rAAV-compatible MiniPromoters for restricted expression in the brain and eye

Stability and Safety of an AAV Vector for TreatingRPGR-ORF15X-Linked Retinitis Pigmentosa

(2015) Wen-Tao Deng et al.   HUMAN GENE THERAPY

Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness

(2015) Miranda L. Scalabrino et al.   HUMAN MOLECULAR GENETICS

Paradigm shifts in genomics through the FANTOM projects

(2015) Michiel de Hoon et al.   MAMMALIAN GENOME

The Expression Pattern of Systemically Injected AAV9 in the Developing Mouse Retina Is Determined by Age

(2015) Leah C Byrne et al.   MOLECULAR THERAPY

Gene therapy returns to centre stage

(2015) Luigi Naldini   NATURE

Transgenic expression of the proneural transcription factor Ascl1 in Müller glia stimulates retinal regeneration in young mice

(2015) Yumi Ueki et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Targeted genetic manipulations of neuronal subtypes using promoter-specific combinatorial AAVs in wild-type animals

(2015) Heinrich S. Gompf et al.   Frontiers in Behavioral Neuroscience

Development of gene therapy for treatment of age-related macular degeneration

(2014) Anne Louise Askou   ACTA OPHTHALMOLOGICA

Characterization of dsRed2-positive cells in the doublecortin-dsRed2 transgenic adult rat retina

(2014) A. Trost et al.   HISTOCHEMISTRY AND CELL BIOLOGY

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial

(2014) Robert E MacLaren et al.   LANCET

Gene Therapy for the Nervous System: Challenges and New Strategies

(2014) Casey A. Maguire et al.   Neurotherapeutics

Creating and validating cis-regulatory maps of tissue-specific gene expression regulation

(2014) Timothy R. O'Connor et al.   NUCLEIC ACIDS RESEARCH

The UCSC Genome Browser database: 2015 update

(2014) Kate R. Rosenbloom et al.   NUCLEIC ACIDS RESEARCH

Optogenetic astrocyte activation modulates response selectivity of visual cortex neurons in vivo

(2014) Gertrudis Perea et al.   Nature Communications

Biology of adeno-associated viral vectors in the central nervous system

(2014) Giridhar Murlidharan et al.   Frontiers in Molecular Neuroscience

Non-coding-regulatory regions of human brain genes delineated by bacterial artificial chromosome knock-in mice

(2013) Jean-François Schmouth et al.   BMC BIOLOGY

JASPAR 2014: an extensively expanded and updated open-access database of transcription factor binding profiles

(2013) Anthony Mathelier et al.   NUCLEIC ACIDS RESEARCH

Analysis of Transduction Efficiency, Tropism and Axonal Transport of AAV Serotypes 1, 2, 5, 6, 8 and 9 in the Mouse Brain

(2013) Dominik F. Aschauer et al.   PLoS One

Predicting tissue specific cis-regulatory modules in the human genome using pairs of co-occurring motifs

(2012) Hani Z Girgis et al.   BMC BIOINFORMATICS

Targeting of >1.5 Mb of Human DNA into the Mouse X Chromosome Reveals Presence of cis -Acting Regulators of Epigenetic Silencing

(2012) Christine Yang et al.   GENETICS

Gene therapy for Leber congenital amaurosis: advances and future directions

(2012) Robert B. Hufnagel et al.   GRAEFES ARCHIVE FOR CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY

Beyond knockouts: cre resources for conditional mutagenesis

(2012) Stephen A. Murray et al.   MAMMALIAN GENOME

The mammalian gene function resource: the international knockout mouse consortium

(2012) Allan Bradley et al.   MAMMALIAN GENOME

An integrated encyclopedia of DNA elements in the human genome

(2012)   NATURE

ENCODE Data in the UCSC Genome Browser: year 5 update

(2012) Kate R. Rosenbloom et al.   NUCLEIC ACIDS RESEARCH

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

Novel adeno-associated viral vectors for retinal gene therapy

(2011) L H Vandenberghe et al.   GENE THERAPY

Optimizing Promoters for Recombinant Adeno-Associated Virus-Mediated Gene Expression in the Peripheral and Central Nervous System Using Self-Complementary Vectors

(2011) Steven J. Gray et al.   HUMAN GENE THERAPY

Charting a course for genomic medicine from base pairs to bedside

(2011) Eric D. Green et al.   NATURE

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

Viral vectors for gene delivery to the central nervous system

(2011) Thomas B. Lentz et al.   NEUROBIOLOGY OF DISEASE

ENCODE whole-genome data in the UCSC Genome Browser: update 2012

(2011) K. R. Rosenbloom et al.   NUCLEIC ACIDS RESEARCH

A Genetically Encoded Tag for Correlated Light and Electron Microscopy of Intact Cells, Tissues, and Organisms

(2011) Xiaokun Shu et al.   PLOS BIOLOGY

A User's Guide to the Encyclopedia of DNA Elements (ENCODE)

(2011)   PLOS BIOLOGY

Perspectives on the RNA polymerase II core promoter

(2011) James T. Kadonaga   Wiley Interdisciplinary Reviews-Developmental Biology

A regulatory toolbox of MiniPromoters to drive selective expression in the brain

(2010) E. Portales-Casamar et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS

(2009) M A Zanta-Boussif et al.   GENE THERAPY

An anatomic gene expression atlas of the adult mouse brain

(2009) Lydia Ng et al.   NATURE NEUROSCIENCE

ENCODE whole-genome data in the UCSC Genome Browser

(2009) K. R. Rosenbloom et al.   NUCLEIC ACIDS RESEARCH

Identification of a set of genes showing regionally enriched expression in the mouse brain

(2008) Cletus A D'Souza et al.   BMC NEUROSCIENCE

Next generation tools for high-throughput promoter and expression analysis employing single-copy knock-ins at the Hprt1 locus

(2008) G.S. Yang et al.   GENOMICS

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY