Name: Indolin-2-one. I found the field of Pharmacology & Pharmacy very interesting. Saw the article Lead Optimization of Benzoxazolone Carboxamides as Orally Bioavailable and CNS Penetrant Acid Ceramidase Inhibitors published in 2020.0, Reprint Addresses Skerlj, R (corresponding author), Lysosomal Therapeut Inc, Cambridge, MA 02139 USA.; Scarpelli, R (corresponding author), Fdn Ist Italiano Tecnol, Drug Discovery & Dev D3 Validat, I-16163 Genoa, Italy.. The CAS is 59-48-3. Through research, I have a further understanding and discovery of Indolin-2-one.
Sphingolipids (SphLs) are a diverse class of molecules that are regulated by a complex network of enzymatic pathways. A disturbance in these pathways leads to lipid accumulation and initiation of several SphL-related disorders. Acid ceramidase is one of the key enzymes that regulate the metabolism of ceramides and glycosphingolipids, which are important members of the SphL family. Herein, we describe the lead optimization studies of benzoxazolone carboxamides resulting in piperidine 22m, where we demonstrated target engagement in two animal models of neuropathic lysosomal storage diseases (LSDs), Gaucher’s and Krabbe’s diseases. After daily intraperitoneal administration at 90 mg kg(-1), 22m significantly reduced the brain levels of the toxic lipids glucosylsphingosine (GluSph) in 4L;C* mice and galactosylsphingosine (GalSph) in Twitcher mice. We believe that 22m is a lead molecule that can be further developed for the correction of severe neurological LSDs where GluSph or GalSph play a significant role in disease pathogenesis.
Name: Indolin-2-one. About Indolin-2-one, If you have any questions, you can contact Di Martino, S; Tardia, P; Cilibrasi, V; Caputo, S; Mazzonna, M; Russo, D; Penna, I; Realini, N; Margaroli, N; Migliore, M; Pizzirani, D; Ottonello, G; Bertozzi, SM; Armirotti, A; Nguyen, D; Sun, Y; Bongarzone, ER; Lansbury, P; Liu, M; Skerlj, R; Scarpelli, R or concate me.
Reference:
Indoline – Wikipedia,
,Indoline | C8H9N – PubChem