期刊
EXPERT OPINION ON DRUG DELIVERY
卷 15, 期 9, 页码 905-913出版社
TAYLOR & FRANCIS LTD
DOI: 10.1080/17425247.2018.1517746
关键词
CRISPR/Cas9; gene therapy; in vivo genome editing; therapeutic strategies; nonviral delivery; viral delivery; intracellular delivery
资金
- NATIONAL INSTITUTE OF BIOMEDICAL IMAGING AND BIOENGINEERING [R01EB022641] Funding Source: NIH RePORTER
- NATIONAL INSTITUTE OF GENERAL MEDICAL SCIENCES [T32GM008515, T32GM118289, R01GM077173] Funding Source: NIH RePORTER
- NIBIB NIH HHS [R01 EB022641] Funding Source: Medline
- NIGMS NIH HHS [T32 GM008515, R01 GM077173] Funding Source: Medline
Introduction: Therapeutic gene editing is becoming a viable biomedical tool with the emergence of the CRISPR/Cas9 system. CRISPR-based technologies have promise as a therapeutic platform for many human genetic diseases previously considered untreatable, providing a flexible approach to high-fidelity gene editing. For many diseases, such as sickle-cell disease and beta thalassemia, curative therapy may already be on the horizon, with CRISPR-based clinical trials slated for the next few years. Translation of CRISPR-based therapy to in vivo application however, is no small feat, and major hurdles remain for efficacious use of the CRISPR/Cas9 system in clinical contexts. Areas covered: In this topical review, we highlight recent advances to in vivo delivery of the CRISPR/Cas9 system using various packaging formats, including viral, mRNA, plasmid, and protein-based approaches. We also discuss some of the barriers which have yet to be overcome for successful translation of this technology. Expert opinion: This review focuses on the challenges to efficacy for various delivery formats, with specific emphasis on overcoming these challenges through the development of carrier vehicles for transient approaches to CRISPR/Cas9 delivery in vivo.
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