Use of Hsp70 as a regulator of enzymatic activity
View Patent ↗The present invention concerns a method for modulating the enzymatic activity of an enzyme, wherein said enzyme interacts with BMP, said method comprising the step of administering or inducing Hsp70, or a functional fragment or variant thereof, in a form suitable for allowing interaction between BMP and Hsp70, or said functional fragment or variant thereof, and thereby modulating the enzymatic activity of an enzyme interacting with BMP.
1. A method for treatment of a lysosomal storage disorder selected from the group consisting of Niemann-Pick type A, Niemann-Pick type B, Niemann-Pick type C and Niemann-Pick type D, comprising the administration of a bioactive agent selected from Hsp70, a functional fragment of Hsp70 or variant thereof having at least 95% sequence identity to Hsp70, to an individual in need thereof.
2. The method according to claim 1 , wherein said bioactive agent is formulated as a pharmaceutical composition.
3. The method according to claim 1 , wherein said lysosomal storage disorder is characterised as having residual enzymatic activity of the defective enzyme involved in the disease pathology.
4. The method according to claim 1 , wherein said Hsp70, or functional fragment or variant thereof, has 100% identity to wild-type Hsp70 protein.
5. The method according to claim 1 , wherein said bioactive agent is Hsp70.
6. The method according to claim 1 , wherein said Hsp70 is full length Hsp70.
7. The method according to claim 1 , wherein said bioactive agent is a functional fragment or variant of Hsp70 having at least 95% sequence identity to Hsp70 .
8. The method according to claim 1 , wherein said functional fragment or variant of Hsp70 comprises all or part of the ATPase domain of Hsp70.
9. The method according to claim 1 , wherein said functional fragment or variant of Hsp70 comprises tryptophan at amino acid position 90 of the Hsp70 ATPase domain.
10. The method according to claim 1 , wherein said Hsp70, or a functional fragment or variant thereof, is recombinant Hsp70 (rHsp70).
11. The method according to claim 1 , wherein said Hsp70 is derived from a mammal selected from the group consisting of human (homo sapiens), mouse (mus musculus), cow, dog, rat, ferret, pig, sheep, and monkey.