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. 2021 Feb 16;296:100416. doi: 10.1016/j.jbc.2021.100416

Table 7.

Routes of administration validated for PS ASOs

A. Local administration
Route Example Indication
Intradermal Ionis CTGF ASO Scarring
Intravitreal Vitravene CMV retinitis
Pulmonary ENAC-2.5 Cystic fibrosis (CF)
Intrathecal Nusinersen Spinal Muscular Atrophy
Rectal Alicaforsen Pouchitis

B. Systemic
Route Example Indication
Subcutaneous Inotersen TTR Amyloidosis
Subcutaneous, intramuscular IONIS HCV ASO Hepatitis C
Subcutaneous, oral Mipomersen Homozygous Familial Hypercholesterolemia