Monday October 22, 2018

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
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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)

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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)