Publications by authors named "Claus Bornaes"
Article Synopsis
- - Gaucher Disease (GD) is caused by mutations in the GBA gene, which disrupts the function of the enzyme GCase, and potential therapy involves manipulating heat shock proteins to improve protein homeostasis.
- - The investigational drug arimoclomol, currently in phase II/III trials, enhances the function and activity of mutated GCase in cells from GD patients, showing promise for neurological symptoms due to its ability to cross the blood-brain barrier.
- - Findings indicate that arimoclomol boosts the production of protective proteins (HSPs), improving GCase function across various genetic mutations, supporting its clinical development as a potential treatment for the neuronopathic forms of GD.
Article Synopsis
- Lysosomal storage diseases (LSDs) present serious systemic and central nervous system symptoms, with current treatments offering limited effectiveness, particularly for neurological issues.
- Recent research shows that recombinant human heat shock protein 70 (HSP70) enhances the function of sphingolipid-degrading enzymes and reverses lysosomal damage in cells from patients with various LSDs.
- HSP70 also improves conditions in animal models of LSDs by reducing harmful lipid accumulation and alleviating neurological symptoms, indicating potential for heat shock protein therapies in clinical settings.
Purpose: Therapeutic proteins may induce antibodies that inhibit their efficacy or have other serious biological effects. There is a great need for strategies to predict whether a certain formulation will induce an immune response. In principle, conventional animals develop an immune response against all human proteins no matter how they are formulated, which restricts their use.
View Article and Find Full Text PDF