Genome Editing: The Next Step in Gene Therapy


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Gene editing – and what it really means to rewrite the code of life
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The scientist was widely condemned by the global scientific community for violating longstanding scientific principles and ethical norms. Read More.

Powerful new gene-editing technologies, such as CRISPR-Cas9, hold great promise for advancing science and treating disease, but they also raise concerns and present complex challenges, particularly because of their potential to be used to make genetic changes that could be passed on to future generations, thereby modifying the human germline. Introduction The National Academy of Sciences and the National Academy of Medicine launched this initiative in to inform decision-making related to recent advances in human genome-editing research.

The science, the history and the future of gene therapy. - Rising Tide Biology

About This Initiative Powerful new gene-editing technologies, such as CRISPR-Cas9, hold great promise for advancing science and treating disease, but they also raise concerns and present complex challenges, particularly because of their potential to be used to make genetic changes that could be passed on to future generations, thereby modifying the human germline. Subscribe to our mailing list for updates by clicking on the button below.

While the approach has previously been shown to work in mice after birth, the latest study showed it was also possible to make the all-important tweak before they were born. However the scientists stressed it will be some time before such an approach would be ready for use in humans.


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  8. Crispr, or to give it its full name, Crispr-Cas9, allows scientists to precisely target and edit pieces of the genome. Crispr is a guide molecule made of RNA, that allows a specific site of interest on the DNA double helix to be targeted.

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    The RNA molecule is attached to a bacterial enzyme called Cas9 that works like a pair of 'molecular scissors' to cut the DNA at the exact point required. This allows scientists to cut, paste and delete individual letters of genetic code.

    Not Just CRISPR: Changing Biology Through Gene-Editing

    Even if the approach were eventually applied in humans, it would not be used for the majority of genetic disorders, Peranteau said. After showing it was possible to make a change at a particular spot in the DNA of liver cells in mouse foetuses, the team focused on a condition known as hereditary tyrosinemia type 1.

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    This is a genetic disease that prevents the body from breaking down an amino acid called tyrosine as it should, and can cause death if left untreated. The team took mice with a genetic mutation that produced a similar condition to hereditary tyrosinemia type 1 and bred them, with the mothers kept on a drug called nitisione.

    The team then injected 26 of the foetuses with a virus carrying the genetic instructions for making the gene-editing tool, and 27 of them with the same virus but without information for the tool.

    Genome Editing: The Next Step in Gene Therapy Genome Editing: The Next Step in Gene Therapy
    Genome Editing: The Next Step in Gene Therapy Genome Editing: The Next Step in Gene Therapy
    Genome Editing: The Next Step in Gene Therapy Genome Editing: The Next Step in Gene Therapy
    Genome Editing: The Next Step in Gene Therapy Genome Editing: The Next Step in Gene Therapy
    Genome Editing: The Next Step in Gene Therapy Genome Editing: The Next Step in Gene Therapy
    Genome Editing: The Next Step in Gene Therapy Genome Editing: The Next Step in Gene Therapy

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