Characterization of the Prion Protein Binding Properties of Antisense Oligonucleotides.

antisense oligonucleotide biomarker cerebrospinal fluid dynamic light scattering isothermal titration calorimetry neurodegeneration nuclear magnetic resonance prion

Journal

Biomolecules
ISSN: 2218-273X
Titre abrégé: Biomolecules
Pays: Switzerland
ID NLM: 101596414

Informations de publication

Date de publication:
18 12 2019
Historique:
received: 18 11 2019
revised: 12 12 2019
accepted: 13 12 2019
entrez: 22 12 2019
pubmed: 22 12 2019
medline: 15 9 2020
Statut: epublish

Résumé

Antisense oligonucleotides (ASOs) designed to lower prion protein (PrP) expression in the brain through RNase H1-mediated degradation of PrP RNA are in development as prion disease therapeutics. ASOs were previously reported to sequence-independently interact with PrP and inhibit prion accumulation in cell culture, yet

Identifiants

pubmed: 31861275
pii: biom10010001
doi: 10.3390/biom10010001
pmc: PMC7022474
pii:
doi:

Substances chimiques

Oligonucleotides, Antisense 0
Prion Proteins 0
Recombinant Proteins 0

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't Research Support, U.S. Gov't, Non-P.H.S.

Langues

eng

Sous-ensembles de citation

IM

Subventions

Organisme : NIH HHS
ID : F31 AI122592
Pays : United States

Déclaration de conflit d'intérêts

HTZ and HBK are employees and shareholders of Ionis Pharmaceuticals. EVM and SV have received research support in the form of charitable contributions from Ionis Pharmaceuticals and Charles River Laboratories. SV has received speaking fees from Illumina. EVM has consulted for Deerfield Management and Guidepoint. SLS serves on the Board of Directors of the Genomics Institute of the Novartis Research Foundation (“GNF”); is a shareholder and serves on the Board of Directors of Jnana Therapeutics; is a shareholder of Forma Therapeutics; is a shareholder and advises Decibel Therapeutics and Eikonizo Therapeutics; serves on the Scientific Advisory Boards of Eisai Co., Ltd., Ono Pharma Foundation, Exo Therapeutics, and F-Prime Capital Partners; and is a Novartis Faculty Scholar.

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Auteurs

Andrew G Reidenbach (AG)

Chemical Biology and Therapeutics Science Program, Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.

Eric Vallabh Minikel (EV)

Chemical Biology and Therapeutics Science Program, Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
Prion Alliance, Cambridge, MA 02139, USA.

Hien T Zhao (HT)

Ionis Pharmaceuticals, Carlsbad, CA 92010, USA.

Stacy G Guzman (SG)

Chemical Biology and Therapeutics Science Program, Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
Exceptional Research Opportunities Program (EXROP), Howard Hughes Medical Institute (HHMI), Chevy Chase, MD 20815, USA.
Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.

Alison J Leed (AJ)

Center for the Development of Therapeutics, Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.

Michael F Mesleh (MF)

Center for the Development of Therapeutics, Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.

Holly B Kordasiewicz (HB)

Ionis Pharmaceuticals, Carlsbad, CA 92010, USA.

Stuart L Schreiber (SL)

Chemical Biology and Therapeutics Science Program, Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
Department of Chemistry & Chemical Biology, Harvard University, Cambridge, MA 02138, USA.

Sonia M Vallabh (SM)

Chemical Biology and Therapeutics Science Program, Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
Stanley Center for Psychiatric Research, Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
Prion Alliance, Cambridge, MA 02139, USA.

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