Splice-Switching Antisense Oligonucleotides Reduce LRRK2 Kinase Activity in Human LRRK2 Transgenic Mice

Korecka, JA; Thomas, R; Hinrich, AJ; Moskites, AM; Macbain, ZK; Hallett, PJ; Isacson, O; Hastings, ML

Korecka, JA (corresponding author), Harvard Med Sch, Neuroregenerat Res Inst, McLean Hosp, Belmont, MA 02478 USA.; Hastings, ML (corresponding author), Rosalind Franklin Univ Med & Sci, Chicago Med Sch, Ctr Genet Dis, N Chicago, IL 60064 USA.; Korecka, JA (corresponding author), Harvard Med Sch, Brigham & Womens Hosp, Boston, MA 02115 USA.

MOLECULAR THERAPY-NUCLEIC ACIDS, 2020; 21 (): 623

Abstract

Parkinson's disease (PD) is a progressive neurological disorder estimated to affect 7-10 million people worldwide. There is no treatment available tha......

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