Genecode As

Baltic Technology Development, Ltd.

Mehis Pilv

Mart Saarma

Mati Karelson

Maxim Bespalov

Disclosed are compounds and methods for treating neurological and other disorders by administering to a subject in need thereof an effective amount of a compound having binding and/or modulation specificity for GFR&#x3b1; receptor molecules, which can be mimetics of glial-derived neurotrophic factor (GDNF) family ligands (GFLs), GFR&#x3b1;/RET signaling pathway agonists, and/or direct RET agonists (activators).

Methods of facilitating neural cell survival using GDNF family ligand (GFL) mimetics or RET signaling pathway activators

The present invention provides compounds having a chelating moiety and an oligonucleotide sequence wherein the oligonucleotide includes one or more modified nucleobases, such as hydroxynucleobases. The disclosed compounds are suitable for antisense therapy. The chelating moiety can be complexed to an ion of a lanthanide metal. These compounds are efficient translation inhibitors of nucleic acids and have increased binding affinity for target nucleic acids. The invention also includes compositions and methods of using these compositions as antisense therapy.

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Tallinn, Estonia

Mehis Pilv