Promoter keyholes enable specific and persistent multi-gene expression programs in primary T cells without genome modification [PDF]
Non-invasive epigenome editing is a promising strategy for engineering gene expression programs, yet potency, specificity, and persistence remain challenging.
Acosta, Reyes+25 more
core
Solid-phase-assisted synthesis of targeting peptide-PEG-oligo(ethane amino)amides for receptor-mediated gene delivery. [PDF]
In the forthcoming era of cancer gene therapy, efforts will be devoted to the development of new efficient and non-toxic gene delivery vectors. In this regard, the use of Fmoc/Boc-protected oligo(ethane amino)acids as building blocks for solid-phase ...
Akinc+78 more
core +3 more sources
The role of host PrP in Transmissible Spongiform Encephalopathies [PDF]
PrP has a central role in the Transmissible Spongiform Encephalopathies (TSEs), and mutations and polymorphisms in host PrP can profoundly alter the host's susceptibility to a TSE agent.
.Nazor+58 more
core +3 more sources
The products of the Cdx genes, Cdx1, Cdx2 and Cdx4, play multiple roles in early vertebrate development, and have been proposed to serve to relay signaling information from Wnt, RA and FGF pathways to orchestrate events related to anterior-posterior vertebral patterning and axial elongation.
Joanne G.A. Savory+3 more
openaire +3 more sources
Inferring Disease and Gene Set Associations with Rank Coherence in Networks [PDF]
A computational challenge to validate the candidate disease genes identified in a high-throughput genomic study is to elucidate the associations between the set of candidate genes and disease phenotypes. The conventional gene set enrichment analysis often fails to reveal associations between disease phenotypes and the gene sets with a short list of ...
arxiv
Gene therapy targeting SARM1 blocks pathological axon degeneration in mice [PDF]
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
Life cycle studies of the hexose transporter of Plasmodium species and genetic validation of their essentiality [PDF]
A Plasmodium falciparum hexose transporter (PfHT) has previously been shown to be a facilitative glucose and fructose transporter. Its expression in Xenopus laevis oocytes and the use of a glucose analogue inhibitor permitted chemical validation of PfHT ...
Doerig, C+6 more
core +2 more sources
Gene Targeting with Viral Vectors
Genetic manipulation of cells for scientific and therapeutic goals can be achieved by both gene-addition and gene-targeting methods. Gene targeting precisely alters a gene in its natural chromosome location, providing distinct advantages over gene-addition approaches.
Paul C. Hendrie, David W. Russell
openaire +3 more sources
Targeting class I histone deacetylases by the novel small molecule inhibitor 4SC-202 blocks oncogenic hedgehog-GLI signaling and overcomes smoothened inhibitor resistance [PDF]
Aberrant activation of Hedgehog (HH)/GLI signaling is causally involved in numerous human malignancies, including basal cell carcinoma (BCC) and medulloblastoma. HH pathway antagonists targeting smoothened (SMO), an essential effector of canonical HH/GLI
Aberger, Fritz+12 more
core +1 more source
Conditional gene targeting. [PDF]
Rajewsky, K.+6 more
openaire +5 more sources