Huntington’s breakthrough may stop disease... Huntington’s breakthrough may stop disease 11 Dec.`17 - The defect that causes the neurodegenerative disease Huntington's has been corrected in patients for the first time, the BBC has learned.
From your quote-- Huntington's is caused by an error in a section of DNA called the huntingtin gene. Normally this contains the instructions for making a protein, called huntingtin, which is vital for brain development. But a genetic error corrupts the protein and turns it into a killer of brain cells. The treatment is designed to silence the gene. If they fully silence that gene, won't it interfere with brain development? IMO they would need to replace the defective gene eventually.
