Challenges of successful gene therapy for neurologic disorders

Challenges of successful gene therapy for neurologic disorders

  • Loss of function accounts for only a small percentage of genetic disorders.
  • Most vectors for introducing DNA into cells are inefficient.
  • Expression of proteins from viral vectors on cell surfaces may trigger host immune responses.
  • Introduction of the normal protein itself may trigger host immune responses.
  • Expression of introduced DNA may be transient.
  • Vectors may introduce genes into nontarget cells.
  • Integrated DNA sequences may be regulated abnormally.
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