Results 121 to 130 of about 59,671 (341)

Dystrophinopathy in the paravertebral muscle of adolescent idiopathic scoliosis: a prospective case-control study in China [PDF]

Asian Spine Journal
Study Design A prospective case-control study. Purpose This prospective case-control study aimed to analyze the paravertebral muscle changes in patients with adolescent idiopathic scoliosis (AIS) and determine paravertebral myopathological changes ...
Junyu Li, Danfeng Zheng, Zekun Li, Jiaxi Li, Zexi Yang, Xiang Zhang, Yingshuang Zhang, Miao Yu   +7 more
doaj   +1 more source

Nature Inspired Delivery Vehicles for CRISPR‐Based Genome Editing

Small, EarlyView.
The review highlights nature‐inspired nanocarriers for CRISPR delivery, emphasizing viral vectors, extracellular vesicles, liposomes, and lipid nanoparticles. It discusses their roles in improving specificity, minimizing immunogenicity, and overcoming barriers in genome editing. Recent advancements, challenges, and therapeutic applications are explored,
Elizabeth Maria Clarissa, Mamata Karmacharya, Hyunmin Choi, Sumit Kumar, Yoon‐Kyoung Cho   +4 more
wiley   +1 more source

Thermodynamic stability, unfolding kinetics, and aggregation of the N-terminal actin-binding domains of utrophin and dystrophin. [PDF]

, 2012
Muscular dystrophy (MD) is the most common genetic lethal disorder in children. Mutations in dystrophin trigger the most common form of MD, Duchenne, and its allelic variant Becker MD.
Aaartsma-Rus, Ahmad, Badorff, Barghorn, Belli, Blake, Blake, Chamberlain, Chenna, Chi, Cotton, Courdier-Fruh, Daragan, Deconinck, Deconinck, Delaglio, Deol, Ebihara, Emery, Ervasti, Espargaró, Fairclough, Fisher, Foster, Galkin, Galkin, Gilbert, Gimona, Grady, Gramolini, Greenfield, Gregorevic, Gregorevic, Hamed, Helliwell, Henderson, Henderson, Hoffman, Ishikawa-Sakurai, Ito, Kahana, Karpati, Keep, Khurana, Khurana, Kim, Klunk, Knuesel, Krishna, Lakowicz, Le Rumeur, Legardinier, Lehman, Levine-III, Li, Lin, Love, Love, Mallela, Matsumura, Mayer, Mirza, Miura, Moores, Muir, Muntoni, Myers, Nagarajan, Norwood, Odom, Odom, Ohlendieck, Orlova, Park, Parsell, Pastoret, Pearce, Phelps, Pons, Prior, Prior, Prochniewicz, Rafael, Rafael, Roberts, Roberts, Roberts, Ruschak, Ruszczak, Rybakova, Rybakova, Santoro, Santoro, Scott, Shatsky, Sieb, Singh, Singh, Sitnik, Sonnemann, Squire, Sutherland-Smith, Tanabe, Tinsley, Tinsley, Tinsley, van Deutekom, Wakefield, Wang, Wang, Winder, Winder   +111 more
core   +1 more source

Lipid Nanoparticles for Delivery of CRISPR Gene Editing Components

Small Methods, EarlyView.
The review presents a comprehensive overview of each component of lipid nanoparticles（LNPs）and their effects on editing efficiency. It specifically highlights strategies for achieving non‐liver delivery, aiming for broader applications in gene editing. Furthermore, this review summarizes the applications of LNPs in gene editing and offers insights for ...
Fan Wu, Nei Li, Yudian Xiao, Rohan Palanki, Hannah Yamagata, Michael J. Mitchell, Xuexiang Han   +6 more
wiley   +1 more source

Silencing disease genes in the laboratory and the clinic

, 2011
Synthetic nucleic acids are commonly used laboratory tools for modulating gene expression and have the potential to be widely used in the clinic. Progress towards nucleic acid drugs, however, has been slow and many challenges remain to be overcome before
Corey, David R., Watts, Jonathan K.
core   +1 more source

Dystrophin S3059 phosphorylation partially attenuates denervation atrophy in mouse tibialis anterior muscles

