Table 1.

Tools for therapeutic manipulation of protein levels in tissues.

	AAV	Modified mRNA	miRNA/siRNA	Small Molecules	Protein
Route of Administration	Local or I.V	Local or I.V	Local or I.V	I.V or oral	Local
Limitation of Gene Size	Yes (4.5 Kb)	No	N/A	N/A	No
Pharmacokinetics	Long term	Short term	Short term	Long or short term	Short term
Multiple Administration	No	Yes	Yes	Yes	Yes
Compromised DNA Integrity	Yes	No	No	No	No
Controlled Expression	No	Yes	Yes	Yes	Yes
Gene Expression Regulation	Up or down	Up or down	Mostly down	Mostly down	Mostly up
Organ Specificity	Yes	No/possibly	No	No	No
Cell Specificity	Yes	No/possibly	No	No	No