18735668. ANTISENSE OLIGONUCLEOTIDES TARGETING ACTL6B simplified abstract (Hoffmann-La Roche Inc.)
Contents
ANTISENSE OLIGONUCLEOTIDES TARGETING ACTL6B
Organization Name
Inventor(s)
Lars Joenson of Viby Sjaelland (DK)
Bettina Nordbo of Hørsholm (DK)
Jonas Vikesaa of Fredensborg (DK)
ANTISENSE OLIGONUCLEOTIDES TARGETING ACTL6B - A simplified explanation of the abstract
This abstract first appeared for US patent application 18735668 titled 'ANTISENSE OLIGONUCLEOTIDES TARGETING ACTL6B
The present invention involves antisense oligonucleotide splice modulators of actin-like 6B (ACTL6B) that can increase or restore the expression of ACTL6B in TDP-43 depleted cells.
- Complementary antisense oligonucleotide splice modulators target the ACTL6B precursor-mRNA.
- These modulators are capable of restoring ACTL6B expression in cells depleted of TDP-43.
- The technology addresses conditions where TDP-43 is functionally depleted.
- The innovation offers a potential solution for medical indications related to TDP-43 depletion.
- Fully complementary antisense oligonucleotide splice modulators are utilized in this technology.
Potential Applications: - Treatment of conditions involving TDP-43 depletion - Medical indications where ACTL6B expression needs to be increased or restored
Problems Solved: - Restoring ACTL6B expression in TDP-43 depleted cells - Addressing medical conditions related to TDP-43 depletion
Benefits: - Potential therapeutic benefits for patients with TDP-43 related conditions - Targeted modulation of gene expression
Commercial Applications: Title: Antisense Oligonucleotide Splice Modulators for TDP-43 Depletion-Related Conditions Description: This technology can be utilized in the development of novel therapeutics for conditions associated with TDP-43 depletion, potentially opening up new market opportunities in the pharmaceutical industry.
Questions about Antisense Oligonucleotide Splice Modulators for TDP-43 Depletion-Related Conditions: 1. How do antisense oligonucleotide splice modulators target ACTL6B expression in TDP-43 depleted cells? 2. What are the potential implications of restoring ACTL6B expression in conditions related to TDP-43 depletion?
Original Abstract Submitted
The present invention relates to antisense oligonucleotide splice modulators of actin-like 6B (ACTL6B). These antisense oligonucleotide splice modulators are complementary, such as fully complementary, to the ACTL6B precursor-mRNA, and are capable of increasing or restoring expression of ACTL6B in TDP-43 depleted cells, such as for use in conditions and medical indications where TDP-43 is functionally depleted.
