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New gene therapy technique, CopyCatcher, more accurate in fruit flies

11-May-2021

Key points from article :

Novel genetic sensor called a CopyCatcher developed.

Detects instances in which a genetic element is copied precisely from one chromosome to another.

System detects a very high rate of copying in fruit flies.

"... one copy of a mutated gene could be repaired from a partially intact second copy of the gene,” said Professor Ethan Bier, senior author.

Needed if parents are carriers for two different mutations in the same gene.

Current gene therapy strategies in which a gene is placed at a different site acts as a crude “patch” - activating cells where they should normally be silent and vice-versa.

Using CopyCatcher, measures to improve copying in flies also translated to enhanced copying in human cells.

Next step is to further optimize the efficiency of corrective editing and develop model systems for human diseases.

Research by University of California San Diego published in Nature Communications.

Targets precise location of mutated gene rather than tagging on anywhere

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Professor, Cell and Developmental Biology, at University of California, San Diego

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Journal covering all topics in physics, chemistry, and biology.