Advances in CRISPR/Cas Systems for Genome Editing Applications in Biomedical Research
A special issue of Biomolecules (ISSN 2218-273X). This special issue belongs to the section "Molecular Biology".
Deadline for manuscript submissions: 30 June 2024 | Viewed by 222
Special Issue Editor
Special Issue Information
Dear Colleagues,
CRISPR/Cas9 gene editing tools play a pivotal role in the precise gene modification in all model organisms. The fast-evolving CRISPR systems, currently under intensive development, have relevant applications in the biomedical research at the cellular and organism levels. CRISPR applications have been reported for genetic and nongenetic diseases, for the diagnosis of infectious diseases, for the detection of compounds such as antibiotics, nutrients or toxins, and for large-scale genetic screens. This Special Issue aims to cover all biomedical-oriented research that utilizes the regular CRISPR/Cas and the CRISPR system 2.0 for genome editing in cellular and animal models. Original articles and reviews focused on CRISPR technology applied to biomedical research, human gene therapy, cancer immunotherapy, diagnosis and the development of biological sensors are welcome.
Dr. Alessandra Recchia
Guest Editor
Manuscript Submission Information
Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.
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Keywords
- genome editing
- CRISPR/Cas
- gene therapy
- cancer immunotherapy
- diagnosis
Planned Papers
The below list represents only planned manuscripts. Some of these manuscripts have not been received by the Editorial Office yet. Papers submitted to MDPI journals are subject to peer-review.
Title: Fiddling with the Nucleus - Efficacy and Safety of (Epi)genome Editing
Authors: Vedrana Vičić Bočkor; Luka Bočkor
Affiliation: 1Epigenetics of Protein Glycosylation, Molecular Biology Department, Division of Biology, Faculty of Science, University of Zagreb, Zagreb, Croatia
2Centre for Applied Bioanthropology, Institute for Anthropological Research, Zagreb, Croatia
Abstract: The advancement of genome editing technologies has been exceptional within the last decade, mostly due to discovery and adaptations of TALE and Cas9 nucleases. Genome editing is a promising treatment for monogenetic disorders, such as Crigler-Najjar Syndrome Type I or hemophilia B, with nowadays some of the treatments for specific genetic diseases remarkably close to the clinic. Recently, another promising strategy arose from development of catalytically inactive Cas9 nucleases that allows for chromatin remodeling without changes in underlying genetic sequence, the epigenome editing. It is a promising strategy in polygenetic diseases, such as diabetes, chronic pain, cancer and psychiatric and neurological disorders, where fine-tuning of gene expression can affect the disease development and progression. Moreover, epigenetic editing allows treatment in a specific manner with low toxicity, contrary to existing conventional epigenetic drugs (especially in cancer therapy) that have a genome-wide effect. However, in order for a treatment to reach the clinic, it has to be effective and safe for patients. Efficacy for both strategies is dependent on delivery strategies, target tissue, type of the final product of the delivered vector and the host immune response. Ideally, both vectors and final products should evade the host immune response, be delivered to targeted organs/tissues/cell types and should perform equally well in males and females. Here we review the current research on genome and epigenome strategies and discuss questions that may contribute to setting protocols for/advancement of efficacy and safety of editing therapies.
