View source for 20240033378. ANTISENSE OLIGOMERS FOR TREATMENT OF NON-SENSE MEDIATED RNA DECAY BASED CONDITIONS AND DISEASES simplified abstract (STOKE THERAPEUTICS, INC.)

=ANTISENSE OLIGOMERS FOR TREATMENT OF NON-SENSE MEDIATED RNA DECAY BASED CONDITIONS AND DISEASES=


==Organization Name==

[[:Category:STOKE THERAPEUTICS, INC.|STOKE THERAPEUTICS, INC.]]


[[Category:STOKE THERAPEUTICS, INC.]]

==Inventor(s)==

[[:Category:ISABEL Aznarez of Boston MA (US)|ISABEL Aznarez of Boston MA (US)]][[Category:ISABEL Aznarez of Boston MA (US)]]

[[:Category:Enxuan Jing of West Roxbury MA (US)|Enxuan Jing of West Roxbury MA (US)]][[Category:Enxuan Jing of West Roxbury MA (US)]]

[[:Category:Jacob Kach of Jamaica Plain MA (US)|Jacob Kach of Jamaica Plain MA (US)]][[Category:Jacob Kach of Jamaica Plain MA (US)]]

[[:Category:Aditya Venkatesh of Malden MA (US)|Aditya Venkatesh of Malden MA (US)]][[Category:Aditya Venkatesh of Malden MA (US)]]

[[:Category:Juergen Scharner of Arlington MA (US)|Juergen Scharner of Arlington MA (US)]][[Category:Juergen Scharner of Arlington MA (US)]]

[[:Category:Baruch Ticho of Newton MA (US)|Baruch Ticho of Newton MA (US)]][[Category:Baruch Ticho of Newton MA (US)]]

[[:Category:Gene Liau of Wayland MA (US)|Gene Liau of Wayland MA (US)]][[Category:Gene Liau of Wayland MA (US)]]

==ANTISENSE OLIGOMERS FOR TREATMENT OF NON-SENSE MEDIATED RNA DECAY BASED CONDITIONS AND DISEASES - A simplified explanation of the abstract==


This abstract first appeared for US patent application 20240033378 titled 'ANTISENSE OLIGOMERS FOR TREATMENT OF NON-SENSE MEDIATED RNA DECAY BASED CONDITIONS AND DISEASES

==Simplified Explanation==

The patent application describes therapeutic agents that can target alternative splicing events in genes to modulate the expression level of functional proteins in patients and/or inhibit aberrant protein expression. These agents can be used to treat conditions or diseases caused by protein deficiency.

* The patent application focuses on alternative splicing events in genes and their impact on protein expression.
* It proposes therapeutic agents that can target these alternative splicing events.
* The agents aim to modulate the expression level of functional proteins in patients.
* Additionally, the agents can inhibit aberrant protein expression.
* The technology described in the patent application can be used to treat conditions or diseases caused by protein deficiency.

== Potential Applications ==
The technology described in the patent application has potential applications in various fields, including:

* Pharmaceutical industry
* Biotechnology industry
* Medical research and development

== Problems Solved ==
The technology described in the patent application addresses the following problems:

* Non-productive mRNA transcripts resulting from alternative splicing events can lead to aberrant protein expression.
* Protein deficiency can cause various conditions or diseases.
* Current treatment options for protein deficiency may be limited or ineffective.

== Benefits ==
The technology described in the patent application offers the following benefits:

* Targeting alternative splicing events can modulate the expression level of functional proteins.
* Inhibiting aberrant protein expression can help prevent or treat conditions caused by protein deficiency.
* Therapeutic agents can provide a more targeted and effective approach to treating protein deficiency-related conditions or diseases.


==Original Abstract Submitted==

alternative splicing events in genes can lead to non-productive mrna transcripts which in turn can lead to aberrant protein expression, and therapeutic agents which can target the alternative splicing events in genes can modulate the expression level of functional proteins in patients and/or inhibit aberrant protein expression. such therapeutic agents can be used to treat a condition or disease caused by protein deficiency.



[[Category:A61K48/00]]
[[Category:C12N15/113]]