Under the hood: The molecular biology driving gene therapy for the treatment of sickle cell disease

被引：0

作者：

Waldron, Evan ^{[1
]}

Tanhehco, Yvette C. ^{[1
,2
]}

机构：

[1] Columbia Univ, Irving Med Ctr, Dept Pathol & Cell Biol, 622W 168th St, New York, NY 10032 USA

[2] Columbia Univ, Irving Med Ctr, Dept Pathol & Cell Biol, Div Transfus Med, 622W 168th St Harkness Pavil,4-418A, New York, NY 10032 USA

来源：

TRANSFUSION AND APHERESIS SCIENCE | 2022年 / 61卷 / 05期

关键词：

Hematopoietic stem cell transplantation; Gene therapy; Sickle cell disease; CRISPR-Cas9; Lentiglobin; RNA interference; DOUBLE-STRANDED-RNA; CRYSTAL-STRUCTURE; COMPLEX; GENOME; HEMOGLOBIN; ALLOIMMUNIZATION; INTERFERENCE; DETERMINANTS; LENTIVIRUS; EXPRESSION;

D O I：

10.1016/j.transci.2022.103566

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Gene therapy will soon become the dominant modality for treating of sickle cell disease (SCD). Currently, three technologies are the most promising: expression of transgenic globin genes via a lentiviral vector, controlled mutation of the 8-globin control cluster by transgenic CRISPR-based ribonucleoprotein, and suppression of BCL11a mRNA by shRNA. In this review, we discuss the mechanism of each technology and how they correct the SCD pathology at the molecular level. We conclude by discussing potential directions future therapy may take.

引用

页数：6

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