Protein Silencing in Pharmaceutical Applications
A special issue of Pharmaceuticals (ISSN 1424-8247). This special issue belongs to the section "Biopharmaceuticals".
Deadline for manuscript submissions: 25 February 2026 | Viewed by 14
Special Issue Editor
Interests: pharmaceutics; targeted delivery; RNA interference; cancer biology; signaling pathways
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
The possibility of silencing virtually any protein in human cells using small interfering RNAs (siRNA) was an exciting notion at the turn of the century. The excitement, however, started to diminish over the next two decades, when the challenges facing siRNA delivery in vivo were realized. Despite these difficulties, multiple siRNA-based medications have entered the clinics, which include patisiran (Onpattro), the first siRNA-based medication approved by the FDA in August 2018 for targeting transthyretin in Polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults. A similar hype was created when preliminary reports surfaced about Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated protein (Cas)9 in the early 2010s, with the possibility of protein silencing permanently. The first clinical studies on CRISPR gene editing were performed in 2016 by injecting engineered T cells into a patient with metastatic non-small cell lung cancer, and the 2020 Nobel Prize in Chemistry being awarded to CRISPR/Cas9 research has added to the excitement for this approach. However, regardless of the selected approach, CRISPR/Cas9 delivery faces similar challenges as other nucleic acids (rapid degradation/excretion, non-targeted distribution, poor cellular internalization, etc.). This Special Issue aims to explore the most recent advances in the use of protein silencing in pharmaceutical applications. Our goal is to create a platform to present information about the new strategies in protein silencing and new potential applications for this approach in clinics.
Dr. Hamidreza Montazeri Aliabadi
Guest Editor
Manuscript Submission Information
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Keywords
- siRNA
- CRISPR/Cas9
- protein silencing
- cancer
- in vivo
- clinical applications
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