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Quantitative relationship between tominersen concentrations in cerebrospinal fluid and biomarker changes in Huntington's disease patients Yamamoto, Yumi; Silber Baumann, Hanna E.; Bjornsson, Marcus; Grimsey, Paul; Hawellek, David J.; Anderson, Karen; Leavitt, Blair R.; Landwehrmeyer, Bernhard G.; Tabrizi, Sarah; Mccolgan, Peter; Wild, Edward J.; Sanwald Ducray, Patricia Share Save
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Challenges and advances for huntingtin detection in cerebrospinal fluid: in support of relative quantification Harding, Rachel J.; Xie, Yuanyun; Caron, Nicholas S.; Findlay-Black, Hailey; Lyu, Caroline; Potluri, Nalini; Chandrasekaran, Renu; Hayden, Michael R.; Leavitt, Blair R.; Langbehn, Douglas R.; Southwell, Amber L. Share Save
Restricting lysine normalizes toxic catabolites associated with ALDH7A1 deficiency in cells and mice Johal, Amritpal S.; Al-Shekaili, Hilal H.; Abedrabbo, Muna; Kehinde, Abisola Z.; Towriss, Morgan; Koe, Jessica C.; Hewton, Keeley G.; Thomson, Sarah B.; Ciernia, Annie, V; Leavitt, Blair; Parker, Seth J. Share Save
New MiniPromoter Ple389 (ADORA2A) drives selective expression in medium spiny neurons in mice and non-human primates Gomes, Alissandra de Moura; Petkau, Terri L.; Korecki, Andrea J.; Fornes, Oriol; Galvan, Adriana; Lu, Ge; Hill, Austin M.; Lam, Siu Ling; Yao, Anqi; Farkas, Rachelle A.; Wasserman, Wyeth W.; Smith, Yoland; Simpson, Elizabeth M.; Leavitt, Blair R. Share Save
Elevated plasma and CSF neurofilament light chain concentrations are stabilized in response to mutant huntingtin lowering in the brains of Huntington's disease mice Caron, Nicholas S.; Byrne, Lauren M.; Lemarie, Fanny L.; Bone, Jeffrey N.; Aly, Amirah E. -E.; Ko, Seunghyun; Anderson, Christine; Casal, Lorenzo L.; Hill, Austin M.; Hawellek, David J.; McColgan, Peter; Wild, Edward J.; Leavitt, Blair R.; Hayden, Michael R. Share Save
Tominersen in Adults with Manifest Huntington's Disease McColgan, Peter; Thobhani, Alpa; Boak, Lauren; Schobel, Scott A.; Nicotra, Alessia; Palermo, Giuseppe; Trundell, Dylan; Zhou, Julian; Schlegel, Valerie; Ducray, Patricia Sanwald; Hawellek, David J.; Dorn, Jonas; Simillion, Cedric; Lindemann, Michael; Wheelock, Vicki; Durr, Alexandra; Anderson, Karen E.; Long, Jeffrey D.; Wild, Edward J.; Landwehrmeyer, G. Bernhard; Leavitt, Blair R.; Tabrizi, Sarah J.; Doody, Rachelle Share Save
Antisense oligonucleotides targeting the miR-29b binding site in the GRN mRNA increase progranulin translation Aggarwal, Geetika; Banerjee, Subhashis; Jones, Spencer A.; Benchaar, Yousri; Belanger, Jasmine; Sevigny, Myriam; Smith, Denise M.; Niehoff, Michael L.; Pavlack, Monica; Vera, Ian Mitchelle S. de; Petkau, Terri L.; Leavitt, Blair R.; Ling, Karen; Jafar-Nejad, Paymaan; Rigo, Frank; Morley, John E.; Farr, Susan A.; Dutchak, Paul A.; Sephton, Chantelle F.; Nguyen, Andrew D. Share Save
Genetic topography and cortical cell loss in Huntington's disease link development and neurodegeneration Estevez-Fraga, Carlos; Altmann, Andre; Parker, Christopher S.; Scahill, Rachael, I; Costa, Beatrice; Chen, Zhongbo; Manzoni, Claudia; Zarkali, Angeliki; Durr, Alexandra; Roos, Raymund A. C.; Landwehrmeyer, Bernhard; Leavitt, Blair R.; Rees, Geraint; Tabrizi, Sarah J.; McColgan, Peter Share Save
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Antisense oligonucleotides targeting microRNA binding sites in the GRN mRNA increase progranulin translation: potential therapeutic strategy for frontotemporal dementia Jones, Spencer; Aggarwal, Geetika; Benchaar, Yousri; Belanger, Jasmine; Sevigny, Myriam; Smith, Denise; Niehoff, Michael; de Vera, Ian Mitchelle; Petkau, Terri; Leavitt, Blair; Ling, Karen; Jafar-Nejad, Paymaan; Rigo, Frank; Morley, John; Nguyen, Andrew Share Save
Huntingtin Decreases Susceptibility to a Spontaneous Seizure Disorder in FVN/B Mice Van Raamsdonk, Jeremy M.; Al-Shekaili, Hilal H.; Wagner, Laura; Bredy, Tim W.; Chan, Laura; Pearson, Jacqueline; Schwab, Claudia; Murphy, Zoe; Devon, Rebecca S.; Lu, Ge; Kobor, Michael S.; Hayden, Michael R.; Leavitt, Blair R. Share Save
AAV5-miHTT-mediated huntingtin lowering improves brain health in a Huntington's disease mouse model Thomson, Sarah B.; Stam, Anouk; Brouwers, Cynthia; Fodale, Valentina; Bresciani, Alberto; Vermeulen, Michael; Mostafavi, Sara; Petkau, Terri L.; Hill, Austin; Yung, Andrew; Russell-Schulz, Bretta; Kozlowski, Piotr; MacKay, Alex; Ma, Da; Beg, Mirza Faisal; Evers, Melvin M.; Valles, Astrid; Leavitt, Blair R. Share Save
Cerebrospinal fluid biomarkers for assessing Huntington disease onset and severity Caron, Nicholas S.; Haqqani, Arsalan S.; Sandhu, Akshdeep; Aly, Amirah E.; Findlay Black, Hailey; Bone, Jeffrey N.; McBride, Jodi L.; Abulrob, Abedelnasser; Stanimirovic, Danica; Leavitt, Blair R.; Hayden, Michael R. Share Save
The Safety of Deutetrabenazine for Chorea in Huntington Disease: An Open-Label Extension Study Frank, Samuel; Testa, Claudia; Edmondson, Mary C.; Goldstein, Jody; Kayson, Elise; Leavitt, Blair R.; Oakes, David; O'Neill, Christine; Vaughan, Christina; Whaley, Jacquelyn; Gross, Nicholas; Gordon, Mark Forrest; Savola, Juha-Matti Share Save
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