Oligonucleotide-based therapies are a promising approach for treating a wide range of hard-
to-treat diseases, particularly genetic and rare diseases. These therapies involve the use of …

Introduction lncRNAs are major players in regulatory networks orchestrating multiple cellular
functions, such as 3D chromosomal interactions, epigenetic modifications, gene expression …

Nucleic acids drugs have been proven in the clinic as a powerful modality to treat inherited
and acquired diseases. However, key challenges including drug stability, renal clearance …

Rare diseases affect almost 500 million people globally, predominantly impacting children
and often leading to significantly impaired quality of life and high treatment costs. While …

Abstract The last decade (2013–2023) has seen unprecedented successes in the clinical
translation of therapeutic antisense oligonucleotides (ASOs). Eight such molecules have …

Phosphorodiamidate Morpholino Oligonucleotides (PMOs) have been well established in
the milieu of FDA-approved oligonucleotide-based drugs in the past decade. Given their …

Boron cluster-conjugated antisense oligonucleotides (B-ASOs) have already been
developed as therapeutic agents with “two faces”, namely as potential antisense inhibitors of …

Phosphorodiamidate morpholino oligomers (PMOs) are a special type of antisense
oligonucleotides (ASOs) that can be used as therapeutic modulators of pre-mRNA splicing …

Macrophages can serve as a reservoir for human immunodeficiency-1 (HIV-1) virus in host
cells, constituting a barrier to eradication, even in patients who are receiving antiretroviral …

New boron carriers with high boron content and targeted cancer-cell delivery are considered
the first choice for boron neutron capture therapy (BNCT) for cancer treatment. Previously …