Review
Version 1
Preserved in Portico This version is not peer-reviewed
In Vivo Gene Editing: A Healthcare Revolution in the Making
Version 1
: Received: 3 August 2023 / Approved: 4 August 2023 / Online: 4 August 2023 (19:15:10 CEST)
A peer-reviewed article of this Preprint also exists.
Niazi, S.K. The Dawn of In Vivo Gene Editing Era: A Revolution in the Making. Biologics 2023, 3, 253-295. Niazi, S.K. The Dawn of In Vivo Gene Editing Era: A Revolution in the Making. Biologics 2023, 3, 253-295.
Abstract
Gene or genome editing (GE) revises, removes, or replaces a mutated gene at the DNA level; it is a tool. Gene therapy (GT) offsets mutations by introducing a "normal" version of the gene into the body while the diseased gene remains in the genome; it is a medicine. So far, no in vivo GE product has been approved, as opposed to 22 GT products approved by the FDA. However, several are under development, and the FDA anticipates their fast entry, as evidenced by the FDA's actions of issuing GE-specific guidelines. The potential of GE in treating diseases far supersedes any other modality due to the recognized role of genes in most human diseases. While GT products add missing genes, GE tools can overcome many aberrations. However, GE presents many safety challenges, including off-target impact, delivery consistency, and long-term effects of gene-fixing. However, most of these concerns will be resolved once the FDA begins approving them, leading to hundreds of products capable of treating untreatable diseases.
Keywords
gene editing 1; CRISPR 2; gRNA 3; autoimmune disorders 4; nuclease 5; hereditary disorders 6
Subject
Medicine and Pharmacology, Clinical Medicine
Copyright: This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
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