Ling Pan1, Andrew Feigin2. 1. Department of Neurology, The Marlene and Paolo Fresco Institute for Parkinson's and Movement Disorders, NYU Langone Health, 222 East 41st Street - 13th Floor, New York, USA. ling.pan@nyulangone.org. 2. Department of Neurology, The Marlene and Paolo Fresco Institute for Parkinson's and Movement Disorders, NYU Langone Health, 222 East 41st Street - 13th Floor, New York, USA.
Abstract
PURPOSE OF REVIEW: This article describes and discusses new potential disease-modifying therapies for Huntington's disease that are currently in human clinical trials as well as promising new therapies in preclinical development. RECENT FINDINGS: Multiple potential disease-modifying therapeutics for HD are in active development, including direct DNA/gene therapies, RNA modulation, and therapies targeted at aberrant downstream pathways. The etiology of Huntington's disease (HD) is well-known as an abnormally expanded trinucleotide repeat within the huntingtin gene. However, the pathogenesis downstream of the mutant huntingtin gene is complex, involving multiple toxic pathways, including abnormal protein fragmentation and neuroinflammation. The current treatment of HD focuses largely on symptomatic management. This article discusses new, potential disease-modifying therapies that are currently in human clinical trials and preclinical development.
PURPOSE OF REVIEW: This article describes and discusses new potential disease-modifying therapies for Huntington's disease that are currently in human clinical trials as well as promising new therapies in preclinical development. RECENT FINDINGS: Multiple potential disease-modifying therapeutics for HD are in active development, including direct DNA/gene therapies, RNA modulation, and therapies targeted at aberrant downstream pathways. The etiology of Huntington's disease (HD) is well-known as an abnormally expanded trinucleotide repeat within the huntingtin gene. However, the pathogenesis downstream of the mutant huntingtin gene is complex, involving multiple toxic pathways, including abnormal protein fragmentation and neuroinflammation. The current treatment of HD focuses largely on symptomatic management. This article discusses new, potential disease-modifying therapies that are currently in human clinical trials and preclinical development.
Authors: J H Cha; C M Kosinski; J A Kerner; S A Alsdorf; L Mangiarini; S W Davies; J B Penney; G P Bates; A B Young Journal: Proc Natl Acad Sci U S A Date: 1998-05-26 Impact factor: 11.205
Authors: L Mangiarini; K Sathasivam; M Seller; B Cozens; A Harper; C Hetherington; M Lawton; Y Trottier; H Lehrach; S W Davies; G P Bates Journal: Cell Date: 1996-11-01 Impact factor: 41.582
Authors: S W Davies; M Turmaine; B A Cozens; M DiFiglia; A H Sharp; C A Ross; E Scherzinger; E E Wanker; L Mangiarini; G P Bates Journal: Cell Date: 1997-08-08 Impact factor: 41.582
Authors: Nan Wang; Michelle Gray; Xiao-Hong Lu; Jeffrey P Cantle; Sandra M Holley; Erin Greiner; Xiaofeng Gu; Dyna Shirasaki; Carlos Cepeda; Yuqing Li; Hongwei Dong; Michael S Levine; X William Yang Journal: Nat Med Date: 2014-04-28 Impact factor: 53.440
Authors: David Brinvillier; Melissa Barrast; Petra Couderc-Murillo; José Bono-Yagüe; Alain Rousteau; Ana Pilar Gómez Escribano; Marcos V Palmeira-Mello; Antonio Doménech-Carbó; Nady Passe-Coutrin; Muriel Sylvestre; Rafael P Vázquez-Manrique; Gerardo Cebrián-Torrejón Journal: ACS Omega Date: 2022-05-04
Authors: Muhammad Afzal; Nadeem Sayyed; Khalid Saad Alharbi; Sami I Alzarea; Mohammed Salem Alshammari; Fadhel A Alomar; Sattam Khulaif Alenezi; Anwarulabedin Mohsin Quazi; Abdulaziz I Alzarea; Imran Kazmi Journal: Biomolecules Date: 2022-07-23