman who had contact with an Ebola sufferer in the village of Mangina in North Kivu province of the Democratic Republic of Congo

Scientists, including one of Indian-origin, have found that the novel synthetic DNA vaccine is safe against Ebola virus and offers a long-term alternative to traditional vaccines.

The team, from The Wistar Institute in Philadelphia, US, optimised a shorter, dose-sparing, immunisation regimen and simplified vaccine that can be directly administered into the skin. They targeted a virus surface protein called glycoprotein.

This new approach induced rapid and protective immunity from virus challenges.

Importantly, the approach showed strong immune responses one year after the last dose, supporting the long-term immunogenicity of the vaccine — a particularly challenging area for Ebola vaccines.

Ebola, UNICEF. congo, DNA
A boy runs past a dispenser containing water mixed with disinfectant, east of Mbandaka, DRC. VOA

“Synthetic non-viral based DNA technology allows for rapid vaccine development by delivery directly into the skin, resulting in consistent, potent and rapid immunity compared to traditional vaccine approaches,” said lead researcher David B. Weiner, Director of Wistar’s Vaccine and Immunotherapy Center.

“An anti-Ebola virus DNA vaccine like this may provide an important new tool for protection, and we are excited to see what future studies will unveil,” he added.

In the study, published in the Journal of Infectious Diseases, the team detected antibody levels were equal or higher to those reported for other vaccines currently being evaluated in the clinic.

“The success of intradermal delivery of a low-dose regimen is very encouraging,” said Ami Patel, Ph.D., associate staff scientist in the Weiner Lab. “The ultimate goal of our work is to create effective and safe vaccines that are optimised for field use in at-risk areas.”

Ebola, UNICEF. congo, DNA
Photo taken Sept 9, 2018, shows health workers walking with a boy suspected of having the Ebola virus at an Ebola treatment centre in Beni, Eastern Congo. VOA

Ebola virus disease is a serious and often fatal illness that can cause fever, headache, muscle pain, weakness, fatigue, diarrhoea, vomiting, stomach pain and haemorrhage (severe bleeding).

First discovered in humans in 1976 in the Democratic Republic of the Congo, the largest outbreak occurred in West Africa from 2014 to 2016, which claimed more than 11,000 lives, according to the World Health Organization.

Also Read: Ebola Increases The Number of Orphans in DRC: UNICEF

The death rate is about 50 per cent and the virus is spread by contact with contaminated body fluids, including blood and semen.

While there are no licensed treatments available for Ebola virus disease yet, multiple experimental therapies are being developed. (IANS)