The Potential of AAV-Mediated Gene Targeting for Gene and Cell Therapy Applications

No abstract is available for this paper.

• Publication year

  2015

• Venue

  Current Stem Cell Reports

• Publication date

  2015-01-16

• Fields of study

  Biology, Medicine, Engineering

• Identifiers
  DOI 10.1007/s40778-014-0001-1
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar

• No claims are published for this paper.

• No concepts are published for this paper.

• Exploiting the unique regenerative capacity of the liver to underpin cell and gene therapy strategies for genetic and acquired liver disease.

  2014cited by this paper
• Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

  2014cited by this paper
• Engineering adeno-associated viruses for clinical gene therapy

  2014cited by this paper
• The Mechanism of Gene Targeting in Human Somatic Cells

  2014cited by this paper
• Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity.

  2013cited by this paper
• The effects of polymorphisms on human gene targeting

  2013cited by this paper
• HLA engineering of human pluripotent stem cells.

  2013cited by this paper
• Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

  2013cited by this paper
• Genome engineering at the dawn of the golden age.

  2013influential reference
• Multiplex Genome Engineering Using CRISPR/Cas Systems

  2013cited by this paper
• DNA targeting specificity of RNA-guided Cas9 nucleases

  2013cited by this paper
• Normal collagen and bone production by gene-targeted human osteogenesis imperfecta iPSCs.

  2012cited by this paper
• The AAV Vector Toolkit: Poised at the Clinical Crossroads.

  2012cited by this paper
• Intracellular transport of recombinant adeno-associated virus vectors

  2012cited by this paper
• Induction of hepatocellular carcinoma by in vivo gene targeting

  2012cited by this paper
• Comparison of gene transfer to the murine liver following intraperitoneal and intraportal delivery of hepatotropic AAV pseudo-serotypes

  2012cited by this paper
• Targeted gene therapies: tools, applications, optimization

  2012cited by this paper
• Hematopoietic stem cell expansion: challenges and opportunities

  2012cited by this paper
• Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

  2011cited by this paper
• In vivo genome editing restores hemostasis in a mouse model of hemophilia

  2011cited by this paper
• AAV-mediated gene targeting methods for human cells

  2011cited by this paper
• Chromosomal position effects on AAV-mediated gene targeting

  2010cited by this paper
• Adeno‐associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo

  2010cited by this paper
• Engineering of human pluripotent stem cells by AAV-mediated gene targeting.

  2010cited by this paper
• Gene targeting of mutant COL1A2 alleles in mesenchymal stem cells from individuals with osteogenesis imperfecta.

  2008cited by this paper
• Human Gene Targeting Favors Insertions Over Deletions

  2008cited by this paper
• Potential of AAV vectors in the treatment of metabolic disease

  2008cited by this paper
• Adeno-associated virus serotypes: vector toolkit for human gene therapy.

  2006cited by this paper
• Gene targeting in vivo by adeno-associated virus vectors

  2006cited by this paper
• Observations of the carrier state in hamsters infected with Leptospira interrogans serotype pomona

  2005cited by this paper
• Gene targeting in mice: functional analysis of the mammalian genome for the twenty-first century

  2005cited by this paper
• Gene targeting with viral vectors.

  2005cited by this paper
• Gene Targeting by Adeno-Associated Virus Vectors Is Cell-Cycle Dependent

  2005cited by this paper
• Efficient PRNP Gene Targeting in Bovine Fibroblasts by Adeno-Associated Virus Vectors

  2004cited by this paper
• Gene Targeting in Stem Cells from Individuals with Osteogenesis Imperfecta

  2004cited by this paper
• Rapid Uncoating of Vector Genomes Is the Key toEfficient Liver Transduction with Pseudotyped Adeno-Associated VirusVectors

  2004cited by this paper
• Chromosomal Integration and Homologous Gene Targetingby Replication-Incompetent Vectors Based on the Autonomous ParvovirusMinute Virus ofMice

  2003cited by this paper
• Human Gene Targeting by Adeno-Associated Virus Vectors Is Enhanced by DNA Double-Strand Breaks

  2003cited by this paper
• Targeted transgene insertion into human chromosomes by adeno-associated virus vectors

  2002cited by this paper
• Introduction of single base substitutions at homologous chromosomal sequences by adeno-associated virus vectors.

