Inhibition of lipofuscin aggregation by molecular tweezers
In various embodiments methods for the treatment and/or prophylaxis of lipofuscin-related disorders are provided. In certain embodiments the methods involve administration of an effective amount of a molecular tweezers to a subject in need thereof.
1. A method of treating a lipofuscin-related disorder in a mammal, said method comprising:
administering to said mammal an effective amount of a molecular tweezers that is capable of inhibiting the accumulation of lipofuscin aggregates;
wherein said lipofuscin-related disorder is Stargardt disease or a neuronal ceroid lipofuscinosis (NCL); and
wherein said molecular tweezers is selected from the group consisting of:
2. The method of claim 1 , wherein said molecular tweezers is capable of inhibiting lipofuscin aggregation, and wherein said effective amount is an amount effective to delay the onset of, or to slow the progression, or stop, or to reverse lipofuscin accumulation/aggregation associated with said lipofuscin-related disorder.
3. The method of claim 1 , wherein said lipofuscin-related disorder is an NCL, wherein said NCL is selected from the group consisting of infantile NCL (Santavuori-Haltia disease), late Infantile NCL (Jansky-Bielschowsky disease), Juvenile NCL (CLN1, Batten disease), Adult NCL (Kufs disease), Finnish Late Infantile NCL, Variant Late Infantile NCL, CLN7 NCL, CLN8 NCL (Northern Epilepsy, progressive epilepsy with mental retardation (EPMR)), Turkish Late Infantile Variant NCL, and CLN10 NCL (Congenital, Cathepsin D Deficiency).
4. The method of claim 3 , wherein said NCL is Batten disease.