Development of nucleic acid medicines based on chemical technology
Y Kawamoto, Y Wu, Y Takahashi, Y Takakura - Advanced Drug Delivery …, 2023 - Elsevier
Oligonucleotide-based therapeutics have attracted attention as an emerging modality that
includes the modulation of genes and their binding proteins related to diseases, allowing us …
includes the modulation of genes and their binding proteins related to diseases, allowing us …
Antisense oligonucleotides and their applications in rare neurological diseases
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 …
and often leading to significantly impaired quality of life and high treatment costs. While …
Thiomorpholino oligonucleotides as a robust class of next generation platforms for alternate mRNA splicing
BT Le, S Paul, K Jastrzebska… - Proceedings of the …, 2022 - National Acad Sciences
Recent advances in drug development have seen numerous successful clinical translations
using synthetic antisense oligonucleotides (ASOs). However, major obstacles, such as …
using synthetic antisense oligonucleotides (ASOs). However, major obstacles, such as …
Precision medicine through antisense oligonucleotide-mediated exon skipping
Clinical implementation of two recently approved antisense RNA therapeutics–Exondys51®
to treat Duchenne muscular dystrophy (Duchenne MD) and Spinraza® as a treatment for …
to treat Duchenne muscular dystrophy (Duchenne MD) and Spinraza® as a treatment for …
Progress in the molecular pathogenesis and nucleic acid therapeutics for Parkinson's disease in the precision medicine era
Parkinson's disease (PD) is one of the most common neurodegenerative disorders that
manifest various motor and nonmotor symptoms. Although currently available therapies can …
manifest various motor and nonmotor symptoms. Although currently available therapies can …
Splice-modulating antisense oligonucleotides as therapeutics for inherited metabolic diseases
Abstract The last decade (2013–2023) has seen unprecedented successes in the clinical
translation of therapeutic antisense oligonucleotides (ASOs). Eight such molecules have …
translation of therapeutic antisense oligonucleotides (ASOs). Eight such molecules have …
Oligonucleotides as therapeutic tools for brain disorders: Focus on major depressive disorder and Parkinson's disease
Remarkable advances in understanding the role of RNA in health and disease have
expanded considerably in the last decade. RNA is becoming an increasingly important …
expanded considerably in the last decade. RNA is becoming an increasingly important …
Therapeutically significant microRNAs in primary and metastatic brain malignancies
Simple Summary The overall survival of brain cancer patients remains grim, with
conventional therapies such as chemotherapy and radiotherapy only providing marginal …
conventional therapies such as chemotherapy and radiotherapy only providing marginal …
Therapeutic antisense oligonucleotides for movement disorders
E Doxakis - Medicinal research reviews, 2021 - Wiley Online Library
Movement disorders are a group of neurological conditions characterized by abnormalities
of movement and posture. They are broadly divided into akinetic and hyperkinetic …
of movement and posture. They are broadly divided into akinetic and hyperkinetic …
Antisense oligonucleotide-mediated splice switching: potential therapeutic approach for cancer mitigation
Simple Summary Splicing is an important mechanism by which precursor mRNA is modified
into mature mRNA. This splicing plays a major role in the generation of different proteins …
into mature mRNA. This splicing plays a major role in the generation of different proteins …