Intron retention is regulated by altered MeCP2-mediated splicing factor recruitment
Nature Communications, 2017 Intron retention is a conserved mechanism that controls gene expression but its regulation is poorly understood. Here, the authors provide evidence that DNA methylation regulates intron retention and find reduced MeCP2 occupancy and splicing factor ...
Justin J. -L. Wong +11 more
doaj +1 more source
In vivo manipulation of interleukin-2 expression by a retroviral tetracycline (tet)-regulated system [PDF]
, 2006 We have used the tetracycline (tet)-regulated system as described previously to evaluate the applicability of controlled gene expression in cancer gene therapy. As a model gene, we used the human interleukin-2 (IL-2) gene, which has been placed under the
Pitzer, Claudia +4 more
core
New directions in cellular therapy of cancer: a summary of the summit on cellular therapy for cancer [PDF]
, 2012 A summit on cellular therapy for cancer discussed and presented advances related to the use of adoptive cellular therapy for melanoma and other cancers. The summit revealed that this field is advancing rapidly.
A Lundqvist +48 more
core +4 more sources
Use of integrase-minus lentiviral vector for transient expression [PDF]
, 2012 Objective: Lentivirus-derived vectors are among the most promising viral vectors for gene therapy which is currently available, but their use in clinical practice is limited due to associated risk of insertional mutagenesis.
Azadeh, H. +5 more
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Adeno‐associated virus serotype 1‐based gene therapy for FTD caused by GRN mutations
Annals of Clinical and Translational Neurology, 2020 Objective Dominant loss‐of‐function mutations in the gene encoding the lysosomal protein, progranulin, cause 5‐10% of frontotemporal dementia cases. As progranulin undergoes secretion and endocytosis, a small number of progranulin‐expressing cells can ...
Christian Hinderer +6 more
doaj +1 more source
AAV gene replacement therapy for treating MPS IIIC: Facilitating bystander effects via EV‐mRNA cargo
Journal of Extracellular VesiclesMPS IIIC is a lysosomal storage disease caused by mutations in heparan‐α‐glucosaminide N‐acetyltransferase (HGSNAT), for which no treatment is available.
Tierra A. Bobo +6 more
doaj +1 more source
Lack of RNA-DNA oligonucleotide (chimeraplast) mutagenic activity in mouse embryos [PDF]
, 2005 There are numerous reports of the use of RNA-DNA oligonucleoticles (chimeraplasts) to correct point mutations in vitro and in vivo, including the human apolipoprotein E gene (ApoE).
Owen, JS, Simons, JP, Tagalakis, AD
core +1 more source
mBio, 2019 Secretion of interleukin-1β (IL-1β) represents a fundamental innate immune response to microbial infection that, at the molecular level, occurs following activation of proteolytic caspases that cleave the immature protein into a secretable form.
Sara Botto +8 more
doaj +1 more source
Habermas, Human Agency, and Human Genetic Enhancement: The Grown, the Made, and Responsibility for Actions [PDF]
, 2012 Recent developments in genomic science hold out the tantalizing prospect of soon being able to treat and prevent a wide variety of medical conditions through gene therapy. In time, it may be possible to use similar techniques not simply to combat disease
Habermas +4 more
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Decrease in gyrase A protein expression in _E. coli_ cells inhibited by antisense ribozymes [PDF]
, 2008 RNase P complexed with external guide sequence (EGS) represents a novel nucleic-acid-based gene interference approach to modulate gene expression. Nucleic acid-based gene interference technologies represent promising strategies for specific inhibition of
Malathi Raghunathan +1 more
core +1 more source