Hot Topics in Cellular Neuropathology (Volume III): Using CRISPR/Cas9 Technology for Deciphering Central Nervous System Disease Targets

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About this Research Topic

Submission deadlines

  1. Manuscript Submission Deadline 31 May 2025

  2. This Research Topic is still accepting articles.

Background

Methodological advancements have played a pivotal role in driving biomedical progress. A striking illustration of this notion is the groundbreaking discovery of the CRISPR/Cas9 technology by Emmanuelle Charpentier and Jennifer A. Doudna in 2012, which earned them the Nobel Prize in Chemistry in 2020. This innovation, which facilitates precise gene editing, has revolutionized the capability to incorporate gene sequences into genomes with an unprecedented level of accuracy and efficiency.

The genesis of the CRISPR/Cas9 technology emerged unexpectedly from investigations into Streptococcus pyogenes. Within this context, Charpentier uncovered an entirely novel molecule, tracrRNA, which constitutes a crucial component of the bacterium's innate defense mechanism aimed at neutralizing viruses by cleaving their DNA strands. Through strategic manipulation of the bacterium's molecular components and the adaptation of its enzymatic machinery, Charpentier and Doudna ingeniously reprogrammed these molecular “scissors”, endowing them with the ability to cleave DNA molecules at predetermined locations. This remarkable technique, when extended to eukaryotic cells, has unlocked unparalleled avenues in the realm of gene editing, thereby profoundly augmenting our comprehension of cellular physiology and pathology.

Consequently, the CRISPR/Cas9 technology has emerged as a cornerstone of modern biotechnology over the past decade. Its global utilization across bioscience domains underscores its impact and significance, and its applications have extended far into the field of clinical neurosciences. So far, CRISPR/Cas9 has inseminated Cellular Neuropathology in the modeling of neurological disorders, concepts of neurodevelopment, gene therapy, and regenerative medicine. It is important to note that while CRISPR/Cas9 technology holds immense potential, there are also challenges to overcome, including off-target effects, delivery methods, and the need for rigorous safety assessments before any clinical applications can be more widely adopted.

In an effort to identify the most promising concepts in translational neurosciences, the Cellular Neuropathology section of Frontiers in Cellular Neurosciences recently launched a platform, the Hot Topics hub. Within this platform, this journal searches for impactful papers in Cellular Neuropathology, which carry landscape-changing potential, broaden imagination horizons and expand current diagnostic or therapeutic possibilities. Within this platform, two previous Research Topics evaluated the role of subtle neuroinflammation in chronic neurodegeneration and the roles of neuronal plasticity in the injured CNS. Within this new Research Topic, we would like to shift the focus from distinct disease pathomechanisms to a methodological tool, which broadly impacts various disease areas. As in the previous Research Topics, suitable manuscripts should push our understanding of neurological diseases, overcome existing limitations, pave the way for therapeutic progress and deserve attention in future research developments. Papers studying disease targets are welcome in the central and peripheral nervous systems, similar to papers studying CRISPR/Cas9-based gene therapies in vitro and in vivo. With this Research Topic, we would like to expand current knowledge in the field of functional genomics and gain further insight into neurodevelopmental disorders. This Research Topic is open for all disease areas. Papers outlining limitations and challenges of CRISPR/Cas9 technologies are particularly invited. In this search for the best ideas and concepts, Original Research, Reviews, Perspectives, and Opinions are envisaged. Papers will be reviewed based on excellence, originality, and innovation potential. Outstanding papers will be featured in an editorial. We are looking forward to your contributions to this new Research Topic.

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This Research Topic accepts the following article types, unless otherwise specified in the Research Topic description:

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  • Hypothesis and Theory
  • Methods
  • Mini Review
  • Opinion

Articles that are accepted for publication by our external editors following rigorous peer review incur a publishing fee charged to Authors, institutions, or funders.

Keywords: CRISPR/Cas9, neurological disorders, neurodevelopment, gene therapy, gene editing

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