Ontology highlight
ABSTRACT:
SUBMITTER: Kleinman ME
PROVIDER: S-EPMC3255577 | biostudies-literature | 2012 Jan
REPOSITORIES: biostudies-literature
Kleinman Mark E ME Kaneko Hiroki H Cho Won Gil WG Dridi Sami S Fowler Benjamin J BJ Blandford Alexander D AD Albuquerque Romulo J C RJ Hirano Yoshio Y Terasaki Hiroko H Kondo Mineo M Fujita Takashi T Ambati Balamurali K BK Tarallo Valeria V Gelfand Bradley D BD Bogdanovich Sasha S Baffi Judit Z JZ Ambati Jayakrishna J
Molecular therapy : the journal of the American Society of Gene Therapy 20111011 1
The discovery of sequence-specific gene silencing by endogenous double-stranded RNAs (dsRNA) has propelled synthetic short-interfering RNAs (siRNAs) to the forefront of targeted pharmaceutical engineering. The first clinical trials utilized 21-nucleotide (nt) siRNAs for the treatment of neovascular age-related macular degeneration (AMD). Surprisingly, these compounds were not formulated for cell permeation, which is required for bona fide RNA interference (RNAi). We showed that these "naked" siR ...[more]