Physiological Reports
The dystrophin protein has well‐characterized roles in force transmission and maintaining membrane integrity during muscle contraction. Studies have reported decreased expression of dystrophin in atrophying muscles during wasting conditions, and that ...
Kristy Swiderski, Timur Naim, Jennifer Trieu, Annabel Chee, Marco J. Herold, Andrew J. Kueh, Craig A. Goodman, Paul Gregorevic, Gordon S. Lynch   +8 more
doaj   +1 more source

A role for the dystrophin-glycoprotein complex as a transmembrane linker between laminin and actin

Journal of Cell Biology, 1993
The dystrophin-glycoprotein complex was tested for interaction with several components of the extracellular matrix as well as actin. The 156-kD dystrophin-associated glycoprotein (156-kD dystroglycan) specifically bound laminin in a calcium-dependent ...
J. Ervasti, K. Campbell
semanticscholar   +1 more source

CRISPR Technology in Disease Management: An Updated Review of Clinical Translation and Therapeutic Potential

Cell Proliferation, EarlyView.
CRISPR‐Cas systems offer transformative genome editing capabilities for precise manipulation of cellular genes. This enables two main therapeutic avenues: ex vivo modification of patient cells for re‐transplantation or direct in vivo gene targeting via advanced delivery methods.
Bahareh Farasati Far, Marziyeh Akbari, Mohammad Amin Habibi, Morteza Katavand, Sherko Nasseri   +4 more
wiley   +1 more source

Height, weight, and body mass index trajectories and their correlation with functional outcome assessments in boys with Duchenne muscular dystrophy

Developmental Medicine &Child Neurology, EarlyView.
The analysis of height, weight, and BMI z‐score trajectories in boys with DMD from the FOR‐DMD study showed that higher baseline height was associated with slower subsequent growth, and older age with greater weight gain after glucocorticoid initiation.
Marianela Schiava, Utkarsh J. Dang, Claire Wood, Sze Choong Wong, Leanne M. Ward, Robert Muni Lofra, Anna Mayhew, William B. Martens, Stephanie Gregory, Robert C. Griggs, Michela Guglieri, on behalf of the FOR‐DMD Muscle Study Group, Adnan Manzur, Amos Rachel, Andrea Gangfuss, Anne Marie Childs, Ashutosh Kumar, Barbara E. Herr, Basil T. Darras, Chris Speed, Craig Campbell, Craig M. McDonald, Ekkehard Wilichowski, Elaine McColl, Elena Pegoraro, Emma Ciafaloni, Erik K. Henricson, Federica Ricci, Gian Luca Vita, Giovanni Baranello, Giuseppe Vita, Helen Roper, Hugh J. McMillan, Iain A. Horrocks, Imelda Hughes, James F. Howard, Janbernd Kirschner, Jay J. Han, Jean K. Mah, Jeffrey M. Statland, Jennifer Wilkinson, Kate Bushby, Kathleen O’Reardon, Kevin M. Flanigan, Kimberly A. Hart, Leslie Morrison, Lorenzo Maggi, Luca Bello, Maja von der Hagen, Mary W. Brown, Mathula Thangarajh, Matthew Wicklund, Michael P. McDermott, Michelle Eagle, Monika Krzesniak‐Swinarska, Nancy L. Kuntz, Nanette Joyce, Perry B. Shieh, Peter B. Kang, Rabi Tawil, Richard J. Barohn, Richard S. Finkel, Russell J. Butterfield, Stefan Spinty, Taeun Chang, Tiziana E. Mongini, Tracey Willis, Ulrike Schara‐Schmidt, Volker Straub, W. Bryan Burnette, Wendy M. King, Zoya Alhaswani   +71 more
wiley   +1 more source

Agrin-induced acetylcholine receptor clustering in mammalian muscle requires tyrosine phosphorylation. [PDF]

, 1996
Agrin is thought to be the nerve-derived factor that initiates acetylcholine receptor (AChR) clustering at the developing neuromuscularjunction. We have investigated the signaling pathway in mouse C2 myotubes and report that agrin induces a rapid but ...
Deiner, M, Ferns, M, Hall, Z
core