Related references
Note: Only part of the references are listed.Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit
Meaghan Van Alstyne et al.
NATURE NEUROSCIENCE (2021)
Transient rest restores functionality in exhausted CAR-T cells through epigenetic remodeling
Evan W. Weber et al.
SCIENCE (2021)
A reversible RNA on-switch that controls gene expression of AAV-delivered therapeutics in vivo
Guocai Zhong et al.
NATURE BIOTECHNOLOGY (2020)
A Small-Molecule-Responsive Riboswitch Enables Conditional Induction of Viral Vector-Mediated Gene Expression in Mice
Benjamin Strobel et al.
ACS SYNTHETIC BIOLOGY (2020)
Identification of preexisting adaptive immunity to Cas9 proteins in humans
Carsten T. Charlesworth et al.
NATURE MEDICINE (2019)
Next-generation AAV vectors-do not judge a virus (only) by its cover
Claire Domenger et al.
HUMAN MOLECULAR GENETICS (2019)
High levels of AAV vector integration into CRISPR-induced DNA breaks
Killian S. Hanlon et al.
NATURE COMMUNICATIONS (2019)
AAV-Mediated Progranulin Delivery to a Mouse Model of Progranulin Deficiency Causes T Cell-Mediated Toxicity
Defne A. Amado et al.
MOLECULAR THERAPY (2019)
High prevalence of Streptococcus pyogenes Cas9-reactive T cells within the adult human population
Dimitrios L. Wagner et al.
NATURE MEDICINE (2019)
Mechanistic studies of a small-molecule modulator of SMN2 splicing
Jingxin Wang et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2018)
Non-AUG translation: a new start for protein synthesis in eukaryotes
Michael G. Kearse et al.
GENES & DEVELOPMENT (2017)
In vivo genome editing and organoid transplantation models of colorectal cancer and metastasis
Jatin Roper et al.
NATURE BIOTECHNOLOGY (2017)
Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems
Ken Y. Chan et al.
NATURE NEUROSCIENCE (2017)
Human splicing diversity and the extent of unannotated splice junctions across human RNA-seq samples on the Sequence Read Archive
Abhinav Nellore et al.
GENOME BIOLOGY (2016)
Quantitative visualization of alternative exon expression from RNA-seq data
Yarden Katz et al.
BIOINFORMATICS (2015)
Inducible in vivo genome editing with CRISPR-Cas9
Lukas E. Dow et al.
NATURE BIOTECHNOLOGY (2015)
SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice
James Palacino et al.
NATURE CHEMICAL BIOLOGY (2015)
Phthalimide conjugation as a strategy for in vivo target protein degradation
Georg E. Winter et al.
SCIENCE (2015)
SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy
Nikolai A. Naryshkin et al.
SCIENCE (2014)
Moderated estimation of fold change and dispersion for RNA-seq data with DESeq2
Michael I. Love et al.
GENOME BIOLOGY (2014)
Integrative Genomics Viewer (IGV): high-performance genomics data visualization and exploration
Helga Thorvaldsdottir et al.
BRIEFINGS IN BIOINFORMATICS (2013)
High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells
Yanfang Fu et al.
NATURE BIOTECHNOLOGY (2013)
Integrative genomics viewer
James T. Robinson et al.
NATURE BIOTECHNOLOGY (2011)
Synthetic translational regulation by an L7Ae-kink-turn RNP switch
Hirohide Saito et al.
NATURE CHEMICAL BIOLOGY (2010)
A robust and high-throughput Cre reporting and characterization system for the whole mouse brain
Linda Madisen et al.
NATURE NEUROSCIENCE (2010)
Genome-Wide Analysis in Vivo of Translation with Nucleotide Resolution Using Ribosome Profiling
Nicholas T. Ingolia et al.
SCIENCE (2009)
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state
Brian D. Brown et al.
NATURE BIOTECHNOLOGY (2007)
Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer
Brian D. Brown et al.
NATURE MEDICINE (2006)
Exogenous control of mammalian gene expression through modulation of RNA self-cleavage
L Yen et al.
NATURE (2004)
Directed evolution of ligand dependence: Small-molecule-activated protein splicing
AR Buskirk et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2004)
Mild overexpression of MeCP2 causes a progressive neurological disorder in mice
AL Collins et al.
HUMAN MOLECULAR GENETICS (2004)
The human genome browser at UCSC
WJ Kent et al.
GENOME RESEARCH (2002)
Disruption of an SF2/ASF-dependent exonic splicing enhancer in SMN2 causes spinal muscular atrophy in the absence of SMN1
L Cartegni et al.
NATURE GENETICS (2002)
Share Paper
