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1 Capecchi, Mario R.Developmental defects of the ear, cranial nerves and hindbrain resulting from targeted disruption of the mouse homeobox gene Hox-1.6.Gene targeting in mouse embryo-derived stem cells has been used to generate mice with a disruption in the homeobox gene Hox-1.6. Mice heterozygous at the Hox-1.6 locus appear normal, whereas Hox-1.6-/Hox-1.6- mice die at or shortly after birth. These homozygotes exhibit profound defects in the forma...Chromosome Mapping; Genetic Vectors; Mice, Inbred C57BL2002-06-27
2 Thomas, Kirk R.; Capecchi, Mario R.Location of crossovers during gene targeting with insertion and replacement vectors.Gene targeting was used to introduce nonselectable genetic changes into chromosomal loci in mouse embryo-derived stem cells. The nonselectable markers were linked to a selectable marker in both insertion- and replacement-type vectors, and the transfer of the two elements to the Hprt locus was assaye...Genetic Vectors; Molecular Sequence Data; Restriction Mapping1993-04
3 Capecchi, Mario R.Patterns of integration of DNA microinjected into cultured mammalian cells: evidence for homologous recombination between injected plasmid DNA molecules.We examined the fate of DNA microinjected into nuclei of cultured mammalian cells. The sequence composition and the physical form of the vector carrying the selectable gene affected the efficiency of DNA-mediated transformation. Introduction of sequences near the simian virus 40 origin of DNA replic...Base Sequence; Cell Line; Genes, Viral; Genetic Vectors; Mice; Microinjections1982-11
4 Baehr, WolfgangPharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosisBACKGROUND: Leber congenital amaurosis (LCA), a heterogeneous early-onset retinal dystrophy, accounts for approximately 15% of inherited congenital blindness. One cause of LCA is loss of the enzyme lecithin:retinol acyl transferase (LRAT), which is required for regeneration of the visual photopigmen...Disease Models, Animal; Genetic Vectors; Molecular Sequence Data2005
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