Abstract:
:Leukemias arise from genetic alterations in normal hematopoietic stem or progenitor cells, leading to impaired regulation of proliferation, differentiation, apoptosis, and survival of the transformed cells. With the advent of RNA interference (RNAi) and the short interfering RNA (siRNA) as its pharmacological mediator, it is becoming possible to modulate specific targets at will. This article summarizes current attempts to utilize RNAi reagents for therapy of leukemias, focusing on acute and chronic myeloid leukemia. We first present unique aspects of RNAi-mediated therapy, followed by a brief background on the delivery technology of RNAi reagents. The need for leukemia-specific delivery of siRNA is discussed by describing approaches that targeted agents to leukemic cells. Pharmacokinetics and biodistribution of RNAi agents are then presented, highlighting the critical issues pertinent to emerging siRNA therapy. Efforts to deliver specific RNAi therapies are then summarized in the context of expected clinical outcomes, focusing on limiting leukemic cell survival, sensitizing malignant cells to chemotherapy, mobilization of leukemic cells, and eradication of leukemic stem cells. We conclude with a perspective on the future of RNAi therapy, emphasizing the technological requirements and mechanistic challenges for clinical entry.
journal_name
Mol Ther Nucleic Acidsjournal_title
Molecular therapy. Nucleic acidsauthors
Landry B,Valencia-Serna J,Gul-Uludag H,Jiang X,Janowska-Wieczorek A,Brandwein J,Uludag Hdoi
10.1038/mtna.2015.13subject
Has Abstractpub_date
2015-05-12 00:00:00pages
e240issn
2162-2531pii
mtna201513journal_volume
4pub_type
杂志文章,评审abstract::PCTAIRE1/CDK16/PCTK1 plays critical roles in cancer cell proliferation and antiapoptosis. To advance our previously published in vitro results with PCTAIRE1 silencing, we examined the in vivo therapeutic potential of this approach by using small interfering RNA (siRNA) encapsulated by lipid nanoparticles. Therapy expe...
journal_title:Molecular therapy. Nucleic acids
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journal_title:Molecular therapy. Nucleic acids
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journal_title:Molecular therapy. Nucleic acids
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journal_title:Molecular therapy. Nucleic acids
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journal_title:Molecular therapy. Nucleic acids
pub_type: 杂志文章
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pub_type: 杂志文章
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journal_title:Molecular therapy. Nucleic acids
pub_type: 杂志文章
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journal_title:Molecular therapy. Nucleic acids
pub_type: 杂志文章
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pub_type: 杂志文章
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journal_title:Molecular therapy. Nucleic acids
pub_type: 杂志文章
doi:10.1016/j.omtn.2017.12.008
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journal_title:Molecular therapy. Nucleic acids
pub_type: 杂志文章
doi:10.1016/j.omtn.2017.10.007
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journal_title:Molecular therapy. Nucleic acids
pub_type: 杂志文章
doi:10.1038/mtna.2014.40
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pub_type: 杂志文章
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journal_title:Molecular therapy. Nucleic acids
pub_type: 杂志文章
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journal_title:Molecular therapy. Nucleic acids
pub_type: 杂志文章
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journal_title:Molecular therapy. Nucleic acids
pub_type: 杂志文章,评审
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journal_title:Molecular therapy. Nucleic acids
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journal_title:Molecular therapy. Nucleic acids
pub_type: 杂志文章
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更新日期:2019-09-06 00:00:00
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