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Current Molecular Progress on Cell and Gene Therapies

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pathology, Diagnostics, and Therapeutics".

Deadline for manuscript submissions: 20 June 2024 | Viewed by 1501

Special Issue Editor


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Guest Editor
Laboratory of Experimental Virology, Department of Medical Microbiology, Amsterdam UMC, Academic Medical Center, University of Amsterdam, 1105 AZ Amsterdam, The Netherlands
Interests: CRISPR-Cas methods; HIV; DNA; gene therapy; antiviral mechanism; cell therapy

Special Issue Information

Dear Colleagues,

Gene and cell therapy have emerged as promising approaches for the treatment of various diseases. This special issue aims to provide a comprehensive overview of the recent advancements and challenges in the field of gene and cell therapy. This special issue aims to provide a comprehensive overview of the recent advancements and challenges in the field of gene and cell therapy, with a particular focus on molecular-level insights. The issue will cover a wide range of topics, including novel gene delivery systems, gene editing techniques, stem cell-based therapies, with an emphasis on molecular mechanisms underlying their efficacy and ssafety. The use of viral and non-viral vectors for gene delivery has significantly improved the efficiency and specificity of gene therapy. Understanding the molecular interactions between the vectors and the target cells is crucial for optimizing their delivery and enhancing therapeutic outcomes. Additionally, the advent of gene editing technologies, such as CRISPR/Cas9, has revolutionized the field by enabling precise modifications of the genome. Moreover, stem cells have demonstrated tremendous potential in regenerative medicine, with their ability to differentiate into various cell types and promote tissue repair. Elucidating the molecular signaling pathways and epigenetic modifications involved in stem cell differentiation and therapeutic effects is essential for harnessing their full therapeutic potential. This special issue invites original research articles, reviews, and perspectives that provide molecular-level insights into gene and cell therapy. Contributions focusing on molecular mechanisms underlying the safety and efficacy of these therapies are particularly encouraged. Moreover, studies highlighting the molecular basis of the preclinical and clinical applications of gene and cell therapies for specific diseases will be of great interest. This special issue aims to advance our understanding of the intricate molecular processes involved in gene and cell therapy facilitating the translation of gene and cell therapies into effective clinical interventions.

Dr. Elena Herrera Carrillo
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.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. International Journal of Molecular Sciences is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. There is an Article Processing Charge (APC) for publication in this open access journal. For details about the APC please see here. Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • gene therapy
  • cell therapy
  • gene delivery
  • gene editing
  • stem cells
  • regenerative medicine
  • CRISPR-Cas
  • viral vectors
  • non-viral vectors
  • immunotherapies

Published Papers (1 paper)

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Research

15 pages, 4222 KiB  
Article
CRISPR-Cas12a for Highly Efficient and Marker-Free Targeted Integration in Human Pluripotent Stem Cells
by Ruba Hammad, Jamal Alzubi, Manuel Rhiel, Kay O. Chmielewski, Laura Mosti, Julia Rositzka, Marcel Heugel, Jan Lawrenz, Valentina Pennucci, Birgitta Gläser, Judith Fischer, Axel Schambach, Thomas Moritz, Nico Lachmann, Tatjana I. Cornu, Claudio Mussolino, Richard Schäfer and Toni Cathomen
Int. J. Mol. Sci. 2024, 25(2), 985; https://doi.org/10.3390/ijms25020985 - 12 Jan 2024
Viewed by 1278
Abstract
The CRISPR-Cas12a platform has attracted interest in the genome editing community because the prototypical Acidaminococcus Cas12a generates a staggered DNA double-strand break upon binding to an AT-rich protospacer-adjacent motif (PAM, 5′-TTTV). The broad application of the platform in primary human cells was enabled [...] Read more.
The CRISPR-Cas12a platform has attracted interest in the genome editing community because the prototypical Acidaminococcus Cas12a generates a staggered DNA double-strand break upon binding to an AT-rich protospacer-adjacent motif (PAM, 5′-TTTV). The broad application of the platform in primary human cells was enabled by the development of an engineered version of the natural Cas12a protein, called Cas12a Ultra. In this study, we confirmed that CRISPR-Cas12a Ultra ribonucleoprotein complexes enabled allelic gene disruption frequencies of over 90% at multiple target sites in human T cells, hematopoietic stem and progenitor cells (HSPCs), and induced pluripotent stem cells (iPSCs). In addition, we demonstrated, for the first time, the efficient knock-in potential of the platform in human iPSCs and achieved targeted integration of a GFP marker gene into the AAVS1 safe harbor site and a CSF2RA super-exon into CSF2RA in up to 90% of alleles without selection. Clonal analysis revealed bi-allelic integration in >50% of the screened iPSC clones without compromising their pluripotency and genomic integrity. Thus, in combination with the adeno-associated virus vector system, CRISPR-Cas12a Ultra provides a highly efficient genome editing platform for performing targeted knock-ins in human iPSCs. Full article
(This article belongs to the Special Issue Current Molecular Progress on Cell and Gene Therapies)
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