Site-specific gene correction of a point mutation in human iPS cells derived from an adult patient with sickle cell disease

Medicine & Life Sciences

• Induced Pluripotent Stem Cells 100%
• Sickle Cell Anemia 96%
• Point Mutation 86%
• Genes 35%
• Homologous Recombination 28%
• Mutation 28%
• Zinc Finger Nucleases 19%
• Alleles 18%
• Globins 15%
• Preclinical Drug Evaluations 15%
• Gene Targeting 14%
• Human Genome 13%
• Introns 12%
• Cell- and Tissue-Based Therapy 11%
• Genetic Therapy 11%
• Plasmids 10%
• Nucleotides 10%
• Clone Cells 10%
• Erythrocytes 9%
• Phenotype 7%
• Pharmaceutical Preparations 5%

Chemical Compounds

• Mutation 74%
• Nucleotide 18%
• Substitution Reaction 13%
• Drug 11%