Therapeutic in vivo delivery of gene editing agents

被引:167
|
作者
Raguram, Aditya [1 ,2 ,3 ]
Banskota, Samagya [1 ,2 ,3 ]
Liu, David R. [1 ,2 ,3 ]
机构
[1] Broad Inst MIT & Harvard, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA
[2] Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA
[3] Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA
关键词
ADENOASSOCIATED VIRUS AAV; HUMAN STEM-CELLS; MESSENGER-RNA; LENTIVIRAL VECTORS; MOUSE MODEL; TRANSGENE EXPRESSION; LIPID NANOPARTICLES; DIRECTED EVOLUTION; PACKAGING CAPACITY; RECEPTOR FOOTPRINT;
D O I
10.1016/j.cell.2022.03.045
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
In vivo gene editing therapies offer the potential to treat the root causes of many genetic diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to safely and efficiently deliver gene editing agents to relevant organs and tissues in vivo. Here, we review current delivery technologies that have been used to enable therapeutic in vivo gene editing, including viral vectors, lipid nanoparticles, and virus-like par-ticles. Since no single delivery modality is likely to be appropriate for every possible application, we compare the benefits and drawbacks of each method and highlight opportunities for future improvements.
引用
收藏
页码:2806 / 2827
页数:22
相关论文
共 50 条
  • [1] In Vivo Delivery of Gene Editing Agents
    不详
    [J]. CLINICAL PHARMACOLOGY & THERAPEUTICS, 2022, 111 (05) : 987 - 987
  • [2] Therapeutic Genome Editing and In Vivo Delivery
    Amanda Catalina Ramirez-Phillips
    Dexi Liu
    [J]. The AAPS Journal, 23
  • [3] Therapeutic Genome Editing and In Vivo Delivery
    Ramirez-Phillips, Amanda Catalina
    Liu, Dexi
    [J]. AAPS JOURNAL, 2021, 23 (04):
  • [4] In Vivo Delivery Systems for Therapeutic Genome Editing
    Wang, Luyao
    Li, Fangfei
    Dang, Lei
    Liang, Chao
    Wang, Chao
    He, Bing
    Liu, Jin
    Li, Defang
    Wu, Xiaohao
    Xu, Xuegong
    Lu, Aiping
    Zhang, Ge
    [J]. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2016, 17 (05)
  • [5] In Vivo Delivery of CRISPR/Cas9 for Therapeutic Gene Editing: Progress and Challenges
    Mout, Rubul
    Ray, Moumita
    Lee, Yi-Wei
    Scaletti, Federica
    Rotello, Vincent M.
    [J]. BIOCONJUGATE CHEMISTRY, 2017, 28 (04) : 880 - 884
  • [6] Enhancement of Gene Editing and Base Editing with Therapeutic Ribonucleoproteins through In Vivo Delivery Based on Absorptive Silica Nanoconstruct
    Kim, Seongchan
    Jeong, You Kyeong
    Cho, Chang Sik
    Lee, SeokHoon
    Sohn, Chang Ho
    Kim, Jeong Hun
    Jeong, Youngdo
    Jo, Dong Hyun
    Bae, Sangsu
    Lee, Hyojin
    [J]. ADVANCED HEALTHCARE MATERIALS, 2023, 12 (04)
  • [7] Therapeutic gene editing: delivery and regulatory perspectives
    Gayong Shim
    Dongyoon Kim
    Gyu Thae Park
    Hyerim Jin
    Soo-Kyung Suh
    Yu-Kyoung Oh
    [J]. Acta Pharmacologica Sinica, 2017, 38 : 738 - 753
  • [8] Therapeutic gene editing: delivery and regulatory perspectives
    Shim, Gayong
    Kim, Dongyoon
    Park, Gyu Thae
    Jin, Hyerim
    Suh, Soo-Kyung
    Oh, Yu-Kyoung
    [J]. ACTA PHARMACOLOGICA SINICA, 2017, 38 (06) : 738 - 753
  • [9] IN VIVO GENOME EDITING: FROM PROOF-OF-CONCEPT TO THERAPEUTIC DELIVERY
    Yin, Hao
    [J]. PEDIATRIC PULMONOLOGY, 2016, 51 : 139 - 139
  • [10] Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents
    Gao, Xue
    Tao, Yong
    Lamas, Veronica
    Huang, Mingqian
    Yeh, Wei-Hsi
    Pan, Bifeng
    Hu, Yu-Juan
    Hu, Johnny H.
    Thompson, David B.
    Shu, Yilai
    Li, Yamin
    Wang, Hongyang
    Yang, Shiming
    Xu, Qiaobing
    Polley, Daniel B.
    Liberman, M. Charles
    Kong, Wei-Jia
    Holt, Jeffrey R.
    Chen, Zheng-Yi
    Liu, David R.
    [J]. NATURE, 2018, 553 (7687) : 217 - +