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Genome Editing Therapies
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Genome Editing Therapies

A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Genetics and Genomics".

Deadline for manuscript submissions: closed (15 October 2019) | Viewed by 24283

Special Issue Editors

Dr. Toshifumi Yokota

E-Mail Website
Guest Editor
Department of Medical Genetics, University of Alberta, Edmonton, AB, Canada
Interests: musculoskeletal disease; facioscapulohumeral muscular dystrophy (FSHD); CRISPR/Cas9 genome-editing; BMP signaling; Fibrodysplasia Ossificans Progressiva (FOP)
Special Issues, Collections and Topics in MDPI journals
Dr. Rika Maruyama

E-Mail Website
Guest Editor
Department of Medical Genetics, University of Alberta, Edmonton, AB, Canada
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

The recent advances in the development of genome editing technologies have significantly improved our ability to directly make changes in the genomes in a precise manner. A particularly attractive application of genome editing is the potential to directly correct genetic mutations for the treatment of currently incurable diseases. Genome-editing therapy has already shown promise in treating inherited genetic diseases, such as Duchenne muscular dystrophy. This Special Issue, Genome Editing Therapies, will focus on the recent progress in the development of novel therapies using genome editing technologies. We intend to invite researchers in the field to submit original research and review articles on the advancement of novel genome-editing therapies, including (but not limited to) the CRISPR-Cas /cpf1 system, TALEN, mutation-based personalized medicine, delivery enhancement, exosome-mediated delivery, off-target effects, bioinformatics, and disease models, to evaluate the efficacy of treatments.

Dr. Toshifumi Yokota
Dr. Rika Maruyama
Guest Editors

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

  • CRISPR
  • TALEN
  • Cas9
  • cpf1
  • genome editing
  • guide RNA

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Published Papers (4 papers)

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Research

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13 pages, 1639 KiB  
Article
Generation of New Isogenic Models of Huntington’s Disease Using CRISPR-Cas9 Technology
by Magdalena Dabrowska, Agata Ciolak, Emilia Kozlowska, Agnieszka Fiszer and Marta Olejniczak
Int. J. Mol. Sci. 2020, 21(5), 1854; https://doi.org/10.3390/ijms21051854 - 8 Mar 2020
Cited by 26 | Viewed by 8683
Abstract
Huntington’s disease (HD) is a fatal neurodegenerative disorder caused by the expansion of CAG repeats in exon 1 of the huntingtin gene (HTT). Despite its monogenic nature, HD pathogenesis is still not fully understood, and no effective therapy is available to [...] Read more.
Huntington’s disease (HD) is a fatal neurodegenerative disorder caused by the expansion of CAG repeats in exon 1 of the huntingtin gene (HTT). Despite its monogenic nature, HD pathogenesis is still not fully understood, and no effective therapy is available to patients. The development of new techniques such as genome engineering has generated new opportunities in the field of disease modeling and enabled the generation of isogenic models with the same genetic background. These models are very valuable for studying the pathogenesis of a disease and for drug screening. Here, we report the generation of a series of homozygous HEK 293T cell lines with different numbers of CAG repeats at the HTT locus and demonstrate their usefulness for testing therapeutic reagents. In addition, using the CRISPR-Cas9 system, we corrected the mutation in HD human induced pluripotent stem cells and generated a knock-out of the HTT gene, thus providing a comprehensive set of isogenic cell lines for HD investigation. Full article
(This article belongs to the Special Issue Genome Editing Therapies)
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16 pages, 972 KiB  
Article
Quantifying the Potential for Future Gene Therapy to Lower Lifetime Risk of Polygenic Late-Onset Diseases
by Roman Teo Oliynyk
Int. J. Mol. Sci. 2019, 20(13), 3352; https://doi.org/10.3390/ijms20133352 - 8 Jul 2019
Cited by 2 | Viewed by 3970
Abstract
Gene therapy techniques and genetic knowledge may sufficiently advance, within the next few decades, to support prophylactic gene therapy for the prevention of polygenic late-onset diseases. The risk of these diseases may, hypothetically, be lowered by correcting the effects of a subset of [...] Read more.
Gene therapy techniques and genetic knowledge may sufficiently advance, within the next few decades, to support prophylactic gene therapy for the prevention of polygenic late-onset diseases. The risk of these diseases may, hypothetically, be lowered by correcting the effects of a subset of common low effect gene variants. In this paper, simulations show that if such gene therapy were to become technically possible; and if the incidences of the treated diseases follow the proportional hazards model with a multiplicative genetic architecture composed of a sufficient number of common small effect gene variants, then: (a) late-onset diseases with the highest familial heritability will have the largest number of variants available for editing; (b) diseases that currently have the highest lifetime risk, particularly those with the highest incidence rate continuing into older ages, will prove the most challenging cases in lowering lifetime risk and delaying the age of onset at a population-wide level; (c) diseases that are characterized by the lowest lifetime risk will show the strongest and longest-lasting response to such therapies; and (d) longer life expectancy is associated with a higher lifetime risk of these diseases, and this tendency, while delayed, will continue after therapy. Full article
(This article belongs to the Special Issue Genome Editing Therapies)
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Review

