International journal of biological sciencesJul 29, 2026

Methods for delivering CRISPR gene editing treatments inside the body

CRISPR Gene Editing Weekly Brief ↗PubMed ↗DOI ↗

Updated Aug 4, 2026

Abstract

CRISPR-based genome and epigenome editing technologies have evolved into a diverse therapeutic toolbox.

The review focuses on delivery methods for CRISPR systems used directly in living organisms.
Anatomical, cellular, and immune factors influence how effectively these editing strategies can target specific organs.
Key milestones in preclinical and clinical progress are highlighted to illustrate the current state of CRISPR therapy.
Delivery methods significantly affect the effectiveness, safety, and scalability of CRISPR applications.
Concerns about unintended changes to the genome or epigenome are raised as critical issues in therapeutic development.

Simplified

CRISPR-based genome and epigenome editing technologies have rapidly evolved from programmable nucleases into a diverse therapeutic toolbox encompassing conventional CRISPR systems, base editing, prime editing, RNA targeting, and epigenetic modulation. While early clinical successes relied on ex vivo manipulation of patient-derived cells, recent advances in delivery chemistry and vector engineering are enabling direct in vivo editing across multiple organs. Here, we provide a comprehensive review of delivery modalities of CRISPR systems solely in vivo that underpin their therapeutic translation. We examine how anatomical, cellular, and immunological constraints shape organ-specific editing strategies in different organ systems and we highlight key preclinical and clinical milestones that define the current translational landscape. Across indications, delivery remains a critical determinant of efficacy, safety, and scalability, governing editor exposure, tissue selectivity, and risk of unintended genomic or epigenomic perturbation. This review, authored by members of the COST Action Genome Editing to treat Human Diseases (GenE-HumDi) Network, delineates the principles guiding in vivo genome and epigenome editing and outlines the remaining barriers to durable, tissue-selective, and broadly deployable CRISPR therapeutics.

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Methods for delivering CRISPR gene editing treatments inside the body

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You found one interesting study. We’ll send the next 7.

What Lands in Your Inbox Each Week:

Recent issues from the crispr gene editing brief