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Abstract
The ability of small nucleic acids to modulate gene expression via a range of processes has been widely explored. Compared with conventional treatments, small nucleic acid therapeutics have the potential to achieve long-lasting or even curative effects via gene editing. As a result of recent technological advances, efficient small nucleic acid delivery for therapeutic and biomedical applications has been achieved, accelerating their clinical translation. Here, we review the increasing number of small nucleic acid therapeutic classes and the most common chemical modifications and delivery platforms. We also discuss the key advances in the design, development and therapeutic application of each delivery platform. Furthermore, this review presents comprehensive profiles of currently approved small nucleic acid drugs, including 11 antisense oligonucleotides (ASOs), 2 aptamers and 6 siRNA drugs, summarizing their modifications, disease-specific mechanisms of action and delivery strategies. Other candidates whose clinical trial status has been recorded and updated are also discussed. We also consider strategic issues such as important safety considerations, novel vectors and hurdles for translating academic breakthroughs to the clinic. Small nucleic acid therapeutics have produced favorable results in clinical trials and have the potential to address previously “undruggable” targets, suggesting that they could be useful for guiding the development of additional clinical candidates.
Academic Publication Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines
Research Abstract & Technology Focus
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Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines
Abstract The ability of small nucleic acids to modulate gene expression via a range of processes has been widely explored. Compared with conventional treatments, small nucleic aci...
Nucleic Acid
The biopharmaceutical sector is advancing next-gen RNA therapeutics and oligonucleotide drugs, evidenced by industry focus events. Simultaneously, nucleic acid synthesis faces escalating global bio...
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Frequently Asked Questions (FAQ)
Curated market intelligence mapped to this research.
What is the core focus of the research titled 'Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines'?
This literature focuses on: Abstract The ability of small nucleic acids to modulate gene expression via a range of processes has been widely explored. Compared with conventional treatments, small nucleic acid therapeutics have the potential to achieve long-...
Are there open-source GitHub repositories related to Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines?
Yes, open-source projects like danveloper/flash-moe (Running a big model on a small laptop) are actively building upon these concepts.
Which startups are commercializing the technology behind Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines?
Products like GitHub Stacked PRs are bringing this to market. Their focus is: Break big changes into small reviewable PRs.
What other academic literature is closely related to 'Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines'?
Yes, highly correlated activity was mapped. An entry titled 'Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines' discusses this: Abstract The ability of small nucleic acids to modulate gene expression via a range of processes has been widely explored. Compar...
Are there commercial applications of 'Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines' in market news publications?
Yes, highly correlated activity was mapped. An entry titled 'Nucleic Acid' discusses this: The biopharmaceutical sector is advancing next-gen RNA therapeutics and oligonucleotide drugs, evidenced by industry focus events. Simultaneously, ...
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Commercial Realization
Startups and Open Source tools heavily associated with the concepts explored in this paper.
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GitHubdanveloper/flash-moe
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GitHubhaydenbleasel/files-sdk
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Product HuntGitHub Stacked PRs
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Product HuntOpenStartup