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New Frontiers in Molecular Hematology
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New Frontiers in Molecular Hematology

A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Hematology".

Deadline for manuscript submissions: closed (15 December 2020) | Viewed by 26334

Special Issue Editor

Prof. Dr. Marina Cavazzana

E-Mail Website
Guest Editor
1. Professor in Haematology, Paris University Medical School, Paris, France
2. Head of the Biotherapy Department, Hôpital Necker-Enfants Malades, Assistance Publique-Hôpitaux de Paris, Paris, France
3. Director of the Clinical Investigation Center for Innovative Therapies, Imagine Institute, INSERM UMR 1163, Paris, France
Interests: gene and cell therapy; hematopoietic stem cell; hematopoietic stem cell transplantation immune deficiencies; immunological disorders; genetic diseases; lentiviral vector

Special Issue Information

Dear Colleagues,

The severe complications that still exist on hematopoietic stem cell transplants in the absence of a compatible donor led us, 20 years ago, to explore another therapeutic strategy for genetic diseases of the hematopoietic system. The key idea behind the treatment of these inherited diseases is simple: The diseased bone marrow is replaced with autologous gene-modified cells, in order to establish a stable expression of the missing protein and thus restore its function. Several inherited immune deficiencies and blood disorders can be cured with the use of a lentiviral vector for people affected by immune deficiencies worldwide. Our aim is to bring this powerful innovation to all affected patients.

Prof. Dr. Marina Cavazzana
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. Journal of Clinical Medicine is an international peer-reviewed open access semimonthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 2600 CHF (Swiss Francs). 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 and cell therapy
  • Hematopoietic stem cell
  • Hematopoietic stem cell transplantation immune deficiencies
  • Immunological disorders
  • Genetic diseases
  • Lentiviral vector

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Published Papers (4 papers)

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Research

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9 pages, 225 KiB  
Article
The Influence of CD28 Gene Polymorphism in Transfusion Reaction after Transfusing Leukoreduced Blood Components
by Ding-Ping Chen, Wei-Tzu Lin, Wei-Ting Wang and Tzong-Shi Chiueh
J. Clin. Med. 2020, 9(3), 877; https://doi.org/10.3390/jcm9030877 - 23 Mar 2020
Cited by 3 | Viewed by 2628
Abstract
CTLA-4 and CD28 belong to co-stimulation molecules, the abnormal expression of which can regulate the T cell activation and then affect the degree of immune response. Moreover, blood transfusion reaction (TR) is a kind of immune reaction. Consequently, the hypothesis of this study [...] Read more.
CTLA-4 and CD28 belong to co-stimulation molecules, the abnormal expression of which can regulate the T cell activation and then affect the degree of immune response. Moreover, blood transfusion reaction (TR) is a kind of immune reaction. Consequently, the hypothesis of this study was that the TR still occurred after transfusing leukoreduced blood components as a result of the sensitivity of immune system, and a small number of immune stimulations were enough to induce an immune response in patients. There were 38 cases and 36 healthy controls included in this study. The selected CD28 gene were sequenced to search single nucleotide polymorphism (SNPs), and the correlation between TR and these SNPs was analyzed. According to our data, only the rs3181097 (promoter, −1059) of CD28 gene polymorphism was associated with TR. The p value of rs3181097 under the co-dominant model was 0.031. GG was used as a reference genotype, the p value of GG vs. AG was 0.010 (OR: 0.210, 95% CI: 0.062–0.712), and GG vs. AG + AA was 0.028 (OR: 0.292, 95% CI: 0.095–0.901). In addition to CTLA-4, CD28 gene was also a crucial SNP in TR, because there was a signification for the haplotype with Grs3181097 (p = 0.015). Consequently, we suggested that the TR was related to CD28 by regulating the degree of immune response. Full article
(This article belongs to the Special Issue New Frontiers in Molecular Hematology)

