| Description | Pabinafusp alfa (JR-141), a transferrin receptor-targeting antibody fusing Iduronate 2-sulfatase with an anti-human transferrin receptor antibody, penetrates the blood-brain barrier and inhibits heparan sulfate (HS) accumulation within the central nervous system of mucopolysaccharidosis II (MPS II) mice. It enhances cognitive function and protects against neuronal damage in the central nervous system [1]. |
| In vivo | Pabinafusp alfa（0.1-2 mg/kg；静脉注射；每周一次，持续36周）在剂量依赖性方面能够预防MPS II小鼠模型中神经组织的病理变化，并对神经缺陷有所改善[1]。 |
| Synonyms | JR-141 |
| molecular weight | N/A |
| CAS | 2140211-48-7 |
| Storage | Powder: -20°C for 3 years | In solvent: -80°C for 1 years |