Stahl Christine M, Feigin Andrew
NYU Langone Health, Marlene and Paolo Fresco Institute for Parkinson's and Movement Disorders, 222 East 41st Street, Floor 13, New York, NY 10017, USA.
NYU Langone Health, Marlene and Paolo Fresco Institute for Parkinson's and Movement Disorders, 222 East 41st Street, Floor 13, New York, NY 10017, USA.
Neurol Clin. 2020 May;38(2):367-378. doi: 10.1016/j.ncl.2020.01.010. Epub 2020 Mar 9.
Huntington disease, a neurodegenerative disease characterized by progressive motor, behavioral, and cognitive decline, is caused by a CAG trinucleotide repeat expansion in the huntingtin gene on chromosome 4. Current treatments target symptom management because there are no disease-modifying therapies at this time. Investigation of RNA-based and DNA-based treatment strategies are emerging and hold promise of possible future disease-modifying therapy.
亨廷顿舞蹈症是一种神经退行性疾病，其特征为进行性运动、行为和认知能力衰退，由4号染色体上亨廷顿基因中的CAG三核苷酸重复序列扩增引起。目前的治疗以症状管理为目标，因为目前尚无疾病修饰疗法。基于RNA和基于DNA的治疗策略的研究正在兴起，并有望在未来实现可能的疾病修饰治疗。