Review

Jump to: Research

24 pages, 2484 KiB  
Review
Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia
by Paul E. Monahan, Claude Négrier, Michael Tarantino, Leonard A. Valentino and Federico Mingozzi
J. Clin. Med. 2021, 10(11), 2471; https://doi.org/10.3390/jcm10112471 - 2 Jun 2021
Cited by 59 | Viewed by 11808
Abstract
Adeno-associated viral (AAV) vector gene therapy has shown promise as a possible cure for hemophilia. However, immune responses directed against AAV vectors remain a hurdle to the broader use of this gene transfer platform. Both innate and adaptive immune responses can affect the [...] Read more.
Adeno-associated viral (AAV) vector gene therapy has shown promise as a possible cure for hemophilia. However, immune responses directed against AAV vectors remain a hurdle to the broader use of this gene transfer platform. Both innate and adaptive immune responses can affect the safety and efficacy of AAV vector–mediated gene transfer in humans. These immune responses may be triggered by the viral capsid, the vector’s nucleic acid payload, or other vector contaminants or excipients, or by the transgene product encoded by the vector itself. Various preclinical and clinical strategies have been explored to overcome the issues of AAV vector immunogenicity and transgene-related immune responses. Although results of these strategies are encouraging, more efficient approaches are needed to deliver safe, predictable, and durable outcomes for people with hemophilia. In addition to durability, long-term follow-up of gene therapy trial participants will allow us to address potential safety concerns related to vector integration. Herein, we describe the challenges with current methodologies to deliver optimal outcomes for people with hemophilia who choose to undergo AAV vector gene therapy and the potential opportunities to improve on the results. Full article
(This article belongs to the Special Issue New Frontiers in Molecular Hematology)
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22 pages, 7491 KiB  
Review
Advances and Obstacles in Homology-Mediated Gene Editing of Hematopoietic Stem Cells
by Christi T. Salisbury-Ruf and Andre Larochelle
J. Clin. Med. 2021, 10(3), 513; https://doi.org/10.3390/jcm10030513 - 1 Feb 2021
Cited by 11 | Viewed by 4200
Abstract
Homology-directed gene editing of hematopoietic stem and progenitor cells (HSPCs) is a promising strategy for the treatment of inherited blood disorders, obviating many of the limitations associated with viral vector-mediated gene therapies. The use of CRISPR/Cas9 or other programmable nucleases and improved methods [...] Read more.
Homology-directed gene editing of hematopoietic stem and progenitor cells (HSPCs) is a promising strategy for the treatment of inherited blood disorders, obviating many of the limitations associated with viral vector-mediated gene therapies. The use of CRISPR/Cas9 or other programmable nucleases and improved methods of homology template delivery have enabled precise ex vivo gene editing. These transformative advances have also highlighted technical challenges to achieve high-efficiency gene editing in HSPCs for therapeutic applications. In this review, we discuss recent pre-clinical investigations utilizing homology-mediated gene editing in HSPCs and highlight various strategies to improve editing efficiency in these cells. Full article
(This article belongs to the Special Issue New Frontiers in Molecular Hematology)
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16 pages, 1460 KiB  
Review
Genome Editing for β-Hemoglobinopathies: Advances and Challenges
by Giacomo Frati and Annarita Miccio
J. Clin. Med. 2021, 10(3), 482; https://doi.org/10.3390/jcm10030482 - 28 Jan 2021
Cited by 23 | Viewed by 6661
Abstract
β-hemoglobinopathies are the most common genetic disorders worldwide and are caused by mutations affecting the production or the structure of adult hemoglobin. Patients affected by these diseases suffer from anemia, impaired oxygen delivery to tissues, and multi-organ damage. In the absence of a [...] Read more.
β-hemoglobinopathies are the most common genetic disorders worldwide and are caused by mutations affecting the production or the structure of adult hemoglobin. Patients affected by these diseases suffer from anemia, impaired oxygen delivery to tissues, and multi-organ damage. In the absence of a compatible donor for allogeneic bone marrow transplantation, the lifelong therapeutic options are symptomatic care, red blood cell transfusions and pharmacological treatments. The last decades of research established lentiviral-mediated gene therapy as an efficacious therapeutic strategy. However, this approach is highly expensive and associated with a variable outcome depending on the effectiveness of the viral vector and the quality of the cell product. In the last years, genome editing emerged as a valuable tool for the development of curative strategies for β-hemoglobinopathies. Moreover, due to the wide range of its applications, genome editing has been extensively used to study regulatory mechanisms underlying globin gene regulation allowing the identification of novel genetic and pharmacological targets. In this work, we review the current advances and challenges of genome editing approaches to β-hemoglobinopathies. Special focus has been directed towards strategies aimed at correcting the defective β-globin gene or at inducing fetal hemoglobin (HbF), which are in an advanced state of clinical development. Full article
(This article belongs to the Special Issue New Frontiers in Molecular Hematology)
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