Dendrimer conjugates for light-activated delivery of antisense oligonucleotides

Exon skipping restores dystrophin expression, but fails to prevent disease progression in later stage dystrophic dko mice

(2014) B Wu et al.   GENE THERAPY

Development of Therapeutic Splice-Switching Oligonucleotides

(2014) Petra Disterer et al.   HUMAN GENE THERAPY

In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight

(2014) James E. Dahlman et al.   Nature Nanotechnology

A nanoparticle formula for delivering siRNA or miRNAs to tumor cells in cell culture and in vivo

(2014) Ki Young Choi et al.   Nature Protocols

Degradable lipid nanoparticles with predictable in vivo siRNA delivery activity

(2014) Kathryn A. Whitehead et al.   Nature Communications

Three-layered polyplex micelle as a multifunctional nanocarrier platform for light-induced systemic gene transfer

(2014) Takahiro Nomoto et al.   Nature Communications

Harnessing photochemical internalization with dual degradable nanoparticles for combinatorial photo–chemotherapy

(2014) George Pasparakis et al.   Nature Communications

Ex Vivo Programming of Dendritic Cells by Mitochondria-Targeted Nanoparticles to Produce Interferon-Gamma for Cancer Immunotherapy

(2013) Sean Marrache et al.   ACS Nano

Albumin-based nanoconjugates for targeted delivery of therapeutic oligonucleotides

(2013) Xin Ming et al.   BIOMATERIALS

Transformation-Associated Changes in Sphingolipid Metabolism Sensitize Cells to Lysosomal Cell Death Induced by Inhibitors of Acid Sphingomyelinase

(2013) Nikolaj H.T. Petersen et al.   CANCER CELL

The small molecule Retro-1 enhances the pharmacological actions of antisense and splice switching oligonucleotides

(2013) Xin Ming et al.   NUCLEIC ACIDS RESEARCH

PAMAM-functionalized water soluble quantum dots for cancer cell targeting

(2012) Mehriban Akin et al.   JOURNAL OF MATERIALS CHEMISTRY

Cancer cell–selective in vivo near infrared photoimmunotherapy targeting specific membrane molecules

(2011) Makoto Mitsunaga et al.   NATURE MEDICINE

Targeting RNA to treat neuromuscular disease

(2011) Francesco Muntoni et al.   NATURE REVIEWS DRUG DISCOVERY

Anti-tumor activity of splice-switching oligonucleotides

(2010) John A. Bauman et al.   NUCLEIC ACIDS RESEARCH

Mechanistic Insight into Cell Growth, Internalization, and Cytotoxicity of PAMAM Dendrimers

(2009) Srinivas Parimi et al.   BIOMACROMOLECULES

Therapeutic Potential of Splice-Switching Oligonucleotides

(2009) John Bauman et al.   OLIGONUCLEOTIDES

The Biological Effect of an Antisense Oligonucleotide Depends on Its Route of Endocytosis and Trafficking

(2009) Md. Rowshon Alam et al.   OLIGONUCLEOTIDES

Intracellular delivery of an anionic antisense oligonucleotide via receptor-mediated endocytosis

(2008) Md Rowshon Alam et al.   NUCLEIC ACIDS RESEARCH