Anastasia Khvorova

Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model

T. Taghian, H. Gray-Edwards, Rachael Miller, 2023, Molecular therapy. Methods & clinical development.

Lactate transporter MCT1 in hepatic stellate cells promotes fibrotic collagen expression in nonalcoholic steatohepatitis

Lawrence M. Lifshitz, Dimas Echeverria, Mark Kelly, 2023, bioRxiv.

Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in rodent and swine photoreceptors

M. Hassler, Dimas Echeverria, Annabelle Biscans, 2023, bioRxiv.

Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy

Annemieke Aartsma-Rus, M. Damha, M. van Putten, 2023, Nucleic acid therapeutics.

Hydrophobically Modified let-7b miRNA Enhances Biodistribution to NSCLC and Downregulates HMGA2 In Vivo

E. Anastasiadou, Annabelle Biscans, Maud-Emmanuelle Gilles, 2022 .

RNAi-based drug design: considerations and future directions.

Anastasia Khvorova, Qi Tang, 2024, Nature reviews. Drug discovery.

A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system

E. Sapp, Dimas Echeverria, Ashley Summers, 2024, Nucleic acids research.

Advancements in APOE and dementia research: Highlights from the 2023 AAIC Advancements: APOE conference.

C. Iadecola, O. Chiba-Falek, J. Ulrich, 2024, Alzheimer's & dementia : the journal of the Alzheimer's Association.

Silencing Apoe with divalent‐siRNAs improves amyloid burden and activates immune response pathways in Alzheimer's disease

J. Buchwald, M. Hassler, Dimas Echeverria, 2024, Alzheimer's & dementia : the journal of the Alzheimer's Association.

mRNA-specific readthrough of nonsense codons by antisense oligonucleotides (R-ASOs)

A. Korostelev, Dimas Echeverria, Anastasia Khvorova, 2024, Nucleic acids research.