Molecular genetics and potential gene therapy
SH Orkin - Clinical immunology and immunopathology, 1986 - Elsevier
Contemporary molecular techniques including gene cloning, DNA sequencing, and gene
transfer permit precise and comprehensive analysis of genetic disorders. For example, the
molecular basis of hemoglobin synthesis disorders (the thalassemias) can now be ascribed
to more than 30 different specific mutations. These affect virtually all aspects of gene
expression. The more recent capacity to reintroduce cloned genes into mammalian cells in a
functional form has raised the prospect of gene therapy, that is, the replacement of an …
transfer permit precise and comprehensive analysis of genetic disorders. For example, the
molecular basis of hemoglobin synthesis disorders (the thalassemias) can now be ascribed
to more than 30 different specific mutations. These affect virtually all aspects of gene
expression. The more recent capacity to reintroduce cloned genes into mammalian cells in a
functional form has raised the prospect of gene therapy, that is, the replacement of an …