A new report, coauthored by several major pharmaceutical companies, reviews the current state of sterile oligonucleotide drug product processing. The article, which provides recommendations to aid in the evaluation and development ...
Feb 22, 2023
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Bone tissue homeostasis is maintained by a balance between bone formation by osteoblasts and bone resorption by osteoclasts. However, when osteoblast function declines with age, bone remodeling becomes out of balance, bone ...
May 23, 2022
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Nucleic acid therapeutics, which involve transferring exogenous genes inside target cells, are a promising clinical treatment option that can regulate gene expression at the transcriptional or post-transcriptional level.
May 5, 2022
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A novel method uses subcellular fractionation to quantify label-free antisense oligonucleotides (AONs)- designed to silence targeted genes—that have crossed into the nucleus of a cell, where they can exert their effects. ...
Nov 14, 2019
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mRNA drugs offer a promising new approach to deliver therapeutic replacement proteins, and novel strategies designed to engineer more stable and active proteins are further enhancing the potential of mRNA therapies. Specific ...
Mar 30, 2018
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Researchers have shown that a wide variety of synthetic antisense oligonucleotides with different chemical modifications can activate the frataxin gene, which contains a mutation that decreases its expression in the inherited ...
Mar 8, 2018
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Researchers have reported the ability to modify the structure of small interfering RNAs (siRNAs) to improve their stability and therapeutic potential without negatively affecting their potency and ability to silence targeted ...
Jan 9, 2018
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A new addition to a series of articles that focus on important topics related to the development of oligonucleotide therapeutics presents an in-depth look at the identification, characterization, and reporting of product-related ...
Dec 12, 2017
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Designing new therapeutic DNA aptamers with diverse side chains can improve their ability to interact with targets, and a new study describes characteristics of these side chains that may determine how long the aptamers remain ...
Nov 29, 2017
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A team of leading European clinicians and scientists presents a unique perspective on how to move forward in the development of exon skipping therapies to treat the severe muscle-wasting disease Duchenne Muscular Dystrophy ...
Oct 16, 2017
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