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SELECTBIO Conferences Exosomes and Microvesicles:  Research, Biomarker Cargo and Therapeutic Potential

Anastasia Khvorova's Biography

Anastasia Khvorova, Professor, RNA Therapeutics Institute, University of Massachusetts Medical School

Anastasia Khvorova, PhD, is Professor in the RNA Therapeutics Institute and the Program in Molecular Medicine at the University of Massachusetts Medical School (UMMS) in Worcester, Massachusetts. Before joining the UMMS faculty, she held leadership positions in industry, including Vice President of Research & Development and Chief Science Officer of Dharmacon, Founder and Scientific Advisor of Advirna, and Chief Science Officer and Senior Vice President of RXi Pharmaceuticals. Dr Khvorova’s industry experience in drug discovery and development collaborations with pharmaceutical companies along with her expertise in chemistry and cell biology allowed her to establish a lab at UMMS that brings together organic chemists and RNA biologists to develop novel approaches and solutions to understanding natural and therapeutic RNA trafficking and delivery. Dr Khvorova is a member of the Board of Directors of the Oligonucleotide Therapeutics Society and sits on the editorial board of Nucleic Acid Research. She is the author of more than 150 abstracts, more than 50 peer-reviewed articles, several book chapters, and more than 250 patents and patent applications; her publications are widely cited.

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Exosomes as Therapeutic RNAs Delivery Vesicles

Tuesday, 22 March 2016 at 09:00

Add to Calendar ▼2016-03-22 09:00:002016-03-22 10:00:00Europe/LondonExosomes as Therapeutic RNAs Delivery

Applications of RNA interference for neuroscience research have been limited by a lack of simple and efficient methods to deliver oligonucleotides to primary neurons in culture and to the brain. Here, we show that primary neurons rapidly internalize hydrophobic siRNAs (hsiRNAs) added directly to the culture medium without lipid formulation. We identify functional hsiRNAs targeting the mRNA of huntingtin, the mutation of which is responsible for Huntington’s disease, and show that direct uptake in neurons induces potent and specific silencing in vitro. Moreover, a single injection of unformulated hsiRNA into mouse brain silences Htt mRNA with minimal neuronal toxicity. Thus hsiRNAs embody a class of therapeutic oligonucleotides that enable simple and straightforward functional studies of genes involved in neuronal biology and neurodegenerative disorders in a native biological context.

Add to Calendar ▼2016-03-21 00:00:002016-03-22 00:00:00Europe/LondonExosomes and Microvesicles: Research, Biomarker Cargo and Therapeutic