Childers, Wayne published the artcileNovel compounds that reverse the disease phenotype in Type 2 Gaucher disease patient-derived cells, Computed Properties of 622-86-6, the main research area is structure preparation compound reversing phenotype Gaucher disease; Diphenylethanono; Gaucher disease; Lysosomal storage disease; Neuronopathic; Phenotypic screening.
Gaucher disease (GD) results from inherited mutations in the lysosomal enzyme β-glucocerobrosidase (GCase). Currently available treatment options for Type 1 GD are not efficacious for treating neuronopathic Type 2 and 3 GD due to their inability to cross the blood-brain barrier. In an effort to identify small mols. which could be optimized for CNS penetration we identified tamoxifen from a high throughput phenotypic screen on Type 2 GD patient-derived fibroblasts which reversed the disease phenotype. Structure activity studies around this scaffold led to novel mols. that displayed improved potency, efficacy and reduced estrogenic/antiestrogenic activity compared to the original hits. Here we present the design, synthesis and structure activity relationships that led to the lead mol. Compound 31.
Bioorganic & Medicinal Chemistry Letters published new progress about Gaucher disease. 622-86-6 belongs to class ethers-buliding-blocks, name is (2-Chloroethoxy)benzene, and the molecular formula is C8H9ClO, Computed Properties of 622-86-6.
Referemce:
Ether – Wikipedia,
Ether | (C2H5)2O – PubChem