NIH VideoCast - NIH COVID-19 Lecture: Nucleic Acid Delivery Systems for RNA Therapy and Gene Editing

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NIH COVID-19 Lecture: Nucleic Acid Delivery Systems for RNA Therapy and Gene Editing

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Air date:	Wednesday, July 15, 2020, 3:00:00 PM Time displayed is Eastern Time, Washington DC Local
Views:	Total views: 1,703 (490 Live, 1,213 On-demand)
Category:	COVID-19
Runtime:	00:59:45
Closed captioning:	Check box to display CC outside of the video Note: You can drag the captioning window around and resize it
Description:	NIH COVID-19 SIG Lecture Series High throughput, combinatorial approaches have revolutionized small-molecule drug discovery. Dan Anderson will describe his work on the combinatorial development of nanoparticulate, intracellular delivery systems for RNA therapy and gene editing. Libraries of degradable polymers and lipid-like materials have been synthesized, formulated, and screened for their ability to deliver macromolecular payloads inside of cells. These nanoformulations facilitate in vivo delivery, enabling gene suppression with small-interfering RNA, gene expression with messenger RNA, or permanent genetic editing using the CRISPR/Cas9 system. Formulations have been developed with in vivo efficacy, and show potential therapeutic applications for a range of different diseases. This lecture will focus on the application of these formulations toward controlling the immune system and in particular as vaccines for infectious disease.

NLM Title:	Nucleic acid delivery systems for RNA therapy and gene editing / Dan Anderson.
Author:	Anderson, Dan. National Institutes of Health (U.S.). COVID-19 Scientific Interest Group,
Publisher:
Abstract:	(CIT): NIH COVID-19 SIG Lecture Series High throughput, combinatorial approaches have revolutionized small-molecule drug discovery. Dan Anderson will describe his work on the combinatorial development of nanoparticulate, intracellular delivery systems for RNA therapy and gene editing. Libraries of degradable polymers and lipid-like materials have been synthesized, formulated, and screened for their ability to deliver macromolecular payloads inside of cells. These nanoformulations facilitate in vivo delivery, enabling gene suppression with small-interfering RNA, gene expression with messenger RNA, or permanent genetic editing using the CRISPR/Cas9 system. Formulations have been developed with in vivo efficacy, and show potential therapeutic applications for a range of different diseases. This lecture will focus on the application of these formulations toward controlling the immune system and in particular as vaccines for infectious disease.
Subjects:	Drug Delivery Systems Gene Editing Nanoparticles RNA, Messenger--immunology Vaccines--immunology
Publication Types:	Lecture Webcast

NLM Classification:	QW 805
NLM ID:	101770493
CIT Live ID:	37814
Permanent link:	https://videocast.nih.gov/watch=37814

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