Has gene editing or base editing succeeded in correcting a gene?

[A. Hammouda]

Has gene editing or base editing succeeded in correcting, not knocking out, a gene for treatment of a disease?

[Dr. Kumar]

I have read about experimental trials to decrease the number of tandem triplet repeats in Huntington gene. I will keep searching.

[John Brown]

The Huntington faulty gene has increased trinucleotide repeats that leads to formation of a faulty protein with a long polyglutamine tail. This abnormal protein misfolds, precipitates, and causes the known pathological changes in the affected person.

Current approaches targeting the gene aim at silencing this faulty gene to decrease the synthesis of the abnormal protein. Antisense oligonucleotide therapy is leading the way. There are trials using micro-RNA and small interfering RNA for the same effect.  The use of CRISPR technology obviously will aim at the same target, knocking out the faulty gene.

Here is a review of this subject: Gene Therapy for Huntington’s Disease: The Final Strategy for a Cure? – PMC (nih.gov)

 

 

• This reply was modified 2 years, 5 months ago by John Brown.
• This reply was modified 2 years, 5 months ago by John Brown.

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