  2001cited by this paper
• Rate limiting steps of AAV transduction and implications for human gene therapy.

  2001cited by this paper
• Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors.

  2000cited by this paper
• Design and Packaging of Adeno-Associated Virus Gene Targeting Vectors

  2000cited by this paper
• Production of High-Titer Recombinant Adeno-Associated Virus Vectors in the Absence of Helper Adenovirus

  1998cited by this paper
• Human gene targeting by viral vectors

  1998cited by this paper
• Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis

  1996cited by this paper
• T Lymphocyte-Directed Gene Therapy for ADA− SCID: Initial Trial Results After 4 Years

  1995cited by this paper
• Retroviral vectors.

  1994cited by this paper
• Adeno-associated virus vectors preferentially transduce cells in S phase.

  1994cited by this paper
• Correction of a deletion mutant by gene targeting with an adenovirus vector

  1993cited by this paper
• Use of adeno-associated virus as a general transduction vector for mammalian cells.

  1992cited by this paper
• Human gene therapy comes of age

  1992cited by this paper
• High-fidelity gene targeting in embryonic stem cells by using sequence replacement vectors

  1992cited by this paper
• Retrovirus-mediated gene transfer into CD4+ and CD8+ human T cell subsets derived from tumor-infiltrating lymphocytes and peripheral blood mononuclear cells

  1991cited by this paper
• Altering the genome by homologous recombination.

  1989cited by this paper
• Germ-line transmission of a disrupted β2microglobulin gene produced by homologous recombination in embryonic stem cells

  1989cited by this paper
• Gene targeting with retroviral vectors: recombination by gene conversion into regions of nonhomology

  1989cited by this paper
• A recombinant plasmid from which an infectious adeno-associated virus genome can be excised in vitro and its use to study viral replication

  1987cited by this paper
• Site-directed mutagenesis by gene targeting in mouse embryo-derived stem cells.

  1987cited by this paper
• Homologous recombination between coinjected DNA sequences peaks in early to mid-S phase

  1987cited by this paper
• High frequency targeting of genes to specific sites in the mammalian genome.

  1986cited by this paper
• Adeno-associated virus vector for high-frequency integration, expression, and rescue of genes in mammalian cells

  1985cited by this paper
• Insertion of DNA sequences into the human chromosomal β-globin locus by homologous recombination

  1985cited by this paper
• Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells.

  1984cited by this paper
• Formation of germ-line chimaeras from embryo-derived teratocarcinoma cell lines

  1984cited by this paper
• Patterns of integration of DNA microinjected into cultured mammalian cells: evidence for homologous recombination between injected plasmid DNA molecules

  1982cited by this paper
• Establishment in culture of pluripotential cells from mouse embryos

  1981cited by this paper
• Transformation of yeast.

  1978cited by this paper
• Restriction endonucleases.

  1976cited by this paper
• Will society be prepared?

  1967cited by this paper
• Adenovirus-Associated Defective Virus Particles

  1965cited by this paper

• Genome editing in the human liver: Progress and translational considerations.

  2021cites this paper
• Efficient in vivo editing of OTC-deficient patient-derived primary human hepatocytes

  2019cites this paper
• An AAVrh10-CAG-CYP21-HA vector allows persistent correction of 21-hydroxylase deficiency in a Cyp21−/− mouse model

  2017cites this paper
• Engineering cell-based therapies to interface robustly with host physiology.

  2016cites this paper
• Light-Activated Nuclear Translocation of Adeno-Associated Virus Nanoparticles Using Phytochrome B for Enhanced, Tunable, and Spatially Programmable Gene Delivery.

  2016cites this paper
• ZINC SUPPLEMENTATION MAY REDUCE THE RISK OF HEPATOCELLULAR CARCINOMA USING BIG DATA NETWORK ANALYSIS

  2016cites this paper
• Genome Editing: Potential Treatment for Lysosomal Storage Diseases

  2015cites this paper