CRISPR Gene-Editing Injection Corrects Mutation in Liver Cells

CRISPR Gene-Editing Injection Corrects Mutation in Liver Cells

CRISPR-based gene editing has huge potential: a new study demonstrates an easy method to achieve gene editing in targeted organs.


Ever since its discovery, the CRISPR Cas 9 for gene editing has been pushing the boundaries of genome modifications. Just last month, we reported how researchers were using CRISPR to fix cholesterol in monkeys. Now, in a major breakthrough, researchers at University College London (UCL) injected the CRISPR-based drug into the blood of six people with a genetic condition. Three of them showed promising results paving way for further trials using this approach.

Transthyretin amyloidosis is a slowly progressing condition caused by a mutation - a genetic defect in the TTR gene. The mutation causes the transthyretin protein produced by liver cells to misfold leading to amyloidosis - abnormal deposits of the protein in the body. Transthyretin amyloids occur in the nerves connecting the brain and spinal cord to muscles and sensory cells. This leads to a loss of sensation such as pain, heat, and touch in the body's extremities. The deposits occur over the years and symptoms can appear anywhere between the age of 20 to 70. The condition occurs rarely in people of European descent but is seen more frequently in Africa. Patisiran is an approved drug to treat this condition.