Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

Piotr S. Kowalski; Arnab Rudra; Lei Miao; Daniel G. Anderson

doi:10.1016/j.ymthe.2019.02.012

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

Piotr S. Kowalski
, Arnab Rudra
, Lei Miao
, Daniel G. Anderson

Research output: Contribution to journal › Review article › peer-review

Abstract

mRNA has broad potential as a therapeutic. Current clinical efforts are focused on vaccination, protein replacement therapies, and treatment of genetic diseases. The clinical translation of mRNA therapeutics has been made possible through advances in the design of mRNA manufacturing and intracellular delivery methods. However, broad application of mRNA is still limited by the need for improved delivery systems. In this review, we discuss the challenges for clinical translation of mRNA-based therapeutics, with an emphasis on recent advances in biomaterials and delivery strategies, and we present an overview of the applications of mRNA-based delivery for protein therapy, gene editing, and vaccination.

Original language	English
Pages (from-to)	710-728
Number of pages	19
Journal	Molecular Therapy
Volume	27
Issue number	4
DOIs	https://doi.org/10.1016/j.ymthe.2019.02.012
Publication status	Published - 10 Apr 2019
Externally published	Yes

Keywords

biomaterials
clinical trial
CRISPR
gene editing
gene therapy
lipid nanoparticles
mRNA delivery
mRNA nanoparticles
mRNA vaccine
protein replacement

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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10.1016/j.ymthe.2019.02.012

Cite this

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title = "Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery",

abstract = "mRNA has broad potential as a therapeutic. Current clinical efforts are focused on vaccination, protein replacement therapies, and treatment of genetic diseases. The clinical translation of mRNA therapeutics has been made possible through advances in the design of mRNA manufacturing and intracellular delivery methods. However, broad application of mRNA is still limited by the need for improved delivery systems. In this review, we discuss the challenges for clinical translation of mRNA-based therapeutics, with an emphasis on recent advances in biomaterials and delivery strategies, and we present an overview of the applications of mRNA-based delivery for protein therapy, gene editing, and vaccination.",

keywords = "biomaterials, clinical trial, CRISPR, gene editing, gene therapy, lipid nanoparticles, mRNA delivery, mRNA nanoparticles, mRNA vaccine, protein replacement",

author = "Kowalski, \{Piotr S.\} and Arnab Rudra and Lei Miao and Anderson, \{Daniel G.\}",

note = "Publisher Copyright: {\textcopyright} 2019",

year = "2019",

month = apr,

day = "10",

doi = "10.1016/j.ymthe.2019.02.012",

language = "English",

volume = "27",

pages = "710--728",

journal = "Molecular Therapy",

issn = "1525-0016",

publisher = "Cell Press",

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}

TY - JOUR

T1 - Delivering the Messenger

T2 - Advances in Technologies for Therapeutic mRNA Delivery

AU - Kowalski, Piotr S.

AU - Rudra, Arnab

AU - Miao, Lei

AU - Anderson, Daniel G.

PY - 2019/4/10

Y1 - 2019/4/10

N2 - mRNA has broad potential as a therapeutic. Current clinical efforts are focused on vaccination, protein replacement therapies, and treatment of genetic diseases. The clinical translation of mRNA therapeutics has been made possible through advances in the design of mRNA manufacturing and intracellular delivery methods. However, broad application of mRNA is still limited by the need for improved delivery systems. In this review, we discuss the challenges for clinical translation of mRNA-based therapeutics, with an emphasis on recent advances in biomaterials and delivery strategies, and we present an overview of the applications of mRNA-based delivery for protein therapy, gene editing, and vaccination.

AB - mRNA has broad potential as a therapeutic. Current clinical efforts are focused on vaccination, protein replacement therapies, and treatment of genetic diseases. The clinical translation of mRNA therapeutics has been made possible through advances in the design of mRNA manufacturing and intracellular delivery methods. However, broad application of mRNA is still limited by the need for improved delivery systems. In this review, we discuss the challenges for clinical translation of mRNA-based therapeutics, with an emphasis on recent advances in biomaterials and delivery strategies, and we present an overview of the applications of mRNA-based delivery for protein therapy, gene editing, and vaccination.

KW - biomaterials

KW - clinical trial

KW - CRISPR

KW - gene editing

KW - gene therapy

KW - lipid nanoparticles

KW - mRNA delivery

KW - mRNA nanoparticles

KW - mRNA vaccine

KW - protein replacement

UR - https://www.scopus.com/pages/publications/85062273938

U2 - 10.1016/j.ymthe.2019.02.012

DO - 10.1016/j.ymthe.2019.02.012

M3 - Review article

C2 - 30846391

AN - SCOPUS:85062273938

SN - 1525-0016

VL - 27

SP - 710

EP - 728

JO - Molecular Therapy

JF - Molecular Therapy

IS - 4

ER -

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

Abstract

Keywords

UN SDGs

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