A. Jyotsana
A. Scharma
A. Chaturvedi
M. Scherr
F. Kuchenbauer
C. L. Sajti
A. Barchanski
R. Lindner
F. Noyan
K.-W. Sühs
D. Grote-Koska
K. Brand
H.-P. Vornlocher
M. Stanulla
B. Bornhauser
J.-P. Bourquin
M. Eder
F. Thol
A. Ganser
R. K. Humphries
E. Ramsay
P. Cullis
M. Heuser

RNA interference efficiently targets human leukemia driven by a fusion oncogene in vivo

Leukemia
2017
Type: Zeitschriftenaufsatz (reviewed)
Abstract
Despite the wide therapeutic potential of RNA interference (RNAi), clinical progress has been slow with only a few examples of successful translation. Efficient knockdown of hepatic transthyretin (87\%) in patients with transthyretin amyloidosis lasted for several weeks after a single dose. Furthermore, in a phase I clinical trial, a single dose of inclisiran (small interfering RNA (siRNA) against the PCSK9 mRNA) efficiently suppressed serum cholesterol for 6 months. However, these studies suggested that siRNA delivery beyond the liver is not yet feasible in the clinic and thus limits the potential benefit of RNAi.