Therapeutic in vivo delivery of gene editing agents

2022/07/01 by Aditya Raguram, Samagya Banskota, David R. Liu · 1 voice · 4 citations
Biochemistry, Genetics and Molecular Biology · #Virus-based gene therapy research #CRISPR and Genetic Engineering #RNA Interference and Gene Delivery

paper · doi:10.1016/j.cell.2022.03.045

openalex publication_date 2022/07/01 · openalex created_date 2025/10/10 · openalex updated_date 2026/07/31

Abstract

In vivo gene editing therapies offer the potential to treat the root causes of many genetic diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to safely and efficiently deliver gene editing agents to relevant organs and tissues in vivo. Here, we review current delivery technologies that have been used to enable therapeutic in vivo gene editing, including viral vectors, lipid nanoparticles, and virus-like particles. Since no single delivery modality is likely to be appropriate for every possible application, we compare the benefits and drawbacks of each method and highlight opportunities for future improvements.

Citations

• A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity
• Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects
• Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing
• Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors
• Engineering precision nanoparticles for drug delivery
• Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine
• Efficacy and Safety of the mRNA-1273 SARS-CoV-2 Vaccine
• mRNA-lipid nanoparticle COVID-19 vaccines: Structure and stability
• CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
• Engineered pegRNAs improve prime editing efficiency
• Drug delivery systems for RNA therapeutics

Cited by

• The mRNA-Based Innovative Strategy: Progress and Challenges
• <i>In Vivo</i> Prime Editing by Lipid Nanoparticle Co-Delivery of Chemically Modified pegRNA and Prime Editor mRNA
• Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines
• Template-independent genome editing and restoration for correcting frameshift disorders

Discussions

• doi.org/10.1016/j.ce... [bsky, 1 points, 1 comments]

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