Viral vector platforms within the gene therapy landscape
Throughout its 40-year history, the field of gene therapy has been marked by many
transitions. It has seen great strides in combating human disease, has given hope to patients …
transitions. It has seen great strides in combating human disease, has given hope to patients …
Gene therapy: trials and tribulations
N Somia, IM Verma - Nature Reviews Genetics, 2000 - nature.com
The art and science of gene therapy has received much attention of late. The tragic death of
18-year-old Jesse Gelsinger, a volunteer in a Phase I clinical trial, has overshadowed the …
18-year-old Jesse Gelsinger, a volunteer in a Phase I clinical trial, has overshadowed the …
A robust and high-throughput Cre reporting and characterization system for the whole mouse brain
L Madisen, TA Zwingman, SM Sunkin, SW Oh… - Nature …, 2010 - nature.com
The Cre/lox system is widely used in mice to achieve cell-type-specific gene expression.
However, a strong and universally responding system to express genes under Cre control is …
However, a strong and universally responding system to express genes under Cre control is …
Dissecting immune circuits by linking CRISPR-pooled screens with single-cell RNA-seq
In multicellular organisms, dedicated regulatory circuits control cell type diversity and
responses. The crosstalk and redundancies within these circuits and substantial cellular …
responses. The crosstalk and redundancies within these circuits and substantial cellular …
Germline transmission and tissue-specific expression of transgenes delivered by lentiviral vectors
C Lois, EJ Hong, S Pease, EJ Brown, D Baltimore - science, 2002 - science.org
Single-cell mouse embryos were infected in vitro with recombinant lentiviral vectors to
generate transgenic mice carrying the green fluorescent protein (GFP) gene driven by a …
generate transgenic mice carrying the green fluorescent protein (GFP) gene driven by a …
BTN3A3 evasion promotes the zoonotic potential of influenza A viruses
Spillover events of avian influenza A viruses (IAVs) to humans could represent the first step
in a future pandemic. Several factors that limit the transmission and replication of avian IAVs …
in a future pandemic. Several factors that limit the transmission and replication of avian IAVs …
Refinement of tools for targeted gene expression in Drosophila
BD Pfeiffer, TTB Ngo, KL Hibbard, C Murphy, A Jenett… - Genetics, 2010 - academic.oup.com
A wide variety of biological experiments rely on the ability to express an exogenous gene in
a transgenic animal at a defined level and in a spatially and temporally controlled pattern …
a transgenic animal at a defined level and in a spatially and temporally controlled pattern …
Distinct morphological stages of dentate granule neuron maturation in the adult mouse hippocampus
C Zhao, EM Teng, RG Summers, G Ming… - Journal of …, 2006 - Soc Neuroscience
Adult neurogenesis in the dentate gyrus may contribute to hippocampus-dependent
functions, yet little is known about when and how newborn neurons are functional because …
functions, yet little is known about when and how newborn neurons are functional because …
Molecular evolution of a novel hyperactive Slee** Beauty transposase enables robust stable gene transfer in vertebrates
L Mátés, MKL Chuah, E Belay, B Jerchow, N Manoj… - Nature …, 2009 - nature.com
Abstract The Slee** Beauty (SB) transposon is a promising technology platform for gene
transfer in vertebrates; however, its efficiency of gene insertion can be a bottleneck in …
transfer in vertebrates; however, its efficiency of gene insertion can be a bottleneck in …
Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease
Lentiviral delivery of glial cell line–derived neurotrophic factor (lenti-GDNF) was tested for its
trophic effects upon degenerating nigrostriatal neurons in nonhuman primate models of …
trophic effects upon degenerating nigrostriatal neurons in nonhuman primate models of …