TY - JOUR
T1 - Design of synthetic materials for intracellular delivery of RNAs
T2 - From siRNA-mediated gene silencing to CRISPR/Cas gene editing
AU - Miller, Jason B.
AU - Siegwart, Daniel J.
N1 - Funding Information:
D. J. S. acknowledges financial support from the Welch Foundation (I-1855), American Cancer Society (RSG-17-012-01), Department of Defense (CA150245P3), and Cancer Prevention and Research Institute of Texas (CPRIT) (R1212).
Publisher Copyright:
© 2018, Tsinghua University Press and Springer-Verlag GmbH Germany, part of Springer Nature.
PY - 2018/10/1
Y1 - 2018/10/1
N2 - Ribonucleic acids (RNAs) possess great therapeutic potential and can be used to treat a variety of diseases. The unique biophysical properties of RNAs, such as high molecular weight, negative charge, hydrophilicity, low stability, and potential immunogenicity, require chemical modification and development of carriers to enable intracellular delivery of RNAs for clinical use. A variety of nanomaterials have been developed for the effective in vivo delivery of short/ small RNAs, messenger RNAs, and RNAs required for gene editing technologies including clustered regularly interspaced palindromic repeat (CRISPR)/Cas. This review outlines the challenges of delivering RNA therapeutics, explores the chemical synthesis of RNA modifications and carriers, and describes the efforts to design nanomaterials that can be used for a variety of clinical indications.
AB - Ribonucleic acids (RNAs) possess great therapeutic potential and can be used to treat a variety of diseases. The unique biophysical properties of RNAs, such as high molecular weight, negative charge, hydrophilicity, low stability, and potential immunogenicity, require chemical modification and development of carriers to enable intracellular delivery of RNAs for clinical use. A variety of nanomaterials have been developed for the effective in vivo delivery of short/ small RNAs, messenger RNAs, and RNAs required for gene editing technologies including clustered regularly interspaced palindromic repeat (CRISPR)/Cas. This review outlines the challenges of delivering RNA therapeutics, explores the chemical synthesis of RNA modifications and carriers, and describes the efforts to design nanomaterials that can be used for a variety of clinical indications.
KW - RNA interference (RNAi)
KW - clustered regularly interspaced palindromic repeat (CRISPR)/Cas
KW - messenger RNA (mRNA)
KW - nanoparticles
KW - nucleic acid therapeutics
KW - synthetic nanomaterials
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U2 - 10.1007/s12274-018-2099-4
DO - 10.1007/s12274-018-2099-4
M3 - Review article
AN - SCOPUS:85051286916
SN - 1998-0124
VL - 11
SP - 5310
EP - 5337
JO - Nano Research
JF - Nano Research
IS - 10
ER -