[HTML][HTML] Drug delivery systems for RNA therapeutics
RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …
Regulation and functions of non-m6A mRNA modifications
Nucleobase modifications are prevalent in eukaryotic mRNA and their discovery has
resulted in the emergence of epitranscriptomics as a research field. The most abundant …
resulted in the emergence of epitranscriptomics as a research field. The most abundant …
[HTML][HTML] The critical contribution of pseudouridine to mRNA COVID-19 vaccines
P Morais, H Adachi, YT Yu - Frontiers in cell and developmental …, 2021 - frontiersin.org
The current COVID-19 pandemic is a massive source of global disruption, having led so far
to two hundred million COVID-19 cases and more than four million deaths worldwide. It was …
to two hundred million COVID-19 cases and more than four million deaths worldwide. It was …
Nucleic acid degradation as barrier to gene delivery: a guide to understand and overcome nuclease activity
Gene therapy is on its way to revolutionize the treatment of both inherited and acquired
diseases, by transferring nucleic acids to correct a disease-causing gene in the target cells …
diseases, by transferring nucleic acids to correct a disease-causing gene in the target cells …
Drug metabolism and pharmacokinetics of antisense oligonucleotide therapeutics: typical profiles, evaluation approaches, and points to consider compared with small …
H Takakusa, N Iwazaki, M Nishikawa… - nucleic acid …, 2023 - liebertpub.com
Oligonucleotide therapeutics are attracting attention as a new treatment modality for a range
of diseases that have been difficult to target using conventional approaches. Technical …
of diseases that have been difficult to target using conventional approaches. Technical …
The promise of nanotechnology in personalized medicine
MA Alghamdi, AN Fallica, N Virzì… - Journal of personalized …, 2022 - mdpi.com
Both personalized medicine and nanomedicine are new to medical practice. Nanomedicine
is an application of the advances of nanotechnology in medicine and is being integrated into …
is an application of the advances of nanotechnology in medicine and is being integrated into …
2′-O-methylation at internal sites on mRNA promotes mRNA stability
O-methylation (Nm) is a prominent RNA modification well known in noncoding RNAs and
more recently also found at many mRNA internal sites. However, their function and base …
more recently also found at many mRNA internal sites. However, their function and base …
The challenge of peptide nucleic acid synthesis
KP Nandhini, D Al Shaer, F Albericio… - Chemical Society …, 2023 - pubs.rsc.org
Peptide nucleic acids (PNAs) are an important class of DNA/RNA mimics that can hybridize
complementary chains of nucleic acids with high affinity and specificity. Because of this …
complementary chains of nucleic acids with high affinity and specificity. Because of this …
Gene therapy in hemophilia: recent advances
EC Rodríguez-Merchán, JA De Pablo-Moreno… - International Journal of …, 2021 - mdpi.com
Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX
genes. The palliative treatment of choice is based on the use of safe and effective …
genes. The palliative treatment of choice is based on the use of safe and effective …
[HTML][HTML] Ion-pair reversed-phase and hydrophilic interaction chromatography methods for analysis of phosphorothioate oligonucleotides
M Gilar, BM Koshel, RE Birdsall - Journal of Chromatography A, 2023 - Elsevier
In this study we investigated the separation of a 25 mer fully phosphorothioated
oligonucleotide from its truncated n-1 (24 mer) species and selected phosphodiester 25 mer …
oligonucleotide from its truncated n-1 (24 mer) species and selected phosphodiester 25 mer …