Results 61 to 70 of about 116,783 (314)

High-throughput screening identifies compounds that enhance lentiviral transduction [PDF]

open access: yesGene Therapy, 2014
A difficulty in the field of gene therapy is the need to increase the susceptibility of hematopoietic stem cells (HSCs) to ex vivo genetic manipulation. To overcome this obstacle a high-throughput screen was performed to identify compounds that could enhance the transduction of target cells by lentiviral vectors.
J M, Johnston   +7 more
openaire   +2 more sources

Predictors of response and rational combinations for the novel MCL‐1 inhibitor MIK665 in acute myeloid leukemia

open access: yesMolecular Oncology, EarlyView.
This study characterizes the responses of primary acute myeloid leukemia (AML) patient samples to the MCL‐1 inhibitor MIK665. The results revealed that monocytic differentiation is associated with MIK665 sensitivity. Conversely, elevated ABCB1 expression is a potential biomarker of resistance to the treatment, which can be overcome by the combination ...
Joseph Saad   +17 more
wiley   +1 more source

Efficient Transduction and Expansion of Ovine Macrophages for Gene Therapy Implementations

open access: yesVeterinary Sciences, 2018
A number of bacteria provoking zoonotic diseases present intracellular survival and a host cell tropism limited to the monocyte/macrophage lineage. Thus, infection is rendered difficult to eradicate, causing chronic inflammatory reactions to the host and
Garyfalia Karponi   +3 more
doaj   +1 more source

Improved in-vivo airway gene transfer via magnetic-guidance, with protocol development informed by synchrotron imaging

open access: yesScientific Reports, 2022
Gene vectors to treat cystic fibrosis lung disease should be targeted to the conducting airways, as peripheral lung transduction does not offer therapeutic benefit. Viral transduction efficiency is directly related to the vector residence time.
Martin Donnelley   +13 more
doaj   +1 more source

Intrabody Gene Therapy Ameliorates Motor, Cognitive, and Neuropathological Symptoms in Multiple Mouse Models of Huntington's Disease [PDF]

open access: yes, 2009
Huntington's disease (HD) is an autosomal dominant neurodegenerative disease resulting from the expansion of a glutamine repeat in the huntingtin (Htt) protein.
Ko, Jan   +2 more
core   +2 more sources

The neural crest‐associated gene ERRFI1 is involved in melanoma progression and resistance toward targeted therapy

open access: yesMolecular Oncology, EarlyView.
ERRFI1, a neural crest (NC)‐associated gene, was upregulated in melanoma and negatively correlated with the expression of melanocytic differentiation markers and the susceptibility of melanoma cells toward BRAF inhibitors (BRAFi). Knocking down ERRFI1 significantly increased the sensitivity of melanoma cells to BRAFi.
Nina Wang   +8 more
wiley   +1 more source

Staurosporine Increases Lentiviral Vector Transduction Efficiency of Human Hematopoietic Stem and Progenitor Cells

open access: yesMolecular Therapy: Methods & Clinical Development, 2018
Lentiviral vector (LVV)-mediated transduction of human CD34+ hematopoietic stem and progenitor cells (HSPCs) holds tremendous promise for the treatment of monogenic hematological diseases.
Gretchen Lewis   +10 more
doaj   +1 more source

The production and development of H7 Influenza virus pseudotypes for the study of humoral responses against avian viruses [PDF]

open access: yes, 2013
In recent years, high pathogenicity avian influenza (HPAI) virus, H5N1, low pathogenicity avian influenza (LPAI) virus, H9N2, and both HPAI and LPAI H7 viruses have proved devastating for the affected economies reliant on poultry industry, and have posed
Böttcher-Frieberthäuser, Eva   +5 more
core   +3 more sources

Intein‐based modular chimeric antigen receptor platform for specific CD19/CD20 co‐targeting

open access: yesMolecular Oncology, EarlyView.
CARtein is a modular CAR platform that uses split inteins to splice antigen‐recognition modules onto a universal signaling backbone, enabling precise, scarless assembly without re‐engineering signaling domains. Deployed here against CD19 and CD20 in B‐cell malignancies, the design supports flexible multi‐antigen targeting to boost T‐cell activation and
Pablo Gonzalez‐Garcia   +9 more
wiley   +1 more source

Gene Expression Profile Changes After Short-activating RNA-mediated Induction of Endogenous Pluripotency Factors in Human Mesenchymal Stem Cells [PDF]

open access: yes, 2012
It is now recognized that small noncoding RNA sequences have the ability to mediate transcriptional activation of specific target genes in human cells.
Alluin, J   +7 more
core   +4 more sources

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