Results 1 to 10 of about 83,412 (192)
Nonviral delivery systems for antisense oligonucleotide therapeutics
Biomaterials Research, 2022 Antisense oligonucleotides (ASOs) are an important tool for the treatment of many genetic disorders. However, similar to other gene drugs, vectors are often required to protect them from degradation and clearance, and to accomplish their transport in ...
Si Huang +5 more
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Simultaneous Down-Regulation of Intracellular hTERT and GPX4 mRNA Using MnO2-Nanosheet Probes to Induce Cancer Cell Death [PDF]
SensorsCancer remains a leading global cause of death, with conventional treatments often limited by toxicity and recurrence. Recent advances in gene therapy and nanodrug delivery offer new avenues for precision oncology. Human telomerase reverse transcriptase (
Yixin Miao +3 more
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Machine Learning To Predict Cell-Penetrating Peptides for Antisense Delivery [PDF]
ACS Central Science, 2018 Justin M. Wolfe +6 more
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Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies
Biomedicines, 2021 The literature surrounding the use of antisense oligonucleotides continues to grow, with new disease and mechanistic applications constantly evolving. Furthermore, the discovery and advancement of novel chemistries continues to improve antisense delivery,
Loren L. Flynn +6 more
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Antisense delivery using protamine-oligonucleotide particles [PDF]
Nucleic Acids Research, 2000 Protamine, a polycationic peptide (mol. wt 4000-4500), was evaluated as a potential penetration enhancer for phosphodiester antisense oligonucleotides (ODNs). Unique complexes in the form of nanoparticles were spontaneously formed, which we call 'proticles'.
M, Junghans, J, Kreuter, A, Zimmer
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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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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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Novel Cell type-specific aptamer-siRNA delivery system for HIV-1 therapy [PDF]
, 2007 The successful use of small interfering RNAs (siRNAs) for therapeutic purposes requires safe and efficient delivery to specific cells and tissues. Here we demonstrate cell type-specific delivery of anti-HIV siRNAs via fusion to an anti-gp120 aptamer. The
Haitang Li +4 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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