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First ever attempt at gene editing inside the body

15-Nov-2017

Key points from article :

Patient lacks a gene that makes an enzyme that breaks down certain carbohydrates.

Symptoms include distorted facial features, hearing loss, breathing trouble and cognitive problems.

Zinc finger nucleases work like molecular scissors that replace a specific piece of DNA.

Only 1% of liver cells would have to be corrected to successfully treat the disease.

It becomes part of your DNA and is there for the rest of your life.

There is no way to erase any mistakes the editing might cause.

Tests will confirm whether it is working in three months.

Topics mentioned on this page:
Gene Therapy