Exosome-mediated delivery of hydrophobically modified siRNA for huntingtin mRNA silencing

Molecular Therapy

Volumn 24, Issue 10, 2016, Pages 1836-1847

  Didiot, Marie Cécile ^a   Hall, Lauren M ^a   Coles, Andrew H ^a   Haraszti, Reka A ^a   Godinho, Bruno M D C ^a   Chase, Kathryn ^a   Sapp, Ellen ^b   Ly, Socheata ^a   Alterman, Julia F ^a   Hassler, Matthew R ^a   Echeverria, Dimas ^a   Raj, Lakshmi ^c   Morrissey, David V ^c   Di Figlia, Marian ^b   Aronin, Neil ^a   Khvorova, Anastasia ^a  

^a UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^b MASSACHUSETTS GENERAL HOSPITAL   (United States)

^c NOVARTIS INSTITUTES FOR BIOMEDICAL RESEARCH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

HUNTINGTIN; MESSENGER RNA; OLIGONUCLEOTIDE; SMALL INTERFERING RNA; HTT PROTEIN, HUMAN;

ANIMAL CELL; ARTICLE; BRAIN CELL; CONTROLLED STUDY; CORPUS STRIATUM; EMBRYO; EXOSOME; GENE DELIVERY SYSTEM; GENE SILENCING; GENE THERAPY; HUNTINGTIN GENE; HYDROPHOBICITY; IMMUNOSTIMULATION; IN VIVO STUDY; INNATE IMMUNITY; MOUSE; NONHUMAN; RNA TRANSPORT; ANIMAL; CELL CULTURE; CHEMICAL PHENOMENA; CHEMISTRY; GENE EXPRESSION REGULATION; GENETICS; HUMAN; METABOLISM; NERVE CELL;

ANIMALS; CELLS, CULTURED; EXOSOMES; GENE EXPRESSION REGULATION; GENE SILENCING; GENETIC THERAPY; HUMANS; HUNTINGTIN PROTEIN; HYDROPHOBIC AND HYDROPHILIC INTERACTIONS; MICE; NEURONS; RNA, SMALL INTERFERING;

EID: 84981275108     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2016.126     Document Type: Article

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