Results 11 to 20 of about 45,567 (253)
Challenges and future perspective of antisense therapy for spinal muscular atrophy: A review
European Journal of Cell Biology, 2023 Spinal muscular atrophy (SMA), the most common genetic cause of infantile death, is caused by a mutation in the survival of motor neuron 1 gene (SMN1), leading to the death of motor neurons and progressive muscle weakness.
Zorica Nakevska, Toshifumi Yokota
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Molecular Therapy: Nucleic Acids, 2023 Phosphorodiamidate morpholino oligonucleotide (PMO)-based antisense reagents cannot enter cells without the help of a delivery technique, which limits their clinical applications. To overcome this problem, self-transfecting guanidinium-linked morpholino (
Ujjal Das +8 more
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Molecular Therapy: Nucleic Acids, 2016 A series of poly(esteramine)s (PEAs) constructed from low molecular weight polyethyleneimine (LPEI) and Pluronic were evaluated for the delivery of antisense oligonuclotides (AOs), 2′-O-methyl phosphorothioate RNA (2′-OMePS) and phosphorodiamidate ...
Mingxing Wang +5 more
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Selected strategies to fight pathogenic bacteria
Journal of Enzyme Inhibition and Medicinal Chemistry, 2023 Natural products and analogues are a source of antibacterial drug discovery. Considering drug resistance levels emerging for antibiotics, identification of bacterial metalloenzymes and the synthesis of selective inhibitors are interesting for ...
Aiva Plotniece +13 more
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Duchenne Muscular Dystrophy: From Diagnosis to Therapy
Molecules, 2015 Duchenne muscular dystrophy (DMD) is an X-linked inherited neuromuscular disorder due to mutations in the dystrophin gene. It is characterized by progressive muscle weakness and wasting due to the absence of dystrophin protein that causes degeneration of
Maria Sofia Falzarano +3 more
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Polymersome delivery of siRNA and antisense oligonucleotides [PDF]
Journal of Controlled Release, 2009 siRNA and antisense oligonucleotides, AON, have similar size and negative charge and are often packaged for in vitro delivery with cationic lipids or polymers-but exposed positive charge is problematic in vivo. Here we demonstrate loading and functional delivery of RNAi and AON with non-ionic, nano-transforming polymersomes.
Younghoon, Kim +11 more
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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 +4 more
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Rationally designing antisense therapy to keep up with evolving bacterial resistance.
PLoS ONE, 2019 Antisense molecules used as antibiotics offer the potential to keep up with acquired resistance, by redesigning the sequence of an antisense. Once bacteria acquire resistance by mutating the targeted sequence, new antisense can readily be designed by ...
Seyfullah Kotil, Eric Jakobsson
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Expression of ssDNA in Mammalian Cells
BioTechniques, 2003 Antisense therapy involves the use of antisense oligonucleotides for altering targeted gene function. However, the low efficiency of cell delivery of antisense oligonucleotides has limited the efficacy of antisense therapeutic approaches.
Yin Chen, Yong-Jie Ji, Charles Conrad
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Molecular Therapy: Nucleic Acids, 2017 We investigated a series of Tween 85 modified low molecular weight polyethylenimine (LPEI, 0.8k/1.2k/2.0k)-copolymers (Zs) through simple formulation and covalent conjugation with phosphorodiamidate morpholino oligomer (PMO) for their potential to ...
Mingxing Wang +6 more
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