Results 61 to 70 of about 23,636 (243)

Increased neointimal thickening in dystrophin-deficient mdx mice.

PLoS ONE, 2012
BackgroundThe dystrophin gene, which is mutated in Duchenne muscular dystrophy (DMD), encodes a large cytoskeletal protein present in muscle fibers. While dystrophin in skeletal muscle has been extensively studied, the function of dystrophin in vascular ...
Uwe Rauch, Annelie Shami, Feng Zhang, Virginie Carmignac, Madeleine Durbeej, Anna Hultgårdh-Nilsson   +5 more
doaj   +1 more source

Importance of proteins and mitochondrial changes as freshness indicators in fish muscle post‐mortem

Journal of the Science of Food and Agriculture, EarlyView.
Abstract Evaluating protein and mitochondrial alterations post‐mortem can contribute to determining correlations between fish‐processing parameters and ultimate fish muscle quality. The myofibrillar protein alteration during rigor mortis directly affects the texture of fish muscle.
Nima Hematyar, Tomas Policar, Turid Rustad   +2 more
wiley   +1 more source

Differential expression of dystrophin, utrophin and dystrophin‐associated proteins in peripheral nerve [PDF]

FEBS Letters, 1993
The dystrophin‐glycoprotein complex is a novel laminin receptor in skeletal muscle. Dystrophin‐associated proteins are comprised of an extracellular glycoprotein of 156 kDa (156DAG), transmembrane glycoproteins of 50 kDa (50DAG), 43 kDa (43DAG) and 35 kDa (35DAG), and a cytoskeletal protein of 59 kDa (59DAP).
Teruo Shimizu, Kiichiro Matsumura, Kevin P. Campbell, Hiroki Yamada   +3 more
openaire   +3 more sources

Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene

Molecular Therapy: Nucleic Acids, 2012
Protein-truncating mutations in the dystrophin gene lead to the progressive muscle wasting disorder Duchenne muscular dystrophy, whereas in-frame deletions typically manifest as the milder allelic condition, Becker muscular dystrophy.
Sue Fletcher, Carl F. Adkin, Penny Meloni, Brenda Wong, Francesco Muntoni, Ryszard Kole, Clayton Fragall, Kane Greer, Russell Johnsen, Steve D. Wilton   +9 more
doaj   +1 more source

A model‐informed clinical trial simulation tool with a graphical user interface for Duchenne muscular dystrophy

CPT: Pharmacometrics &Systems Pharmacology, EarlyView.
Abstract Quantitative model‐based clinical trial simulation tools play a critical role in informing study designs through simulation before actual execution. These tools help drug developers explore various trial scenarios in silico to select a clinical trial design to detect therapeutic effects more efficiently, therefore reducing time, expense, and ...
Jongjin Kim, Juan Francisco Morales, Sanghoon Kang, Marian Klose, Rebecca J. Willcocks, Michael J. Daniels, Ramona Belfiore‐Oshan, Glenn A. Walter, William D. Rooney, Krista Vandenborne, Sarah Kim   +10 more
wiley   +1 more source

A computational tool to optimize clinical trial parameter selection in Duchenne muscular dystrophy: A practical guide and case studies

CPT: Pharmacometrics &Systems Pharmacology, EarlyView.
Abstract Duchenne muscular dystrophy (DMD), a rare pediatric disease, presents numerous challenges when designing clinical trials, mainly due to the scarcity of available trial participants and the heterogeneity of disease progression. A quantitative clinical trial simulator (CTS) has been developed based on previously published five disease ...
Jordan Wilk, Varun Aggarwal, Mike Pauley, Diane Corey, Daniela J. Conrado, Karthik Lingineni, Juan Francisco Morales, Deok Yong Yoon, Yi Zhang, Zihan Cui, Jackson Burton, Jane Larkindale, Shu Chin Ma, Collin Hovinga, Terina Martinez, Klaus Romero, Ramona Belfiore‐Oshan, Sarah Kim, the Duchenne Regulatory Science Consortium (D‐RSC) and the CINRG DNHS investigators   +18 more
wiley   +1 more source

Direct binding of Torpedo syntrophin to dystrophin and the 87 kDa dystrophin homologue [PDF]

FEBS Letters, 1995
Syntrophin, a 58‐kDa membrane‐associated protein, is one component of a protein complex associated with dystrophin and other members of the dystrophin family, including the 87‐kDa homologue (87K protein). To characterize interactions between syntrophin and 87K protein, we used an in vitro overlay binding assay.
Tim Dwyer, Tim Dwyer, Stanley C. Froehner   +2 more
openaire   +2 more sources

A Population Pharmacokinetic Model to Inform Extension of the Eteplirsen Dosing Regimen Across the Broad DMD Population

CPT: Pharmacometrics &Systems Pharmacology, EarlyView.
ABSTRACT Duchenne muscular dystrophy (DMD) is characterized by progressive, irreversible muscle damage that usually leads to premature death from cardiac or respiratory failure. Eteplirsen is a phosphorodiamidate morpholino oligomer and the first antisense oligonucleotide (ASO) approved for the treatment of patients with exon 51 skip‐amenable DMD. This
Yogesh Patel, Larry Orogun, Nicole Yocum, Louise R. Rodino‐Klapac, Lilly East   +4 more
wiley   +1 more source

A sensitive, reproducible and objective immunofluorescence analysis method of dystrophin in individual fibers in samples from patients with duchenne muscular dystrophy.

PLoS ONE, 2014
Duchenne muscular dystrophy (DMD) is characterized by the absence or reduced levels of dystrophin expression on the inner surface of the sarcolemmal membrane of muscle fibers.
Chantal Beekman, Jessica A Sipkens, Janwillem Testerink, Stavros Giannakopoulos, Dyonne Kreuger, Judith C van Deutekom, Giles V Campion, Sjef J de Kimpe, Afrodite Lourbakos   +8 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