Modular Lentiviral Vectors for Highly Efficient Transgene Expression in Resting Immune Cells
Gene/cell therapies are promising strategies for the many presently incurable diseases. A key step in this process is the efficient delivery of genes and gene-editing enzymes to many cell types that may be resistant to lentiviral vector transduction ...
Christina Fichter +11 more
doaj +1 more source
A requirement for STAG2 in replication fork progression creates a targetable synthetic lethality in cohesin-mutant cancers. [PDF]
Cohesin is a multiprotein ring that is responsible for cohesion of sister chromatids and formation of DNA loops to regulate gene expression. Genomic analyses have identified that the cohesin subunit STAG2 is frequently inactivated by mutations in cancer.
Ashworth, Alan +4 more
core +2 more sources
Gene delivery to pancreatic exocrine cells
Background Effective gene transfer to the pancreas or to pancreatic cells has remained elusive although it is essential for studies of genetic lineage tracing and modulation of gene expression. Different transduction methods and viral vectors were tested
Houbracken Isabelle +4 more
doaj +1 more source
Preclinical correction of human Fanconi anemia complementation group A bone marrow cells using a safety-modified lentiviral vector. [PDF]
One of the major hurdles for the development of gene therapy for Fanconi anemia (FA) is the increased sensitivity of FA stem cells to free radical-induced DNA damage during ex vivo culture and manipulation.
Adair, J +10 more
core +2 more sources
An efficient method for the transduction of primary pediatric glioma neurospheres
Pediatric high grade glioma (pHGG) and diffuse intrinsic pontine glioma (DIPG) are rare, but rapidly fatal malignancies of the central nervous system (CNS), and the leading cause of cancer-related death in children.
Michaël H. Meel +4 more
doaj +1 more source
Anti-Apoptotic Effects of Lentiviral Vector Transduction Promote Increased Rituximab Tolerance in Cancerous B-Cells. [PDF]
Diffuse large B-cell lymphoma (DLBCL) is characterized by great genetic and clinical heterogeneity which complicates prognostic prediction and influences treatment efficacy. The most common regimen, R-CHOP, consists of a combination of anthracycline- and
Benyamin Ranjbar +6 more
doaj +1 more source
Driving DNA transposition by lentiviral protein transduction [PDF]
Gene vectors derived from DNA transposable elements have become powerful molecular tools in biomedical research and are slowly moving into the clinic as carriers of therapeutic genes. Conventional uses of DNA transposon-based gene vehicles rely on the intracellular production of the transposase protein from transfected nucleic acids.
Cai, Yujia, Mikkelsen, Jacob Giehm
openaire +2 more sources
Using lentiviral vector products in clinical applications requires an accurate method for measuring transduction titer. For vectors lacking a marker gene, quantitative polymerase chain reaction is used to evaluate the number of vector DNA copies in ...
Michele E Murphy +4 more
doaj +1 more source
Expression of a large coding sequence: Gene therapy vectors for Ataxia Telangiectasia
Ataxia telangiectasia is a monogenetic disorder caused by mutations in the ATM gene. Its encoded protein kinase ATM plays a fundamental role in DNA repair of double strand breaks (DSBs).
Tanja Hirch +6 more
doaj +1 more source
Immunogenicity of targeted lentivectors [PDF]
To increase the safety and possibly efficacy of HIV-1 derived lentivectors (LVs) as an anti-cancer vaccine, we recently developed the Nanobody (Nb) display technology to target LVs to antigen presenting cells (APCs).
Adotévi +67 more
core +3 more sources

