2,3,4-Trihydroxy-6-oxo-5-sulfooxyhexanoic acid

• 分子结构分类: 有机化合物 - 有机氧化合物
• 英文名称: 2,3,4-Trihydroxy-6-oxo-5-sulfooxyhexanoic acid
• 化学式: C₆H₁₀O₁₀S
• 分子量: 274.2
• InChiKey: PURMPUDWXOWORS-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -3
• 重原子数：
  17
• 可旋转键数：
  7
• 环数：
  0.0
• sp3杂化的碳原子比例：
  0.67
• 拓扑面积：
  187
• 氢给体数：
  5
• 氢受体数：
  10

文献信息

• Adeno Associated Virus Vectors for the Treatment of Hunter Disease
  申请人：Shire Human Genetic Therapies, Inc.

  公开号：US20210332383A1

  公开（公告）日：2021-10-28

  The present disclosure provides, among other things, a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 or AAV9 capsid and a codon-optimized sequence encoding a human iduronate-2-sulfatase (I2S) enzyme. The disclosure also provides a method of treating a subject having Hunter syndrome (MPS II), comprising administering to the subject in need thereof a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 or AAV9 capsid, and a promoter operably linked to a nucleic acid sequence that encodes iduronate-2-sulfatase (I2S), and wherein administering results in an increase in I2S enzymatic activity in the subject.
• [EN] ADENO ASSOCIATED VIRUS VECTORS FOR THE TREATMENT OF HUNTER DISEASE<br/>[FR] VECTEURS DE VIRUS ADÉNO-ASSOCIÉS POUR LE TRAITEMENT DE LA MALADIE DE HUNTER
  申请人：SHIRE HUMAN GENETIC THERAPIES

  公开号：WO2021119053A1

  公开（公告）日：2021-06-17

  The present disclosure provides, among other things, a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 or AAV9 capsid and a codon-optimized sequence encoding a human iduronate-2-sulfatase (I2S) enzyme. The disclosure also provides a method of treating a subject having Hunter syndrome (MPS II), comprising administering to the subject in need thereof a recombinant adeno-associated virus (rAAV) vector comprising an AAV8 or AAV9 capsid, and a promoter operably linked to a nucleic acid sequence that encodes iduronate-2-sulfatase (I2S), and wherein administering results in an increase in I2S enzymatic activity in the subject.