Columns

Promoting Shorter HTT Protein Production Shows Promise in Mice

ProQR Therapeutics’ experimental therapy QRX-704 significantly reduced levels of the toxic huntingtin (HTT) protein clumps that fuel Huntington’s disease in a mouse model of the disease, a study showed. The therapy, an antisense oligonucleotide (ASO) designed to promote the generation of a shorter, but working version of…