Hassan H. Fakih

One Scaffold, Many Targets: Fully Modified RNA Nanostructures for Multi-Gene Silencing Beyond the Liver

This paper introduces fully modified RNA nanostructures (FMRNs) for multi-gene silencing beyond the liver, showing enhanced pharmacokinetics, extrahepatic delivery, and programmable control over siRNA stoichiometry.

Albumin-binding dendrimer-conjugated siRNA enables safe and effective gene silencing throughout the central nervous system

This study develops D-siRNA, an albumin-binding dendrimer-conjugated siRNA, showing effective and durable gene silencing in the CNS with no detectable toxicity.

Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse model

Therapeutic siRNAs provide safe and durable modulation of MECP2 for the treatment of mild and severe MECP2 Duplication Syndrome.

Mismatch repair dissection by in vivo RNAi reveals dose-dependent modulators of somatic instability and proteome remodeling in Huntingtons disease

The paper uses RNAi to identify dose-dependent MMR gene modulators of somatic instability and proteome changes in HD mouse models.

Therapeutic delivery of albumin-binding siRNA targeting IRS2 to diverse cell types reduces mammary tumor growth

This study delivers IRS2-targeting siRNAs to mammary tumors via albumin-binding dendrimers, reducing tumor growth by inhibiting Irs2 expression and altering tumor microenvironment features.

Potent and durable gene modulation in heart and muscle with chemically defined siRNAs

This study develops chemically defined siRNAs for durable gene modulation in muscle and heart, showing long-term silencing and functional benefits with minimal toxicity.