相关实验视频

Updated: Jul 17, 2025

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
06:51

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

Published on: August 10, 2018

7.7K

工程ArylsulfataseA增加了活性,稳定性和大脑输送,用于治疗元染色性白血病

Claudia Yaghootfam1, Marc Sylvester2, Boris Turk3

  • 1Institute of Biochemistry and Molecular Biology, Medical Faculty, University of Bonn, Nussallee 11, 53115 Bonn, Germany.

Molecular therapy : the journal of the American Society of Gene Therapy
|August 30, 2023
PubMed
概括

改造的人类阿里硫酶A (hASA),命名为SuPerTurbo-ASA,显示显著改善半衰期和治疗元染色性白血病 (MLD) 的活性. 这种增强的酶有效地减少了MLD小鼠模型中中枢神经系统的硫酸盐储存.

关键词:
亚利硫酶A是一种酸盐.酶替代疗法是一种酶替代疗法.基因工程是基因工程,是基因工程.lysosomal 储存疾病 lysosomal 储存疾病甲基染色性白血病变异症是什么

更多相关视频

Metabolic Glycoengineering of Sialic Acid Using N-acyl-modified Mannosamines12:06

Metabolic Glycoengineering of Sialic Acid Using N-acyl-modified Mannosamines

Published on: November 25, 2017

12.7K
In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease10:16

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease

Published on: December 20, 2017

8.1K

相关概念视频

Antiepileptic Drugs: Modulators of Neurotransmitter Release Mediated by SV2A Protein01:20

Antiepileptic Drugs: Modulators of Neurotransmitter Release Mediated by SV2A Protein

Antiepileptic drugs, such as levetiracetam (Keppra) and brivaracetam (Briviact), have emerged as crucial tools in managing epilepsy. These medications exert their therapeutic effects by targeting the synaptic vesicle protein SV2A, a transmembrane glycoprotein primarily found in the brain.
SV2A is a transmembrane glycoprotein located predominantly in the brain, modulating the release of neurotransmitters for neuronal communication. Both levetiracetam and brivaracetam exhibit a high affinity for...
341