[HTML][HTML] Huntingtin lowering strategies for disease modification in Huntington's disease

SJ Tabrizi, R Ghosh, BR Leavitt - Neuron, 2019 - cell.com
Huntington’s disease is caused by an abnormally … of function in the mutant huntingtin (mHTT)
protein. There are currently no disease-modifying therapies available, but approaches that …

Huntington disease: new insights into molecular pathogenesis and therapeutic opportunities

SJ Tabrizi, MD Flower, CA Ross, EJ Wild - Nature Reviews Neurology, 2020 - nature.com
Huntington disease (HD) is a neurodegenerative disease caused by CAG repeat expansion
in the huntingtin gene (HTT) and involves a complex web of pathogenic mechanisms. …

Targeting huntingtin expression in patients with Huntington's disease

SJ Tabrizi, BR Leavitt… - … England Journal of …, 2019 - Mass Medical Soc
… expansion in the huntingtin (HTT) gene, which encodes huntingtin protein (HTT). … Huntington’s
disease, we sought to inhibit HTT expression and thus directly target the primary disease

Huntingtin-lowering therapies for Huntington disease: a review of the evidence of potential benefits and risks

BR Leavitt, HB Kordasiewicz, SA Schobel - JAMA neurology, 2020 - jamanetwork.com
… Row 2 represents the common genotype of individuals with Huntington disease (HD) … the
Huntington gene (HTT), which are estimated to have less than 50% of normal HTT function and …

Huntington's disease: New frontiers in therapeutics

L Pan, A Feigin - Current Neurology and Neuroscience Reports, 2021 - Springer
… 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 …

Potential disease-modifying therapies for Huntington's disease: lessons learned and future opportunities

SJ Tabrizi, C Estevez-Fraga… - The Lancet …, 2022 - thelancet.com
… for the development of disease-modifying … the function of the wild-type huntingtin protein
through non-allele-selective compared with allele-selective approaches targeting the disease-…

[HTML][HTML] New avenues for the treatment of Huntington's disease

A Kim, K Lalonde, A Truesdell, P Gomes Welter… - International journal of …, 2021 - mdpi.com
… An attractive alternative approach is gene therapy using viral vectors containing the
BDNF gene, which allows for the constant and local production of the protein by the desired …

[HTML][HTML] Huntington disease: Advances in the understanding of its mechanisms

EM Gatto, NG Rojas, G Persi, JL Etcheverry… - … & Related Disorders, 2020 - Elsevier
… exon 1 of the gene coding for huntingtin, placed in the short arm of … disease are unclear
and complex. Here, we review some of the currently known functions of the wild-type huntingtin

[HTML][HTML] Therapeutic advances for Huntington's disease

A Kumar, V Kumar, K Singh, S Kumar, YS Kim, YM Lee… - Brain sciences, 2020 - mdpi.com
approaches include targeting the mutant huntingtin (mHTT) … role in other inherited and
neurological disorders. In this review, we aim to discuss current therapeutic research approaches

Mutant huntingtin is cleared from the brain via active mechanisms in Huntington disease

NS Caron, R Banos, C Yanick, AE Aly… - Journal of …, 2021 - Soc Neuroscience
Huntington disease (HD) is a neurodegenerative disease caused by a CAG trinucleotide
repeat expansion in the huntingtin (HTT) gene. Therapeutics that lower HTT have shown …