Results 81 to 90 of about 12,501,640 (402)

In vitro correction of cystic fibrosis epithelial cell lines by small fragment homologous replacement (SFHR) technique

open access: yesBMC Medical Genetics, 2002
Background SFHR (small fragment homologous replacement)-mediated targeting is a process that has been used to correct specific mutations in mammalian cells. This process involves both chemical and cellular factors that are not yet defined.
Bonifazi Emanuela   +7 more
doaj   +1 more source

Screening for the optimal siRNA targeting a novel gene (HA117) and construction and evaluation of a derivative recombinant adenovirus [PDF]

open access: yes, 2010
We found a novel gene named as HA117 in our previous research. At this study, we screened for an optimal siRNA targeting the novel gene HA117 using the pSOS-HUS method, verified the results of pSOS-HUS siRNA screening for optimal affinity for the target ...
Gaihuan Zheng   +4 more
core   +1 more source

Non-Canonical Thinking for Targeting ALK-Fusion Onco-Proteins in Lung Cancer. [PDF]

open access: yes, 2017
Anaplastic lymphoma kinase (ALK) gene rearrangements have been identified in lung cancer at 3-7% frequency, thus representing an important subset of genetic lesions that drive oncogenesis in this disease. Despite the availability of multiple FDA-approved
Bivona, Trever G   +2 more
core   +3 more sources

Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

open access: yesNature, 2014
Site-specific gene addition can allow stable transgene expression for gene therapy. When possible, this is preferred over the use of promiscuously integrating vectors, which are sometimes associated with clonal expansion and oncogenesis.
A. Barzel   +10 more
semanticscholar   +1 more source

Biological and technical complexities in analyzing extracellular vesicle immune interactions in B‐cell malignancies

open access: yesFEBS Letters, EarlyView.
Extracellular vesicles (EVs) mediate intercellular communication in tumor immune microenvironments. However, their role in B‐cell malignancies remains poorly defined, owing to biological complexity and technical challenges in EV isolation and analysis.
Daniel Bachurski, Michael Hallek
wiley   +1 more source

Gene-carried hepatoma targeting complex induced high gene transfection efficiency with low toxicity and significant antitumor activity

open access: yesInternational Journal of Nanomedicine, 2012
Qing-Qing Zhao,1,2 Yu-Lan Hu,1 Yang Zhou,3 Ni Li,1 Min Han,1 Gu-Ping Tang,4 Feng Qiu,2 Yasuhiko Tabata,5 Jian-Qing Gao,11Institute of Pharmaceutics, Zhejiang University, Hangzhou, China; 2Department of Pharmacy, The First Affiliated Hospital of Chongqing
Zhao QQ   +8 more
doaj  

RNA-guided retargeting of Sleeping Beauty transposition in human cells

open access: yeseLife, 2020
An ideal tool for gene therapy would enable efficient gene integration at predetermined sites in the human genome. Here we demonstrate biased genome-wide integration of the Sleeping Beauty (SB) transposon by combining it with components of the CRISPR ...
Adrian Kovač   +5 more
doaj   +1 more source

A RNase P Ribozyme Inhibits Gene Expression and Replication of Hepatitis B Virus in Cultured Cells

open access: yesMicroorganisms, 2023
Hepatitis B virus (HBV), an international public health concern, is a leading viral cause of liver disease, such as hepatocellular carcinoma. Sequence-specific ribozymes derived from ribonuclease P (RNase P) catalytic RNA are being explored for gene ...
Bin Yan   +3 more
doaj   +1 more source

Gene therapy targeting SARM1 blocks pathological axon degeneration in mice [PDF]

open access: yes, 2019
Axonal degeneration (AxD) following nerve injury, chemotherapy, and in several neurological disorders is an active process driven by SARM1, an injury-activated NADase.
DiAntonio, Aaron   +8 more
core   +1 more source

Gene targeting using the Agrobacterium tumefaciens-mediated CRISPR-Cas system in rice

open access: yesRice, 2014
BackgroundThe type II clustered, regularly interspaced, short palindromic repeat (CRISPR)/ CRISPR-associated protein 9 (Cas9) system is a novel molecular tool for site-specific genome modification.
R. Xu   +6 more
semanticscholar   +1 more source

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