Which HTT transcript to lower?

Min Zhang1

  • 1Guangdong Key Laboratory for Biomedical Measurements and Ultrasound Imaging, National-Regional Key Technology Engineering Laboratory for Medical Ultrasound, School of Biomedical Engineering, Shenzhen University Medical School, Shenzhen University, Shenzhen, China.

Huntington's disease (HD) RNA therapeutics have focused on lowering canonical huntingtin (HTT), yet clinical benefit remains uncertain. Papadopoulou et al. and Bragg et al. suggest that HTT1a-engaging strategies can produce stronger molecular rescue than full-length HTT lowering that spares HTT1a, highlighting transcript-species coverage as a key variable in HTT-lowering pharmacology.

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