Skip to main content

6 Result(s)

within Matthew R Hassler Remove this filter English Remove this filter

and

  1. Article

    Open Access

    Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington’s disease models

    Small interfering RNAs are a new class of drugs, exhibiting sequence-driven, potent, and sustained silencing of gene expression in vivo. We recently demonstrated that siRNA chemical architectures can be optimi...

    Faith Conroy, Rachael Miller, Julia F. Alterman… in Nature Communications (2022)

    Download PDF (1099 KB) View Article

  2. No Access

    Article

    A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

    Sustained silencing of gene expression throughout the brain using small interfering RNAs (siRNAs) has not been achieved. Here we describe an siRNA architecture, divalent siRNA (di-siRNA), that supports potent,...

    Julia F. Alterman, Bruno M. D. C. Godinho, Matthew R. Hassler… in Nature Biotechnology (2019)

  3. No Access

    Article

    RNAi modulation of placental sFLT1 for the treatment of preeclampsia

    Preeclampsia, a hypertension condition with no approved therapies, is treated in monkeys by gene silencing in the placenta.

    Anton A Turanov, Agnes Lo, Matthew R Hassler, Angela Makris… in Nature Biotechnology (2018)

  4. Article

    Open Access

    Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing

    RNA-based drugs depend on chemical modifications to increase potency and to decrease immunogenicity in vivo. Chemical modification will likely improve the guide RNAs involved in CRISPR-Cas9-based therapeutics ...

    Aamir Mir, Julia F. Alterman, Matthew R. Hassler… in Nature Communications (2018)

    Download PDF (1091 KB) View Article

  5. Article

    Correction: Corrigendum: Taking charge of siRNA delivery

    Nat. Biotechnol. 32, 1197–1198 (2014); published online 9 December 2014; corrected after print 29 April 2015 In the version of this article initially published, in two instances molecules were misidentified in...

    Anastasia Khvorova, Maire F Osborn, Matthew R Hassler in Nature Biotechnology (2015)

    Download PDF (125 KB) View Article

  6. No Access

    Article

    Taking charge of siRNA delivery

    Delivery of siRNA into cells is achieved by neutralizing the negative charge of the phosphate backbone in a reversible manner.

    Anastasia Khvorova, Maire F Osborn, Matthew R Hassler in Nature Biotechnology (2014)