[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 …
protein. There are currently no disease-modifying therapies available, but approaches that …
Huntington disease: new insights into molecular pathogenesis and therapeutic opportunities
… 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. …
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 …
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 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 …
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-…
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 …
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 …
and complex. Here, we review some of the currently known functions of the wild-type huntingtin …
[HTML][HTML] Therapeutic advances for Huntington's disease
… 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 …
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
… 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 …
repeat expansion in the huntingtin (HTT) gene. Therapeutics that lower HTT have shown …