Gene Editing Therapies for Hereditary Diseases
A special issue of Cells (ISSN 2073-4409).
Deadline for manuscript submissions: 25 June 2025 | Viewed by 148
Special Issue Editor
Interests: Duchenne muscular dystrophy; Friedreich ataxia; Alzheimer’s disease; CRISPR; base editing; myoblast transplantation
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Special Issue Information
Dear Colleagues,
The development of genome editing technologies has improved the prospect of treatments for several hereditary diseases. For most of them, high-precision DNA correction will be feasible. Indeed, techniques such as base editing permit us to correct the four most common single-base substitutions, while prime editing can install any substitutions, insertions, and/or deletions of dozens of base pairs. Nuclease-dependent editing approaches involving double-strand DNA breaks (DSBs) often result in a high percentage of uncontrolled editing outcomes, frequently a mixture of insertions and/or deletions (indels), large deletions, and sometimes chromosomal rearrangements. Base editing and prime editing techniques have higher rates of efficiency with fewer byproducts, even in slowly dividing or non-dividing cells, which are most of the cells in adult animals. Viral and non-viral in vivo delivery methods have now been used to deliver the components of base editors and prime editors in numerous animal models. Thus, these techniques are effective agents for in vivo therapeutic genome editing, not only in animal models but also in humans. I am thus proposing the publication of a Special Issue of Cells to present the fantastic progress in these technologies and their rapid use for the development of genetically improved plants and real personalized medical treatments.
Yours faithfully,
Prof. Dr. Jacques P. Tremblay
Guest Editor
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Keywords
- gene therapy
- base editing
- prime editing
- genome editing
- hereditary diseases
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