Advance genome editing technologies in the treatment of human diseases: CRISPR therapy (Review)

被引:17
|
作者
Alagoz, Meryem [1 ]
Kherad, Nasim [1 ]
机构
[1] Biruni Univ, Mol Biol & Genet, Protokol Yolu 45,10 Yil Cd, TR-34010 Istanbul, Turkey
关键词
clustered regularly interspaced short palindromic repeats; Cas9; gene editing; gene therapy; human diseases; neurodegenerative diseases; in vivoanimal models; ex vivomodels; DUCHENNE MUSCULAR-DYSTROPHY; ZINC-FINGER NUCLEASES; STRAND BREAK REPAIR; RNA-GUIDED ENDONUCLEASE; PLURIPOTENT STEM-CELLS; R-LOOP FORMATION; MOUSE MODEL; T-CELLS; ALZHEIMERS-DISEASE; CRYSTAL-STRUCTURE;
D O I
10.3892/ijmm.2020.4609
中图分类号
R-3 [医学研究方法]; R3 [基础医学];
学科分类号
1001 ;
摘要
Genome editing techniques are considered to be one of the most challenging yet efficient tools for assisting therapeutic approaches. Several studies have focused on the development of novel methods to improve the efficiency of gene editing, as well as minimise their off-target effects. Clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein (Cas9) is a tool that has revolutionised genome editing technologies. New applications of CRISPR/Cas9 in a broad range of diseases have demonstrated its efficiency and have been used inex vivomodels of somatic and pluripotent stem cells, as well as inin vivoanimal models, and may eventually be used to correct defective genes. The focus of the present review was the recent applications of CRISPR/Cas9 and its contribution to the treatment of challenging human diseases, such as various types of cancer, neurodegenerative diseases and a broad spectrum of other disorders. CRISPR technology is a novel method for disease treatment, enhancing the effectiveness of drugs and improving the development of personalised medicine.
引用
收藏
页码:521 / 534
页数:14
相关论文
共 50 条
  • [1] Genome-editing Technologies for Gene and Cell Therapy
    Maeder, Morgan L.
    Gersbach, Charles A.
    MOLECULAR THERAPY, 2016, 24 (03) : 430 - 446
  • [2] CRISPR-mediated genome editing and human diseases
    Cai, Liquan
    Fisher, Alfred L.
    Huang, Haochu
    Xie, Zijian
    GENES & DISEASES, 2016, 3 (04) : 244 - 251
  • [3] CRISPR Genome Editing Technology and Its Application in Genetic Diseases: A Review
    Khatibi, Sepideh
    Sahebkar, Amirhossein
    Aghaee-Bakhtiari, Seyed H.
    CURRENT PHARMACEUTICAL BIOTECHNOLOGY, 2021, 22 (04) : 468 - 479
  • [4] Progress and challenges in CRISPR-mediated therapeutic genome editing for monogenic diseases
    Konishi, Colin T.
    Long, Chengzu
    JOURNAL OF BIOMEDICAL RESEARCH, 2021, 35 (02): : 148 - 162
  • [5] Application of Genome Editing Technologies for Disease Treatment: Review
    Belachew, Girum Tefera
    Hanumanthaiah, Paramesh
    Tekelemariam, Bitaniya Abera
    JOURNAL OF PHARMACEUTICAL RESEARCH INTERNATIONAL, 2021, 33 (19A) : 1 - 17
  • [6] CRISPR/CAS9 GENOME EDITING FOR NEURODEGENERATIVE DISEASES
    Nojadeh, Jafar Nouri
    Eryilmaz, Nur Seren Bildiren
    Erguder, Berrin Imge
    EXCLI JOURNAL, 2023, 22 : 567 - 582
  • [7] Ocular delivery of CRISPR/Cas genome editing components for treatment of eye diseases
    Yu, Wenhan
    Wu, Zhijian
    ADVANCED DRUG DELIVERY REVIEWS, 2021, 168 : 181 - 195
  • [8] Application and perspective of CRISPR/Cas9 genome editing technology in human diseases modeling and gene therapy
    Zhang, Man-Ling
    Li, Hong-Bin
    Jin, Yong
    FRONTIERS IN GENETICS, 2024, 15
  • [9] Genome editing: A perspective on the application of CRISPR/Cas9 to study human diseases (Review)
    Raquel Rodriguez-Rodriguez, Diana
    Ramirez-Solis, Ramiro
    Alberto Garza-Elizondo, Mario
    De Lourdes Garza-Rodriguez, Maria
    Alberto Barrera-Saldana, Hugo
    INTERNATIONAL JOURNAL OF MOLECULAR MEDICINE, 2019, 43 (04) : 1559 - 1574
  • [10] Recent advances in CRISPR technologies for genome editing
    Song, Myeonghoon
    Koo, Taeyoung
    ARCHIVES OF PHARMACAL RESEARCH, 2021, 44 (06) : 537 - 552