Tag: gene engineering

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Paralysed Mice Walk Again with ‘Designer’ Cytokines

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Scientists have sought a means to regenerate spinal cord injuries which leaves patients paraplegic or quadriplegic – and now a breakthrough by researchers at Ruhr-University Bochum, Germany, may see that dream realised.

By the time humans reach adulthood, after an injury they can no longer regenerate the axons which transfer nerve impulses from brain to muscles. In 2013, the researchers discovered that a cytokine called interleukin-6 (IL-6) promoted the regeneration of optic axon fibres in vitro. IL-6 was known to be involved in nerve regeneration as well as in neuropathic pain from peripheral nerve injuries. As promising as this experiment was, delivery of the cytokine to the injury location deep in the body was an obstacles, as was the fact that it had a fairly weak effect on stimulating nerve tissue regrowth.

The team subsequently developed hyper-IL-6, an artificial variant of IL-6 that was far more potent than its natural counterpart. However, the “designer” cytokine still could not be delivered to the injured tissue where it was needed. To get around this, the researchers turned to a somewhat novel delivery method: gene therapy. A few motor neurons in the brain’s sensorimotor cortex are altered via engineered viruses to produce hyper-IL-6, which is then distributed along the axon’s length to the injury site.

“Thus, gene therapy treatment of only a few nerve cells stimulated the axonal regeneration of various nerve cells in the brain and several motor tracts in the spinal cord simultaneously,” explained senior author Dr Dietmar Fischer.

After a single injection of the engineered virus and its hyper-IL-6 payload, mice with severed spinal cords were able to walk again after two to three weeks.

“This came as a great surprise to us at the beginning, as it had never been shown to be possible before after full paraplegia,” said Dr. Fischer.
Following the success of these experiments, Dr. Fischer’s team is looking at combining the engineered cytokine treatment to other promising applications, such as tissue grafts. Additionally, they are investigating whether the hyper-IL-6 treatment can regenerate spinal cord damage that occurred weeks beforehand.

“This aspect would be particularly relevant for application in humans. We are now breaking new scientific ground. These further experiments will show, among other things, whether it will be possible to transfer these new approaches to humans in the future.”

However, adapting this designer cytokine treatment to be one that is safe for humans will take several years.

Source: Medical News Today

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New Book on CRISPR Gene-editing Technology

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A new book, “Editing Humanity: The Crispr Revolution and the New Era of Genome Editing”, by Kevin Davies lays out the story that led to developing the revolutionary CRISPR gene-editing technology.

It describes the development of the technology involved clearly and succinctly, from the beginnings of gene sequencing to the 2012 discovery that an ancient bacterial immune system could be adapted and become the basis of the CRISPR gene-editing technology.

It also describes the misuse of the technology, such as with early gene therapy being done outside of the US on girls and young women to avoid US oversight. And despite a 2015 consensus that human sperm, eggs or embryos should not be edited, a scientist announced in 2018 that he had used gene editing on a pair of twin girls to confer HIV protection. 

The book however does not address how ethical issues should be resolved, such as releasing genetically engineered mosquitoes into the environment to suppress malaria.

Source: Nature

Categories : Genetics HIV T-cellsTags : Leave a comment

Long-term HIV Immunisation in Mice with Gene Technology

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While some COVID vaccines are entering the final phases of approval less than a year before the disease was first identified, HIV still has no vaccine after decades of research.

Now, engineered immune cells have elicited a response against HIV in mice, presenting an important first step forward in the quest for a vaccine. These broadly neutralising antibodies (bnabs) are effective against a variety of viruses and neutralise the glycan protecting HIV’s proteins.

Previous research had engineered B cells that produced the same antibodies as seen in rare HIV patients who are able to produce bnabs against HIV after many years. Now, this research has shown that it was possible to mature these into memory and plasma cells, conferring long-lasting protection and even showing improved antibodies can be produced, as in the immunisation process.

Principal investigator James Voss, PhD, of Scripps Research said, “This is the first time it has been shown that modified B cells can create a durable engineered antibody response in a relevant animal model.”

Currently it appears it would be an expensive therapy and a great barrier to many of the 38 million living with HIV around the world. A blood draw would be taken to the lab to engineer a vaccine for the patient, but Voss says that his team is looking to make the procedure inexpensive.

“People think of cell therapies as being very expensive,” Voss said. “We’re doing a lot of work towards trying to make the technology affordable as a preventative HIV vaccine or functional cure that would replace daily antiviral therapy.”

Source: Science Daily