Open AccessmRNA Treatment Rescues Niemann–Pick Disease Type C1 in Patient Fibroblasts
Author(s) -
Denzil Furtado,
Christina CortezJugo,
Ya Hui Hung,
Ashley I. Bush,
Frank Caruso
Publication year - 2022
Publication title -
molecular pharmaceutics
Language(s) - Uncategorized
Resource type - Journals
SCImago Journal Rank - 1.13
H-Index - 127
eISSN - 1543-8392
pISSN - 1543-8384
DOI - 10.1021/acs.molpharmaceut.2c00463
Subject(s) - niemann–pick disease , niemann–pick disease, type c , disease , lysosomal storage disease , chemistry , fibroblast , medicine , cancer research , microbiology and biotechnology , biology , biochemistry , in vitro
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