glucosylceramide synthase

Sinbaglustat (OGT2378) (OGT2378) 是葡萄糖神经酰胺合成酶 (GCS) 和非溶酶体葡萄糖神经酰胺酶 (GBA2) 的双重抑制剂。Sinbaglustat 是一种可透过血脑屏障的的口服N-烷基亚氨基糖。Sinbaglustat 可用于治疗溶酶体贮积症和研究溶酶体功能障碍相关的中枢神经退行性疾病。

Ibiglustat (L-Malic acid) (Ibiglustat L-Malic acid) 是一种选择性的、脑渗透性的和别构的葡萄糖神经酰胺合酶抑制剂。 Ibiglustat (L-Malic acid) 可用于关于 PD 帕金森病、法布里和戈谢氏 SRT 的研究。

G43 是一种有效的选择性 glucosyltransferase 抑制剂，对 GtfB 和 GtfC 具有抑制作用，Kd 值分别为3.7 μM和46.9 nM。G43 在体内外具有抗变形链球菌 (S. mutans)活性，可用于研究龋齿。

AMP-Deoxynojirimycin (AMP-DNM) 是一种有效的神经酰胺葡萄糖基转移酶和 GCase 2 抑制剂。AMP-Deoxynojirimycin 是一种有效的 GlcCer 生物合成抑制剂，可用于研究帕金森和糖尿病。

Eliglustat (Genz 99067) 是一种高效的、特异性的、有口服活性的葡糖脑苷脂合成酶抑制剂(IC50:24 nM)。

Ibiglustat (GZ402671) 是一种可透过血脑屏障的、具有口服活性的葡萄糖神经酰胺合成酶(GCS)抑制剂。它可用于戈谢病 3 型、法布瑞氏症、与 GBA 突变相关的帕金森病、GM2 神经节苷脂病和常染色体显性多囊肾病的研究。

Miglustat hydrochloride (N-Butyldeoxynojirimycin hydrochloride) 是一种葡萄糖神经酰胺合酶抑制剂，可用于 I 型戈谢病的相关研究。

Glucosylceramide synthase-IN-3 (compound BZ1) is a highly potent, brain-penetrant, and orally active inhibitor of glucosylceramide synthase (GCS), exhibiting an IC50 value of 16 nM for human GCS. This compound, designated as Glucosylceramide synthase-IN-3, finds potential applications in Gaucher's disease research [1] [2].

Glucosylceramide synthase-IN-2 (compound T-690) 是一种有效的、具有血脑屏障通透性的、口服具有活力的 glucosylceramide synthase (GCS) 抑制剂，作用于人 GCS (IC50: 15 nM) 和小鼠 GCS (IC50: 190 nM)。Glucosylceramide synthase-IN-2 能够非竞争性抑制 C8 神经酰胺和 UDP 葡萄糖。Glucosylceramide synthase-IN-2 能够用于研究戈谢病。

Glucosylceramide synthase-IN-1 (T-036) 是一种有效的、口服具有活力的、具有血脑屏障通透性的 glucosylceramide synthase (GCS) 抑制剂，作用于人 GCS (IC50: 31 nM) 和小鼠 GCS (IC50: 51 nM)。Glucosylceramide synthase-IN-1 能够用于研究戈谢病。

Glucosylceramide synthase-IN-4 (compound 12) 是一种高效 GCS 抑制剂，具有 6.8 nM 的 IC50 值。该化合物在人肝细胞中展现出优良的 PK 属性和稳定性。此外，Glucosylceramide synthase-IN-4 还具备较好的中枢神经系统穿透能力和显著的 PXR 选择性。

Genz-123346 blocks the conversion of ceramide to glucosylceramide (GL1) and inhibits GM1 with an IC50 value of 14 nM. Genz-123346 is a potent, orally available glucosylceramide synthase inhibitor.

