Results 61 to 70 of about 304,862 (304)

Aminoglycoside Enhances the Delivery of Antisense Morpholino Oligonucleotides In Vitro and in mdx Mice

Molecular Therapy: Nucleic Acids, 2019
Antisense oligonucleotide (AO) therapy has been the specific treatment for Duchenne muscular dystrophy, with ongoing clinical trials. However, therapeutic applications of AOs remain limited, particularly because of the lack of efficient cellular delivery
Mingxing Wang, Bo Wu, Sapana N. Shah, Peijuan Lu, Qilong Lu   +4 more
doaj   +1 more source

The muscular dystrophies

BMJ, 1998
The muscular dystrophies are inherited myogenic disorders characterised by progressive muscle wasting and weakness of variable distribution and severity. They can be subdivided into several groups, including congenital forms, in accordance with the distribution of predominant muscle weakness: Duchenne and Becker; Emery-Dreifuss; distal ...
openaire   +4 more sources

Revertant fibres and dystrophin traces in Duchenne muscular dystrophy: Implication for clinical trials [PDF]

, 2010
Duchenne muscular dystrophy (DMD) is characterised by the absence of dystrophin in muscle biopsies, although residual dystrophin can be present, either as dystrophin-positive (revertant) fibres or traces.
Main, M, Morgan, JE, Straub, V, Feng, L, Sewry, CA, Muntoni, F, Lehovsky, J, Arechavala-Gomeza, V, Kinali, M, Hunt, D, Guglieri, M, Bushby, K, Edge, G   +12 more
core  

Intelligent Micro/Nanorobots for Targeted Interventional Therapy: From Bench to Clinic

Advanced Robotics Research, EarlyView.
Zirui Zhang et al. reviewed the application and challenges of mobile nanomachines in interventional therapy. By converting exogenous energy, including chemical, magnetic, optical, and ultrasonic sources, into mechanical forces, micro/nanorobots (MNRs) enable precise actuation at unprecedented scales. Evolving far beyond traditional drug delivery, these
Zirui Zhang, Yu Dong, Yongsheng Du, Rui Gao, Lin Zhang, Fei Tong   +5 more
wiley   +1 more source

AAV-DJ is superior to AAV9 for targeting brain and spinal cord, and de-targeting liver across multiple delivery routes in mice

Journal of Translational Medicine
Highly efficient adeno associated viruses (AAVs) targeting the central nervous system (CNS) are needed to deliver safe and effective therapies for inherited neurological disorders.
Monika Chauhan, Audrey L. Daugherty, Fatemeh (Ellie) Khadir, Ozgun F. Duzenli, Alexandra Hoffman, Jennifer A. Tinklenberg, Peter B. Kang, George Aslanidi, Christina A. Pacak   +8 more
doaj   +1 more source

Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne muscular dystrophy

BMC Pediatrics, 2019
Background Adolescents with DMD treated with chronic high dose GC therapy typically have profound pubertal delay. Testosterone, the main circulating androgen in men, promotes virilisation and growth with associated accrual of fat-free muscle mass and ...
C. L. Wood, T. D Cheetham, K. G Hollingsworth, M. Guglieri, Y. Ailins-Sahun, S. Punniyakodi, A. Mayhew, V. Straub   +7 more
doaj   +1 more source

iMyoblasts for ex vivo and in vivo investigations of human myogenesis and disease modeling

eLife, 2022
Skeletal muscle myoblasts (iMyoblasts) were generated from human induced pluripotent stem cells (iPSCs) using an efficient and reliable transgene-free induction and stem cell selection protocol.
Dongsheng Guo, Katelyn Daman, Jennifer JC Chen, Meng-Jiao Shi, Jing Yan, Zdenka Matijasevic, Amanda M Rickard, Monica H Bennett, Alex Kiselyov, Haowen Zhou, Anne G Bang, Kathryn R Wagner, René Maehr, Oliver D King, Lawrence J Hayward, Charles P Emerson Jr   +15 more
doaj   +1 more source

Superpulsed low-level laser therapy protects skeletal muscle of mdx mice against damage, inflammation and morphological changes delaying dystrophy progression. [PDF]

, 2014
Aim: To evaluate the effects of preventive treatment with low-level laser therapy (LLLT) on progression of dystrophy in mdx mice. Methods: Ten animals were randomly divided into 2 experimental groups treated with superpulsed LLLT (904 nm, 15 mW, 700 Hz ...
de Carvalho, Paulo de Tarso Camillo, Leal-Junior Ernesto Cesar Pinto, Lucio Frigo, Patrícia de Almeida, Lopes-Martins, Rodrigo Alvaro Brandao, de Almeida, Patrícia, Joensen Jon, Tomazoni, Shaiane Silva, Tomazoni Shaiane Silva, Bjordal, JM, Leal, Ernesto Cesar Pinto Jr, Leal-Junior, EC, Lopes-Martins, R, Joensen, J, Johnson, Mark I., Lopes-Martins Rodrigo Álvaro Brandão, Frigo, Lúcio, Paulo de Tarso Camillo de Carvalho, Tomazoni, SS, Bjordal, Jan Magnus, Bjordal Jan Magnus, Johnson, MI, Jon Joensen, Rodrigo Álvaro Brandão Lopes-Martins, de Carvalho, PT, Mark I Johnson, Ernesto Cesar Pinto Leal-Junior, Joensen, Jon, de Almeida, P, Frigo, L, Jan Magnus Bjordal, Frigo Lucio, Johnson Mark I., de Carvalho Paulo de Tarso Camillo, de Almeida Patrícia, Shaiane Silva Tomazoni   +35 more
core   +2 more sources

Innate Immunocompetent hiPSC‐Derived Neurospheroids Capture Early CNS Responses to rAAV

Advanced Science, EarlyView.
Knowledge of human CNS immune responses to AAV‐based gene therapies remains limited due to the lack of immune‐competent human models. Here, a hiPSC‐derived 3D neuroimmune platform integrating neurospheroids and microglia is established using stirred‐tank bioreactors.
Catarina M. Gomes, Gabriela Silva, Mafalda Aleixo, Marta Gomes, Daniel Simão, Stephan J. Holtkamp, Diana D. Lobo, Pradeep Harish, Rosalind Jenkins, Lekh N. Dahal, Rui J. Nobre, Luís de Pereira de Almeida, Mark Trautwein, Paula M. Alves, Catarina Brito   +14 more
wiley   +1 more source

Chiral Nanoparticles Suppress Inflammatory Infiltration to Promote Extracellular Matrix Remodeling for Ectopia Lentis Therapy

Advanced Science, EarlyView.
L‐cysteine‐configured chiral polyurethane nanoparticles suppress ocular inflammation and reduce extracellular matrix (ECM) degradation in ectopia lentis by regulating macrophages polarization and inhibiting nuclear factor kappa B signaling pathway. By promoting zonular fiber‐associated protein restoration and tissue repair, this minimally invasive ...
Yinuo Wen, Yan Yang, Haihan Gao, Yan Liu, Xinyue Wang, Linghao Song, Ruohong Li, Ao Miao, Jie Xu, Zhennan Zhao, Dan Li, Shenjie Peng, Linzhao Li, Min Zhang, Yonghang Liu, Liren Wang, Tianhui Chen, Yongxiang Jiang   +17 more
wiley   +1 more source