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
收藏
页数：6
相关论文
共 50 条
  • [1] Gene Therapy: A New Hope in Sickle Cell Disease Treatment
    Christy, Margaret
    Fisher, Beth
    JOURNAL OF PEDIATRIC HEALTH CARE, 2025, 39 (01) : 122 - 129
  • [2] Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study
    Urbinati, Fabrizia
    Fernandez, Beatriz Campo
    Masiuk, Katelyn E.
    Poletti, Valentina
    Hollis, Roger P.
    Koziol, Colin
    Kaufman, Michael L.
    Brown, Devin
    Mavilio, Fulvio
    Kohn, Donald B.
    HUMAN GENE THERAPY, 2018, 29 (10) : 1153 - 1166
  • [3] Gene therapy for sickle cell disease
    Olowoyeye, Abiola
    Okwundu, Charles I.
    COCHRANE DATABASE OF SYSTEMATIC REVIEWS, 2012, (11):
  • [4] Diverse Approaches to Gene Therapy of Sickle Cell Disease
    White, Shanna L.
    Hart, Kevyn
    Kohn, Donald B.
    ANNUAL REVIEW OF MEDICINE, 2023, 74 : 473 - 487
  • [5] Towards gene therapy of sickle cell disease
    Bouhassira, EE
    Acharya, AS
    EXPERT OPINION ON THERAPEUTIC PATENTS, 2000, 10 (07) : 1081 - 1093
  • [6] Gene therapy for sickle cell disease and thalassemia
    Scaramellini, Natalia
    Panzieri, Daniele Lello
    Cappellini, Maria Domenica
    CURRENT OPINION IN HEMATOLOGY, 2025, 32 (03) : 120 - 129
  • [7] Gene therapy in the fight against pediatric blood disorders: sickle cell disease and β-thalassemia
    Hu, Wei
    Wang, Bingyuan
    Song, Guowei
    Wang, Xiaojing
    Wang, Xiaoyun
    DRUGS OF THE FUTURE, 2022, 47 (06) : 443 - 456
  • [8] Gene therapy for sickle cell disease
    Olowoyeye, Abiola
    Okwundu, Charles I.
    COCHRANE DATABASE OF SYSTEMATIC REVIEWS, 2010, (08):
  • [9] Gene therapy for sickle cell disease
    Olowoyeye, Abiola
    Okwundu, Charles I.
    COCHRANE DATABASE OF SYSTEMATIC REVIEWS, 2018, (11):
  • [10] Gene therapy for sickle cell disease
    Olowoyeye, Abiola
    Okwundu, Charles I.
    COCHRANE DATABASE OF SYSTEMATIC REVIEWS, 2016, (11):
12345