What is the Future of Catalysis in Gaucher Disease?
The future of treating Gaucher disease with catalysis lies in improving the efficacy and accessibility of therapies. Advances in gene editing technologies, such as CRISPR/Cas9, offer the potential for permanent correction of the genetic defect that causes the enzyme deficiency. Furthermore, ongoing research in small-molecule chaperones aims to stabilize the mutant enzyme, enhancing its catalytic activity and preventing substrate accumulation.
