Maastricht Economic and social Research and  training centre on Innovation and Technology

Levelling Latin America
Mining innovation can bring more sustainable and inclusive growth, especially across the Americas…

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  • A mystery source is producing banned ozone-destroying chemicals
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  • In an interplanetary first, NASA to fly a helicopter on Mars
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  • Gene editing staves off deafness in mice
    Genome editing has been used to reduce hearing loss in 'Beethoven' mice, which carry a mutation that causes deafness in both mice and humans.

    The research from the Broad Institute of MIT and Harvard in Cambridge, Massachusetts relies on a technique called CRISPR-Cas9 to knock out a mutant form of the gene Tmc1. In doing so, it lays out a potential pathway for treating other genetic causes of hearing loss. It also addresses a major problem facing the field of genome editing: how to deliver the protein and RNA needed for the CRISPR-Cas9 technique into the cells of a living animal.

    In this case, the researchers encapsulated the CRISPR components in positively charged fatty molecules called lipids, which are capable of crossing cell membranes. They then injected those particles directly into the inner ears of the mice, where the lipids were taken up by the hair cells that sense acoustic vibrations.

    The method could be used to enable gene therapy in people, if additional testing shows it to be safe and effective, according to the researchers.

    In all the tests, mice with treated ears performed better than control mice that had not been injected with the gene-editing components. For example, eight weeks after the injection, untreated control mice did not react to an abrupt 120-decibel noise - roughly the volume of a rock concert or a chainsaw. Mice that received the treatment, however, were significantly startled by the noise. It will take further tests in animals and people before it will be clear whether the same approach could work for humans who have TMC1 mutations.

    Nature    December 20, 2017