Initial Biomarker and Clinical Findings from the CANaspire Canavan Disease Gene Therapy Trial: Exploration of Connections between NAA and Disease Severity

被引：0
|
作者：
Eichler, Florian [1 ]
Nagy, Amanda [1 ]
Laforet, Genevieve [2 ]
Burton, Christine [2 ]
Kirby, Kathleen M. [3 ]
Kinane, Bernard [1 ]
Townsend, Elise [1 ]
Kiefer, Michael [1 ]
Leiro, Beth [2 ]
Williams, Rachel [2 ]
Balser, John [4 ]
Kratz, Lisa [5 ]
Shaywitz, Adam [2 ]
Bley, Annette [6 ]
机构：
[1] Massachusetts Gen Hosp, Boston, MA USA
[2] Aspa Therapeut, Palo Alto, CA USA
[3] Viridian Strategies, Barnstable, MA USA
[4] Veristat, Southborough, MA USA
[5] Kennedy Krieger Inst, Baltimore, MD USA
[6] Univ Med Ctr Hamburg Eppendorf, Hamburg, Germany
来源：
MOLECULAR THERAPY | 2023年 / 31卷 / 04期
关键词：
D O I：
暂无
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
358
引用
收藏
页码：193 / 193
页数：1
相关论文
共 50 条
  • [1] CANaspire Trial of Systemic AAV9-mediated Gene Therapy for Canavan Disease: Biomarker, Imaging and Clinical Findings from the Low-dose Cohort
    Laforet, G.
    Fay, A.
    Nagy, A.
    Harmatz, P.
    Mallack, E.
    Burton, C.
    Langer, K.
    Townsend, E.
    Kiefer, M.
    Leiro, B.
    Williams, R.
    Shaywitz, A.
    Bley, A.
    Eichler, F.
    ANNALS OF NEUROLOGY, 2025, 96 : S24 - S25
  • [2] From Gene Therapy to Gene Enhancement: Optimized Pre-Clinical Gene Therapy Transforms Mice with the Severest Canavan Disease Phenotype Into "Supermice"
    Gessler, Dominic J.
    Li, Jia
    Su, Qin
    Matalon, Reuben
    Gao, Guangping
    MOLECULAR THERAPY, 2015, 23 : S198 - S198
  • [3] Exploring Galectin-3 as a Biomarker for Disease Progression and Gene Therapy of Canavan and Other Neurodegenerative Diseases
    Mooney, Morgan
    Guo, Peiyi
    Wang, Jie
    Lotun, Anoushka
    NhatAhn Nguyen
    Gao, Guangping
    Gessler, Dominic J.
    MOLECULAR THERAPY, 2024, 32 (04) : 316 - 317
  • [4] Novel Gene Therapy for XLRS: Initial Findings from a Clinical Trial Showing Visual Improvement
    Zhou, Wenhui
    Shen, Yin
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2024, 65 (07)
  • [5] Fabry disease biomarker evaluation during a five-year gene therapy clinical trial
    Auray-Blais, Christiane
    Lavoie, Pamela
    Martineau, Tristan
    Rupar, Tony
    Barber, Dwayne L.
    Keating, Armand
    Foley, Ronan
    Khan, Aneal
    West, Michael L.
    Medin, Jeffrey A.
    MOLECULAR GENETICS AND METABOLISM, 2025, 144 (02)
  • [6] Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial
    MacLaren, Robert E.
    Groppe, Markus
    Barnard, Alun R.
    Cottriall, Charles L.
    Tolmachova, Tanya
    Seymour, Len
    Clark, K. Reed
    During, Matthew J.
    Cremers, Frans P. M.
    Black, Graeme C. M.
    Lotery, Andrew J.
    Downes, Susan M.
    Webster, Andrew R.
    Seabra, Miguel C.
    LANCET, 2014, 383 (9923): : 1129 - 1137
  • [7] The Interplay between Central Nervous System and Peripheral Organs: The Lesson from the Treatment of Canavan Disease with rAAV Gene Therapy
    Wang, Jie
    Lotun, Anoushka
    Li, Danning
    Nguyen, NhatAnh B.
    Mooney, Morgan
    Gao, Guangping
    Gessler, Dominic J.
    MOLECULAR THERAPY, 2023, 31 (04) : 19 - 19
  • [8] Uncoupling N-acetylaspartate from brain pathology: implications for Canavan disease gene therapy
    von Jonquieres, Georg
    Spencer, Ziggy H. T.
    Rowlands, Benjamin D.
    Klugmann, Claudia B.
    Bongers, Andre
    Harasta, Anne E.
    Parley, Kristina E.
    Cederholm, Jennie
    Teahan, Orla
    Pickford, Russell
    Delerue, Fabien
    Ittner, Lars M.
    Frohlich, Dominik
    McLean, Catriona A.
    Don, Anthony S.
    Schneider, Miriam
    Housley, Gary D.
    Rae, Caroline D.
    Klugmann, Matthias
    ACTA NEUROPATHOLOGICA, 2018, 135 (01) : 95 - 113
  • [9] Uncoupling N-acetylaspartate from brain pathology: implications for Canavan disease gene therapy
    Georg von Jonquieres
    Ziggy H. T. Spencer
    Benjamin D. Rowlands
    Claudia B. Klugmann
    Andre Bongers
    Anne E. Harasta
    Kristina E. Parley
    Jennie Cederholm
    Orla Teahan
    Russell Pickford
    Fabien Delerue
    Lars M. Ittner
    Dominik Fröhlich
    Catriona A. McLean
    Anthony S. Don
    Miriam Schneider
    Gary D. Housley
    Caroline D. Rae
    Matthias Klugmann
    Acta Neuropathologica, 2018, 135 : 95 - 113
  • [10] Relationship between the clinical findings and radiographic severity in Osgood-Schlatter disease
    Hanada, Mitsuru
    Koyama, Hiroshi
    Takahashi, Masaaki
    Matsuyama, Yukihiro
    OPEN ACCESS JOURNAL OF SPORTS MEDICINE, 2012, 3 : 17 - 20
12345