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Scientists perform first ‘in body’ gene editing

Scientists perform first ‘in body’ gene editing

Scientists think they have achieved the first gene editing inside the body, altering DNA in adults to try to treat a disease. However, it is unknown whether it will help or not.

Dr. Joseph Muenzer of the University of North Carolina at Chapel Hill administered the treatment.

The treatment was conducted by California based Sangamo Therapeutics.

Sangamo's studies involve men with Hunter or Hurler syndrome, diseases caused by a missing gene that makes an enzyme to break down certain sugar compounds. Without it, sugars build up and damage organs, often killing people in their teens.

What is Gene editing?

Genome editing is a way of making specific changes to the DNA of a cell or organism.

An enzyme cuts the DNA at a specific sequence, and when this is repaired by the cell a change or ‘edit’ is made to the sequence.

CRISPR-Cas9

It is the most common and cheap and efficient system used for genome editing.

CRISPR stands for clustered regularly interspaced short palindromic repeats.

Cas9 stands for CRISPR-associated protein 9, and is the nuclease part that cuts the DNA.

What can be the benefits?

CRISPR could be used to modify disease-causing genes in embryos brought to term, removing the faulty script from the genetic code of that person’s future descendants as well.

Genome editing (Gene editing) could potentially decrease, or even eliminate, the incidence of many serious genetic diseases, reducing human suffering worldwide.

It might also be possible to install genes that offer lifelong protection against infection.

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