THREOSE NUCLEIC ACID ANTISENSE OLIGONUCLEOTIDES AND METHODS THEREOF

Organization Name

HOFFMANN-LA ROCHE INC.

Inventor(s)

Jessica Marine Aurore Bastien of Basel (CH)

Konrad Bleicher of Freiburg (DE)

Helle Christiansen of Lyngby (DK)

Erich Koller of Basel (CH)

Meiling Li of Rheinfelden, Aargau (CH)

Helle Nymark of Farum (DK)

Adrian Schaeublin of Oberdorf (CH)

Steffen Schmidt of Copenhagen SV (DK)

THREOSE NUCLEIC ACID ANTISENSE OLIGONUCLEOTIDES AND METHODS THEREOF - A simplified explanation of the abstract

This abstract first appeared for US patent application 18749156 titled 'THREOSE NUCLEIC ACID ANTISENSE OLIGONUCLEOTIDES AND METHODS THEREOF

The patent application describes the use of antisense oligonucleotides containing α-L-threofuranosyl (TNA) nucleosides to modulate their properties, especially in antisense gapmer oligonucleotides.

Antisense oligonucleotides with TNA nucleosides are introduced to alter their properties.
The focus is on antisense gapmer oligonucleotides, where the TNA nucleosides can have a significant impact.
Methods are provided to effectively incorporate TNA nucleosides into antisense oligonucleotides.
The innovation aims to enhance the effectiveness and specificity of antisense oligonucleotides in gene regulation.
TNA nucleosides offer a new avenue for improving the design and function of antisense oligonucleotides.

Potential Applications: - Gene regulation and targeted therapy - Development of novel antisense oligonucleotide-based drugs - Research in molecular biology and genetic engineering

Problems Solved: - Enhancing the specificity and efficacy of antisense oligonucleotides - Improving the design and functionality of antisense oligonucleotides - Advancing the field of gene regulation and targeted therapy

Benefits: - Increased precision in gene targeting - Enhanced therapeutic potential of antisense oligonucleotides - Improved understanding of gene expression regulation mechanisms

Commercial Applications: Title: "Innovative Antisense Oligonucleotides with TNA Nucleosides for Targeted Gene Regulation" This technology could revolutionize the development of precision medicine and targeted therapies, potentially leading to the creation of new drugs for various genetic disorders and diseases.

Questions about Antisense Oligonucleotides with TNA Nucleosides: 1. How do TNA nucleosides enhance the properties of antisense oligonucleotides? 2. What are the potential implications of using TNA nucleosides in gene therapy applications?

Frequently Updated Research: Researchers are continually exploring the applications of TNA nucleosides in antisense oligonucleotides and their impact on gene regulation and targeted therapy. Stay updated on the latest advancements in this field for potential breakthroughs in precision medicine.

Original Abstract Submitted

Described are antisense oligonucleotides comprising one or more α-L-threofuranosyl (TNA) nucleosides as well as methods to modulate the properties of antisense oligonucleotides by the introduction of TNA nucleosides. These are particularly applicable for antisense gapmer oligonucleotides.