This study examines how lipophilic-modified antisense oligonucleotides (ASOs) enhance exon skipping in Duchenne muscular dystrophy (DMD). The results show improved cellular uptake, nanoparticle formation, and higher exon-skipping efficiency, making these ASOs promising therapeutic candidates.

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Duchenne muscular dystrophy (DMD) is a genetic disorder leading to muscle degeneration. This study introduces a predictive computational model for exon skipping therapy, achieving 89% accuracy for PMOs and 76% accuracy for 2’-O-Methyl RNA, aiding in oligonucleotide selection.

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In this article, I emphasize a concept of kindness cherished in Persian poetry and how this belief has strongly influenced my professional and motivational goals. Additionally, the importance of the lives of children with Duchenne Muscular Dystrophy (DMD) is discussed in my beliefs.

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While reviewing old notes, I found a powerful biblical reminder from Proverbs 24:33-34, which warns that idleness leads to poverty. This verse emphasizes the importance of diligence, perseverance, and productivity. Some sayings remain timeless, urging us to stay committed to our work and strive for success. It serves as a call to action, encouraging us to embrace discipline and purpose in our daily lives.

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Nikilab.net has surpassed 10,000 blog visits, reinforcing our mission to simplify neuromuscular research for patients, families, and researchers. Covering dystrophy, cell therapy, and antisense approaches, our goal is to make complex science accessible, offering hope and knowledge to those affected by neuromuscular diseases. This milestone strengthens our commitment to advancing research and raising awareness, ensuring that every child with DMD matters.

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Renato Dulbecco, a Nobel Prize-winning virologist, revolutionized cell culture by developing Dulbecco’s Modified Eagle’s Medium (DMEM) in the 1950s. His modifications improved mammalian cell growth, making DMEM one of the most widely used media in biomedical research. Dulbecco’s contributions extend beyond cell culture, including pioneering studies on oncoviruses and cancer biology. His work remains fundamental to modern cell biology and medical advancements.

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In neuromuscular research, growth media supports skeletal muscle cell proliferation, while differentiation media promotes myoblast fusion into myotubes. Growth media contains DMEM/F-12, FBS, and skeletal muscle supplements, whereas differentiation media replaces FBS with heat-inactivated horse serum for myotube formation. Proper media preparation ensures cell viability and stability, making it essential for studying neuromuscular diseases and muscle regeneration.

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Immortalized skeletal muscle cells derived from healthy and DMD patients serve as reproducible models for neuromuscular research. These cells aid in studying disease mechanisms and evaluating exon-skipping therapies. A key contributor, Kamel Mamchaoui, developed DMD myoblast cell lines, enhancing preclinical research. These models support dystrophin restoration studies and therapeutic advancements, helping researchers refine potential treatments for Duchenne muscular dystrophy (DMD).

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Cell scrapers are vital for adherent cell culture, allowing gentle, non-enzymatic cell harvesting from flasks, petri dishes, and multi-well plates. They help preserve cell integrity, making them ideal for subculturing, functional assays, and molecular analysis. Unlike trypsinization, scrapers avoid enzymatic effects that may alter cell surface markers or phenotypes. Their cost-effectiveness and ease of use make them essential tools for sensitive experiments requiring minimal cell disruption.

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Anti-dystrophin antibodies are crucial for Western blot analysis in Duchenne muscular dystrophy (DMD) research, enabling detection, quantification, and therapeutic validation of dystrophin restoration. These antibodies help measure protein levels following exon-skipping, gene therapy, and other treatments. Standardizing antibody specificity and sensitivity ensures reliable dystrophin quantification, driving consistent, reproducible results in DMD therapy evaluation and biomarker research.

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