Towards mutation-independent silencing of genes involved in retinal degeneration by RNA interference

No abstract is available for this paper.

• Publication year

  2005

• Venue

  Gene Therapy

• Publication date

  2005-08-01

• Fields of study

  Biology, Medicine

• Identifiers
  DOI 10.1038/sj.gt.3302512PMID 15877050
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

• Rapid and Efficient Site-directed Mutagenesis by the Single-tube Megaprimer PCR Method.

  2006cited by this paper
• RNA silencing: Methods and protocols

  2006cited by this paper
• Mutation Independent Knock Down of Rhodopsin by Short Hairpin RNAs (shRNA) and Replacement by a shRNA–resistant Rhodopsin Transgene

  2004cited by this paper
• Rapid and efficient site-directed mutagenesis by the single-tube megaprimer PCR method

  2004cited by this paper
• siRNA-mediated gene silencing in vitro and in vivo

  2002cited by this paper
• RNA silencing.

  2002cited by this paper
• Hammerhead ribozymes designed to cleave all human rod opsin mRNAs which cause autosomal dominant retinitis pigmentosa.

  2002cited by this paper
• A System for Stable Expression of Short Interfering RNAs in Mammalian Cells

  2002cited by this paper
• A Rhodopsin Mutant Linked to Autosomal Dominant Retinitis Pigmentosa Is Prone to Aggregate and Interacts with the Ubiquitin Proteasome System*

  2002cited by this paper
• Ribozyme-based therapeutic approaches for autosomal dominant retinitis pigmentosa.

  2000cited by this paper
• Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa

  1998cited by this paper
• Strategems in vitro for gene therapies directed to dominant mutations.

  1997cited by this paper
• Inhibition of fibrillin 1 expression using U1 snRNA as a vehicle for the presentation of antisense targeting sequence.

  1997cited by this paper
• How photons start vision.

  1996cited by this paper
• Characterization of 911: A New Helper Cell Line for the Titration and Propagation of Early Region 1-Deleted Adenoviral Vectors

  1996cited by this paper
• Retinitis punctata albescens associated with the Arg135Trp mutation in the rhodopsin gene.

  1996cited by this paper
• Structure and function in rhodopsin: correct folding and misfolding in two point mutants in the intradiscal domain of rhodopsin identified in retinitis pigmentosa.

  1996cited by this paper
• Heterozygous missense mutation in the rhodopsin gene as a cause of congenital stationary night blindness

  1993cited by this paper

• Method for siRNA delivery in retina explants.

  2023cites this paper
• CRISPR/SaCas9-based gene editing rescues photoreceptor degeneration throughout a rhodopsin-associated autosomal dominant retinitis pigmentosa mouse model

  2023cites this paper
• New Method for Efficient siRNA Delivery in Retina Explants: Reverse Magnetofection.

  2021cites this paper
• Gene Therapy in Retinal Dystrophies

  2019cites this paper
• Systematic Screening, Rational Development, and Initial Optimization of Efficacious RNA Silencing Agents for Human Rod Opsin Therapeutics

  2019cites this paper
• Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy

  2018cites this paper
• A cellular high-throughput screening approach for therapeutic trans-cleaving ribozymes and RNAi against arbitrary mRNA disease targets.

  2016cites this paper
• Future Therapies for Retinitis Pigmentosa

  2014cites this paper
• Small‐interfering RNAs (siRNAs) as a promising tool for ocular therapy

  2013cites this paper
• Novel p.M96T variant of NRL and shRNA‐based suppression and replacement of NRL mutants associated with autosomal dominant retinitis pigmentosa

  2012cites this paper
• Mutation-independent rescue of a novel mouse model of Retinitis Pigmentosa

  2012cites this paper
• Gene therapy in animal models of autosomal dominant retinitis pigmentosa

  2012cites this paper
• Photobiomodulation in inherited retinal degeneration

  2012cites this paper
• Gene delivery of wild-type rhodopsin rescues retinal function in an autosomal dominant retinitis pigmentosa mouse model.

  2012cites this paper
• Artificial mirtron-mediated gene knockdown: functional DMPK silencing in mammalian cells.

  2012cites this paper
• Long-Term Rescue of Retinal Structure and Function by Rhodopsin RNA Replacement with a Single Adeno-Associated Viral Vector in P23H RHO Transgenic Mice

  2012cites this paper
• AAV Delivery of Wild-Type Rhodopsin Preserves Retinal Function in a Mouse Model of Autosomal Dominant Retinitis Pigmentosa

  2011cites this paper
• Retinal Degenerations: Genetics, Mechanisms, and Therapies

  2011cites this paper
• Variables and Strategies in Development of Therapeutic Post-Transcriptional Gene Silencing Agents

  2011cites this paper
• Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa

  2011cites this paper
• Rapid, cell-based toxicity screen of potentially therapeutic post-transcriptional gene silencing agents.

  2011cites this paper
• Adeno-Associated Virus-Mediated Rhodopsin Replacement Provides Therapeutic Benefit in Mice with a Targeted Disruption of the Rhodopsin Gene

  2010cites this paper
• Engineered zinc finger nuclease-mediated homologous recombination of the human rhodopsin gene.

  2010cites this paper
• AAV-mediated knockdown of Peripherin-2 in vivo using miRNA-based hairpins

  2010cites this paper
• Gene therapeutic approaches for dominant retinopathies.

  2010cites this paper
• Implantation und Explantation von inaktiven, epiretinal fixierten Retina Implant Systemen am Minipig

  2010cites this paper
• Viral vector-mediated RNA interference in the retina

  2010cites this paper
• Retinitis pigmentosa

  2009cites this paper
• Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy.

  2009cites this paper
• In vitro analysis of ribozyme-mediated knockdown of an ADRP associated rhodopsin mutation.

  2008influential citation
• Gene therapy for mouse models of ADRP.

  2008cites this paper
• Allele‐specific silencing of the dominant disease allele in sialuria by RNA interference

  2008cites this paper
• A transgenic mouse model for gene therapy of rhodopsin-linked Retinitis Pigmentosa.

  2008cites this paper
• Nanoparticle applications in ocular gene therapy.

  2008cites this paper
• Bottlenecks in development of retinal therapeutic post-transcriptional gene silencing agents.

  2008cites this paper
• AAV-mediated gene therapy for retinal disorders: from mouse to man

  2008cites this paper
• Rescue of Sight by Gene Therapy—Closer than It May Appear

  2007cites this paper
• Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme.

  2007cites this paper
• Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery.

  2007cites this paper
• Cell-based therapy for retina degeneration: the promise of a cure.

  2007cites this paper
• RNA interference-mediated suppression and replacement of human rhodopsin in vivo.

  2007cites this paper
• RNAi‐based suppression and replacement of rds‐peripherin in retinal organotypic culture

  2006influential citation
• Efficient Non-Viral Ocular Gene Transfer with Compacted DNA Nanoparticles

  2006cites this paper
• Inhibition of choroidal neovascularization by adenovirus-mediated delivery of short hairpin RNAs targeting VEGF as a potential therapy for AMD.

  2006cites this paper
• Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model.

  2006cites this paper
• Retinitis pigmentosa.

  2006cites this paper
• Application of genetic approaches to ocular disease.

  2006cites this paper
• RNA based gene therapy for dominantly inherited diseases.

  2006cites this paper
• AAV-mediated gene transfer for retinal diseases

  2006cites this paper