Article
Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno-associated virus vector.
The Journal of clinical investigation - 1 Oct 1994
Walsh C E, Nienhuis A W, Samulski R J, Brown M G, Miller J L, Young N S, Liu J M
Abstract excerpt
Fanconi anemia (FA) is a recessive inherited disease characterized by defective DNA repair. FA cells are hypersensitive to DNA cross-linking agents that cause chromosomal instability and cell death. FA is manifested clinically by progressive pancytopenia, variable physical anomalies, and predispo...
Topics
- Base Sequence
- Cell Cycle
- Cell Cycle Proteins
- Cell Division
- Cell Line, Transformed
- Chromosome Aberrations
- DNA
- DNA-Binding Proteins
- Dependovirus
- Fanconi Anemia
- Fanconi Anemia Complementation Group C Protein
- Fanconi Anemia Complementation Group Proteins
- Gene Expression
- Gene Transfer Techniques