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27 pages, 432 KiB  
Review
Genome Editing for the Understanding and Treatment of Inherited Cardiomyopathies
by Quynh Nguyen, Kenji Rowel Q. Lim and Toshifumi Yokota
Int. J. Mol. Sci. 2020, 21(3), 733; https://doi.org/10.3390/ijms21030733 - 22 Jan 2020
Cited by 25 | Viewed by 5268
Abstract
Cardiomyopathies are diseases of heart muscle, a significant percentage of which are genetic in origin. Cardiomyopathies can be classified as dilated, hypertrophic, restrictive, arrhythmogenic right ventricular or left ventricular non-compaction, although mixed morphologies are possible. A subset of neuromuscular disorders, notably Duchenne and [...] Read more.
Cardiomyopathies are diseases of heart muscle, a significant percentage of which are genetic in origin. Cardiomyopathies can be classified as dilated, hypertrophic, restrictive, arrhythmogenic right ventricular or left ventricular non-compaction, although mixed morphologies are possible. A subset of neuromuscular disorders, notably Duchenne and Becker muscular dystrophies, are also characterized by cardiomyopathy aside from skeletal myopathy. The global burden of cardiomyopathies is certainly high, necessitating further research and novel therapies. Genome editing tools, which include zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeats (CRISPR) systems have emerged as increasingly important technologies in studying this group of cardiovascular disorders. In this review, we discuss the applications of genome editing in the understanding and treatment of cardiomyopathy. We also describe recent advances in genome editing that may help improve these applications, and some future prospects for genome editing in cardiomyopathy treatment. Full article
(This article belongs to the Special Issue Genome Editing Therapies)
18 pages, 2155 KiB  
Review
Transplacental Gene Delivery (TPGD) as a Noninvasive Tool for Fetal Gene Manipulation in Mice
by Shingo Nakamura, Satoshi Watanabe, Naoko Ando, Masayuki Ishihara and Masahiro Sato
Int. J. Mol. Sci. 2019, 20(23), 5926; https://doi.org/10.3390/ijms20235926 - 25 Nov 2019
Cited by 15 | Viewed by 5477
Abstract
Transplacental gene delivery (TPGD) is a technique for delivering nucleic acids to fetal tissues via tail-vein injections in pregnant mice. After transplacental transport, administered nucleic acids enter fetal circulation and are distributed among fetal tissues. TPGD was established in 1995 by Tsukamoto et [...] Read more.
Transplacental gene delivery (TPGD) is a technique for delivering nucleic acids to fetal tissues via tail-vein injections in pregnant mice. After transplacental transport, administered nucleic acids enter fetal circulation and are distributed among fetal tissues. TPGD was established in 1995 by Tsukamoto et al., and its mechanisms, and potential applications have been further characterized since. Recently, discoveries of sequence specific nucleases, such as zinc-finger nuclease (ZFN), transcription activator-like effector nucleases (TALEN), and clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein-9 nuclease (Cas9) (CRISPR/Cas9), have revolutionized genome editing. In 2019, we demonstrated that intravenous injection of plasmid DNA containing CRISPR/Cas9 produced indels in fetal myocardial cells, which are comparatively amenable to transfection with exogenous DNA. In the future, this unique technique will allow manipulation of fetal cell functions in basic studies of fetal gene therapy. In this review, we describe developments of TPGD and discuss their applications to the manipulation of fetal cells. Full article
(This article belongs to the Special Issue Genome Editing Therapies)
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