CRISPR: The Game Changer in Gene and Cell Therapy

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Background

Gene and cell therapy involves transferring genetic material into cells in vivo or ex vivo to treat or prevent disease by correcting the underlying genetic disorders or placing the engineered functional cells into the body. After the first successful gene therapy on a patient suffering from adenosine deaminase deficiency in 1990, gene and cell therapy showed its potential and has been explored from many different directions and levels. Notably, the advance of genome engineering technology substantially accelerates the development of this field. CRISPR, as a simple, precise, efficient, and cost-effective gene-editing tool kit, revolutionizes genome engineering and rewrites the future of gene and cell therapy in medicine. The CRISPR toolkit provides multiple customizable approaches applied to gene knockout, gene activation/ inhibition, base editing, precise insertion or deletion, RNA editing et al. Powerful examples of CRISPR-based gene and cell therapy include ex vivo CRISPR-based chimeric antigen receptor therapy for cancer; hematopoietic stem and progenitor cell ex vivo editing for sickle cell disease; and in vivo CRISPR gene therapy for congenital disease and infectious disease such as HIV. Chimeric antigen receptor therapies include those based on T cells (CAR T cells), natural killer cells (CAR NK cells), and tumor-infiltrating lymphocytes (TILs). Gene and cell therapy have been around for decades, however, the invention of CRISPR booms in this field and brings in the hope of permanent cures for genetic disease as well as cancer.

Besides the promising opportunities of CRISPR in gene and cell therapy, the challenges such as safety, efficacy, manufacturing, ethics, regulatory guidelines, and management of CRISPR technology for clinical use are still the current issues that need to be addressed. Over the past decades, extensive efforts have been made to alleviate the issues including limiting off-target through evolving CRISPR enzyme as well as the guide RNA scaffold; designing CRISPR enzymes that do not introduce double strand breaks for precise DNA fragment insertion or deletion; developing a non-viral or viral delivery system to precisely transport genome editing tools into cell nucleus ex vivo or specific tissue in vivo, et al. Meanwhile, through CRISPR-based genetic screening, more and more gene targets were unveiled to modulate cell function, therefore improving treatment efficacy such as CAR-T therapy for cancer. Other screenings include CRISPR mediated CAR/TCR-library integration screen, which compared various CARs/TCRs-T cell functions for cancer immunotherapy and provided guidance for CAR/TCR design in certain circumstances.

The Research Topic aims to inspire novel designs of CRISPR-based gene and cell therapies to improve treatment efficacy and safety for patients suffering from genetic diseases, infectious diseases, and cancer. We encourage Original Articles or Protocols making efforts to develop new CRISPR tools, enhance genome editing precision including knocking out, knocking in, and especially precise point mutation through HDR repairing, increasing the screening efficiency based on techniques or tools used, reduce safety concerns, expand targeted delivery systems based on research with different animal models like zebrafish, drosophila, mouse et al, optimize manufacturing procedure for clinical use, et al. Meanwhile, Reviews, Perspectives, Case Reports, or Opinions that summarize or comment on the cutting-edge progress of CRISPR in gene and cell therapy are welcomed. Policy and Practice Reviews discussing ethics or regulatory guidelines and management regarding CRISPR in clinical applications are equally encouraged.

The subtopics of interest may include, but are not restricted to, the following:

1. Evolvement of novel CRISPR enzymes or guide RNA scaffold with promising characteristics.
2. CRISPR-based genetic screening to identify novel targets for gene and cell therapy improvement.
3. Design of novel CRISPR-based CAR/TCR integrated T/NK/TIL cells for cancer immunotherapy.
4. Development of novel non-viral or viral CRISPR delivery system to achieve safe and precise transport in vivo.
5. Original Research from the clinical trial of CRISPR-based gene and cell therapy.
6. Optimization of the manufacturing process for CRISPR-based gene and cell therapy in clinical applications.
7. Summarize and characterize the current progress of CRISPR-based gene and cell therapy. Thoughts and perspectives.
8. Connecting circular RNA and CRISPR for gene or cell therapy.
9. Apply CRISPR/Cas9 to understand the role of specific genes or diseases.

Keywords: CRISPR, Gene therapy, Cell therapy, genome engineering, genetic disease, immunotherapy

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