Compositions and methods for the treatment of juvenile neuronal ceroid lipofuscinosis and related disorders
US-2017049761-A1 · Feb 23, 2017 · US
US12286452B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-12286452-B2 |
| Application number | US-202017119301-A |
| Country | US |
| Kind code | B2 |
| Filing date | Dec 11, 2020 |
| Priority date | Jun 12, 2018 |
| Publication date | Apr 29, 2025 |
| Grant date | Apr 29, 2025 |
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The present invention includes novel molecules and methods of using an amphiphilic kanamycin molecule having substitutions at the O-4″, O-6″, or O-4″ and O-6″ positions having reduced antimicrobial activity for targeting Connexin Hemichannels.
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What is claimed is: 1. An amphiphilic molecule with a structure having reduced antimicrobial activity when compared to unmodified kanamycin; wherein Y is H 2 , Z is HR 4 , R 1 , R 2 , R 3 , R 4 are, methyl, ethyl, propyl, butyl, linear alkyl, alkylcarbonyl chains, or cyclic groups. 2. The molecule of claim 1 , wherein the molecule has at least one of: substantially no antimicrobial activity, no antibacterial activity, or inhibits connexin hemichannels. 3. A molecule of formula: wherein R in compounds 7b to 7 h is selected from at least one of: R Compound 7b 7c 7d 7e 7f 7g 7h. 4. A molecule comprising the formula: wherein Code/compound # R K46B01/7 Benzyl; K46BM5/8 Benzyl and all hydroxyl groups were methylated; K46B09/10 K46B03/11 K46B10/12 K46B04/13 K46B05/14 K46B11/15 K46B07/16 5. A molecule having the formula:
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