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Gene Therapy (2) (Challenges (Viral Vectors (Toxic, immune, inflammatory…
Gene Therapy (2)
Challenges
Short Lived
Immune Response
Increases with repeat molecule
Viral Vectors
Toxic, immune, inflammatory response
Can cause disease
Multigene Disorders
Need more than 1 gene to treat
HBP
May induce tumor in tumor suppressor gene
Success
Safety!
Gene Expression Regulation
Gene Expression Persistence
Efficient Gene Transfer into target cells
Non-Viral Gene Delivery
Lipid-DNA Complex
+ve Lipids + -ve DNA
Pros
Stable Complex
Carry large DNA
Target specific cells
No induction of immunological reactions
Cons
Low Transfection Efficiency
Transcient Expression
Inhibited by Serum
Some Cell Toxicity
Viral Gene Therapy Concern
Safety!
NP-Based Gene Therapy
Lipofection
Liposome Transfection (Fig 54)
Intratumoral Challenges (Fig 53)