CAS 169105-89-9

Isofagomine

Description:

Isofagomine is a chemical compound characterized as a potent and selective inhibitor of the enzyme glucocerebrosidase, which is crucial in the metabolism of glycosphingolipids. It is primarily studied for its potential therapeutic applications in treating Gaucher disease, a genetic disorder resulting from glucocerebrosidase deficiency. Isofagomine is known for its ability to stabilize the enzyme in its active form, thereby enhancing its activity and promoting the breakdown of glucocerebrosides. The compound features a unique bicyclic structure that contributes to its binding affinity and specificity for the enzyme. Isofagomine is typically administered in a pharmaceutical context, and its efficacy is evaluated through various biochemical assays and clinical studies. Additionally, it is important to consider its pharmacokinetics, potential side effects, and interactions with other medications when assessing its therapeutic use. Overall, isofagomine represents a significant advancement in the treatment of lysosomal storage disorders, highlighting the importance of enzyme replacement and enhancement therapies in modern medicine.

Formula: C₆H₁₄ClNO₃

InChI: InChI=1/C6H13NO3/c8-3-4-1-7-2-5(9)6(4)10/h4-10H,1-3H2/t4-,5+,6+/m0/s1

InChI key: InChIKey=QPYJXFZUIJOGNX-HSUXUTPPSA-N

SMILES: C(O)[C@@H]1[C@@H](O)[C@H](O)CNC1

Synonyms:

• (3R,4R,5R)-5-(Hydroxymethyl)-3,4-Piperidinediol, Hydrochloride
• (3R,4R,5R)-5-(Hydroxymethyl)-3,4-piperidinediol
• (3R,4R,5R)-5-(Hydroxymethyl)-3,4-piperidinediol,HCl
• (3R,4R,5S)-5-(hydroxymethyl)piperidine-3,4-diol
• 3,4-Piperidinediol, 5-(hydroxymethyl)-, (3R,4R,5R)-
• 3,4-Piperidinediol, 5-(hydroxymethyl)-, [3R-(3α,4β,5α)]-
• <span class="text-smallcaps">D</span>-Isofagomine
• Afegostat
• Isofagomine
• IsofagomineHydrochlroide
• Plicera
• D-Isofagomine
• 5-Hydroxymethyl-3,4-piperidinediol
• (3R,4R,5R)-5-(Hydroxymethyl)piperidine-3,4-diol hydrochloride
• ISOFAGOMINE, HYDROCHLORIDE
• (3R,4R,5R)-5-(Hydroxymethyl)piperidine-3,4-diol

(3R,4R,5R)-5-(Hydroxymethyl)piperidine-3,4-diol

CAS: 169105-89-9

Formula: C₆H₁₃NO₃

Molecular weight: 147.1723

Ref: IN-DA001YGP

Isofagomine

Controlled Product

CAS: 169105-89-9

Formula: C₆H₁₃NO₃

Color and Shape: Neat

Molecular weight: 147.172

Ref: TR-I816015

Isofagomine

CAS: 169105-89-9

Isofagomine is a chiral molecule that has been used as a model system for studying the stereochemical effects of enzyme inhibition. It is an inhibitor of the enzyme glucosylceramide synthase, which is involved in the synthesis of glucosylceramide (a major component of the myelin sheath). Isofagomine binds to an allosteric site on this enzyme and inhibits its activity. The binding site on glucosylceramide synthase has been mapped using X-ray crystallography and NMR spectroscopy. Isofagomine also inhibits other enzymes, such as phosphatidylethanolamine N-methyltransferase, which catalyzes the transfer of methyl groups from S-adenosylmethionine to phosphatidylethanolamine. Isofagomine has been shown to have neuroprotective properties against oxidative injury by reducing lipid peroxidation in rat brain cells. This may

Formula: C₆H₁₃NO₃

Purity: Min. 95%

Molecular weight: 147.17 g/mol

Ref: 3D-UGA10589

Afegostat

CAS: 169105-89-9

Afegostat is a pharmacological chaperone that specifically and reversibly binds acid-β-glucosidase (GCase) in the endoplasmic reticulum with high affinity.

Formula: C₆H₁₃NO₃

Purity: 98%

Color and Shape: Yellow Low-Melting Solid

Molecular weight: 147.17

Ref: TM-T19181