Miglustat (NB-DNJ) 是一种葡萄糖神经酰胺合成酶抑制剂，可用于I 型戈谢病。

Lucerastat（NBDGJ，芦舍司他）是一种口服的葡萄糖神经酰胺合酶（Glucosylceramide Synthase, GCS）的抑制剂，具有治疗法布里病的潜力。 ​GCS是鞘糖脂合成的关键酶，抑制其活性可减少有害底物的积累。

(-)-L-threo-PDMP is an enhancer of ganglioside biosynthesis. The (-)-L-threo-PDMP (1S,2S) isomer is the active stimulatory component of DL-threo-PDMP in contrast to (+)-D-threo-PDMP , which is an inhibitor of glucosylceramide synthetase. (-)-L-threo-PDMP upregulates GM3, GD3, and GQ1b synthase levels, which are glycosyltransferases involved in ganglioside biosynthesis, and increases ganglioside levels in cells. It increases neurite outgrowth and synapse formation in vitro and improves retention of a long-term memory learned prior to forebrain ischemia in rats when administered following ischemia at a dose of 40 mg/kg per day.

PDMP is a ceramide analog first prepared in a search for inhibitors of glucosylceramide synthase. PDMP has two adjacent chiral centers (C1 and C2) allowing for the formation of four possible isomers. PDMP contains all four of these stereoisomers. PDMP inhibits glucosylceramide synthase by 90% when used at a concentration of 0.8 μM in MDCK cell homogenates, however, the ability to inhibit glucosylceramide synthase has been found to reside in the D-threo (1R,2R) enantiomer. The D-threo PDMP enantiomer is also responsible for inhibition of β-1,4-galactosyltransferase 6 and prevention of lactosylceramide synthesis, which is a promotor of neuroinflammation in mice during chronic experimental autoimmune encephalomyelitis (EAE), a model of multiple sclerosis. PDMP enhances curcumin-induced inhibition of proliferation, JNK activation, and Akt inhibition, as well as induction of apoptosis in WM-115 melanoma cells in vitro.

D-threo-PPMP is a glucosylceramide (GlyCer) synthetase inhibitor.1,2It is the active enanantiomer and enzymatic inhibitory component of the racemic DL-threo-PPMP . In MDCK kidney epithelial cells, D-threo-PPMP induces a 70% reduction in cell growthin vitroat 20 μM and significantly inhibits DNA synthesis at 3 μM.3[Matreya, LLC. Catalog No. 1865] 1.Shen, W., Henry, A.G., Paumier, K.L., et al.Inhibition of glucosylceramide synthase stimulates autophagy flux in neuronsJ. Neurochem.129(5)884-894(2014) 2.Lee, L., Abe, A., and Shayman, J.A.Improved inhibitors of glucosylceramide synthaseJ. Biol. Chem.274(21)14662-14669(1999) 3.Abe, A., Inokuchi, J.-i., Jimbo, M., et al.Improved inhibitors of glucosylceramide synthaseJ. Biochem.111(2)191-196(1992)

(+)-D-threo-PDMP is a ceramide analog and is one of the four possible stereoisomers of PDMP . (+)-D-threo-PDMP is an inhibitor of glucosylceramide synthase. It inhibits glucosylceramide synthase by 50% when used at a concentration of 5 μM in an enzyme assay. (+)-D-threo-PDMP is the active component of racemic DL-threo-PDMP .

Deoxynojirimycin (dNM) tetrabenzyl ether is an intermediate for the synthesis of glucosylceramide synthase inhibitors such as 1-dNM, a glucose analog that potently inhibits α-glucosidase I and II.

D-threo-PPMP, a powerful glucosylceramide (GlcCer) synthase inhibitor, effectively impedes karyokinesis and decreases cyst formation.

Ibiglustat (Venglustat) succinate is an orally active, brain-penetrant inhibitor of glucosylceramide synthase (GCS). It is utilized in the investigation of Gaucher disease type 3, Parkinson's disease associated with GBA mutations, Fabry disease, GM2 gangliosidosis, and autosomal dominant polycystic kidney disease.

Ibiglustat hydrochloride is a potent and selective Glucosylceramide synthase inhibitor and ceramide glucosyltransferase inhibitor.

CCG-203586 is a novel potent glucosylceramide synthase (GCS) inhibitor.

Miglustat-d9 (hydrochloride)为Miglustat (hydrochloride)的氘代物，属于葡萄糖神经酰胺合成酶抑制剂，主要用于治疗I型戈谢病。