日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

AAV-mediated gene transfer of a novel microdystrophin ameliorates pathology and enhances muscle function in a mouse model of DMD.

AAV介导的新型微型肌营养不良蛋白基因转移可改善DMD小鼠模型的病理并增强肌肉功能。

Owusu Lawrence, Kim SunJung, Patel Hiren, Foltz Steven, Chan Gary N Y, Kim Kwi Hye, Kopen Aaron, Yang Lin, Lawlor Michael W, Buss Nicholas, Cunningham Justine J, Liu Ye, Danos Olivier, Fiscella Michele

Myosin inhibition partially rescues the myofiber proteome in X-linked myotubular myopathy

肌球蛋白抑制可部分挽救X连锁肌管性肌病中的肌纤维蛋白质组。

Gerlach Melhedegaard, Elise; Rostedt, Fanny; Gineste, Charlotte; Seaborne, Robert Ae; Dugdale, Hannah F; Belhac, Vladimir; Zanoteli, Edmar; Lawlor, Michael W; Mack, David L; Wallgren-Pettersson, Carina; Hessel, Anthony L; Jungbluth, Heinz; Laporte, Jocelyn; Saito, Yoshihiko; Nishino, Ichizo; Ochala, Julien; Laitila, Jenni

Corrigendum: Enhancing AAV-microdystrophin gene therapy after repeat dosing by blocking phagocytosis

更正:通过阻断吞噬作用增强重复给药后的AAV-微型肌营养不良蛋白基因治疗效果

Spathis, Rita; Kuriplach, Deeva Robles; Narvesen, Sabrina; Eybs, Matthew; Huang, Karen; Torres, Steven; King, Madison; Bagley, Elizabeth; Elustondo, Pia; Lawlor, Michael W; Nagaraju, Kanneboyina; Morales, Melissa

Enhancing AAV-microdystrophin gene therapy after repeat dosing by blocking phagocytosis

通过阻断吞噬作用增强重复给药后的AAV-微型肌营养不良蛋白基因治疗效果

Spathis, Rita; Kuriplach, Deeva Robles; Narvesen, Sabrina; Eybs, Matthew; Huang, Karen; Torres, Steven; King, Madison; Bagley, Elizabeth; Elustondo, Pia; Lawlor, Michael W; Nagaraju, Kanneboyina; Morales, Melissa

Pathogenic TNNT1 variants are associated with aberrant thin filament compliance and myofibre hyper-contractility.

致病性 TNNT1 变异与细丝顺应性异常和肌纤维过度收缩有关

Laitila Jenni, Lewis Christopher T A, Hessel Anthony L, Primiano Guido, Hernandez-Lain Aurelio, Fiorillo Chiara, Lawlor Michael W, Ottenheijm Coen A C, Jungbluth Heinz, Man Ka Fu, Fornili Arianna, Ochala Julien

Establishment of normal myofiber size distribution in children and young adults

建立儿童和青少年正常肌纤维大小分布

Lawlor, Michael W; Margeta, Marta; Jones, Karra A; Schoser, Benedikt; Cotter, Jennifer A; Rajaram, Veena; Moore, Steven A; Prom, Mariah J; Beatka, Margaret; Ott, Emily; Slick, Rebecca A; Collins, Michael P; Esselman, Ann; Shahnoor, Nazima; Danielson, Susan; Meng, Hui; Varfaj, Fatbardha; Prasad, Suyash; Rico, Salvador; Lee, Jun; Kumar, Suresh N; Dressman, Heather Gordish

Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO study

针对患有 X 连锁肌管性肌病儿童的基因疗法:ASPIRO 研究出版物的通俗易懂的摘要

Shieh, Perry B; Hughes, Wendy; Wood, Marie; Beggs, Alan H; Lawlor, Michael W; Coats, Julie; Varfaj, Fatbardha; Graham, Robert J; Kuntz, Nancy L; Dowling, James J; Müller-Felber, Wolfgang; Bönnemann, Carsten G; Buj Bello, Ana; Servais, Laurent; MacBean, Vicky; Muntoni, Francesco; Foley, A Reghan; Blaschek, Astrid; James, Emma S; Seferian, Andreea; Alfano, Lindsay N; Duong, Tina; Noursalehi, Mojtaba; Miller, Weston; Lee, Jun; Prasad, Suyash; Rico, Salvador

Characterization of NEB pathogenic variants in patients reveals novel nemaline myopathy disease mechanisms and omecamtiv mecarbil force effects

对患者中NEB致病变异的特征分析揭示了新的线状肌病疾病机制和omecamtiv mecarbil的作用机制。

Karimi, Esmat; Gohlke, Jochen; van der Borgh, Mila; Lindqvist, Johan; Hourani, Zaynab; Kolb, Justin; Cossette, Stacy; Lawlor, Michael W; Ottenheijm, Coen; Granzier, Henk

Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy

在杜氏肌营养不良症GRMD模型中评估全身性AAV-微型肌营养不良蛋白基因治疗

Birch, Sharla M; Lawlor, Michael W; Conlon, Thomas J; Guo, Lee-Jae; Crudele, Julie M; Hawkins, Eleanor C; Nghiem, Peter P; Ahn, Mihye; Meng, Hui; Beatka, Margaret J; Fickau, Brittany A; Prieto, Juan C; Styner, Martin A; Struharik, Michael J; Shanks, Courtney; Brown, Kristy J; Golebiowski, Diane; Bettis, Amanda K; Balog-Alvarez, Cynthia J; Clement, Nathalie; Coleman, Kirsten E; Corti, Manuela; Pan, Xiufang; Hauschka, Stephen D; Gonzalez, J Patrick; Morris, Carl A; Schneider, Joel S; Duan, Dongsheng; Chamberlain, Jeffrey S; Byrne, Barry J; Kornegay, Joe N

Effects of Regulatory T Cell Depletion in BALB/c Mice Infected with Low Doses of Borrelia burgdorferi

低剂量伯氏疏螺旋体感染BALB/c小鼠后调节性T细胞耗竭的影响

Santiago, Kaitlyn N; Kozlik, Tanya; Liedhegner, Elizabeth S; Slick, Rebecca A; Lawlor, Michael W; Nardelli, Dean T