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.