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  1. Article ; Online: Gene regulation in inborn errors of immunity: Implications for gene therapy design and efficacy.

    Ghanim, Hana Y / Porteus, Matthew H

    Immunological reviews

    2024  Volume 322, Issue 1, Page(s) 157–177

    Abstract: Inborn errors of immunity (IEI) present a unique paradigm in the realm of gene therapy, emphasizing the need for precision in therapeutic design. As gene therapy transitions from broad-spectrum gene addition to careful modification of specific genes, the ...

    Abstract Inborn errors of immunity (IEI) present a unique paradigm in the realm of gene therapy, emphasizing the need for precision in therapeutic design. As gene therapy transitions from broad-spectrum gene addition to careful modification of specific genes, the enduring safety and effectiveness of these therapies in clinical settings have become crucial. This review discusses the significance of IEIs as foundational models for pioneering and refining precision medicine. We explore the capabilities of gene addition and gene correction platforms in modifying the DNA sequence of primary cells tailored for IEIs. The review uses four specific IEIs to highlight key issues in gene therapy strategies: X-linked agammaglobulinemia (XLA), X-linked chronic granulomatous disease (X-CGD), X-linked hyper IgM syndrome (XHIGM), and immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX). We detail the regulatory intricacies and therapeutic innovations for each disorder, incorporating insights from relevant clinical trials. For most IEIs, regulated expression is a vital aspect of the underlying biology, and we discuss the importance of endogenous regulation in developing gene therapy strategies.
    MeSH term(s) Humans ; Genetic Diseases, X-Linked/genetics ; Genetic Diseases, X-Linked/therapy ; Intestinal Diseases/genetics ; Intestinal Diseases/therapy ; Agammaglobulinemia/genetics ; Agammaglobulinemia/therapy ; Genetic Therapy
    Language English
    Publishing date 2024-01-17
    Publishing country England
    Document type Journal Article ; Review
    ZDB-ID 391796-4
    ISSN 1600-065X ; 0105-2896
    ISSN (online) 1600-065X
    ISSN 0105-2896
    DOI 10.1111/imr.13305
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  2. Article ; Online: A New Class of Medicines through DNA Editing.

    Porteus, Matthew H

    The New England journal of medicine

    2019  Volume 380, Issue 10, Page(s) 947–959

    MeSH term(s) CRISPR-Associated Protein 9 ; CRISPR-Cas Systems ; DNA ; Drug Discovery/methods ; Gene Editing/history ; Gene Editing/methods ; History, 20th Century ; History, 21st Century
    Chemical Substances DNA (9007-49-2) ; CRISPR-Associated Protein 9 (EC 3.1.-)
    Language English
    Publishing date 2019-03-11
    Publishing country United States
    Document type Historical Article ; Journal Article ; Review
    ZDB-ID 207154-x
    ISSN 1533-4406 ; 0028-4793
    ISSN (online) 1533-4406
    ISSN 0028-4793
    DOI 10.1056/NEJMra1800729
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  3. Article ; Online: A Curative DNA Code for Hematopoietic Defects: Novel Cell Therapies for Monogenic Diseases of the Blood and Immune System.

    Porteus, Matthew H / Pavel-Dinu, Mara / Pai, Sung-Yun

    Hematology/oncology clinics of North America

    2022  Volume 36, Issue 4, Page(s) 647–665

    Abstract: Innovations in programmable nucleases have expanded genetic engineering capabilities, raising the possibility of a new approach to curing monogenic hematological diseases. Feasibility studies using ex vivo targeted genome-editing, and nonintegrating ... ...

    Abstract Innovations in programmable nucleases have expanded genetic engineering capabilities, raising the possibility of a new approach to curing monogenic hematological diseases. Feasibility studies using ex vivo targeted genome-editing, and nonintegrating viral vectors show outstanding potential for correcting genetic conditions at their root cause. This article reviews the latest technological advances in the CRISPR/Cas9 system alone and combined with engineered viruses as editing tools for human hematopoietic stem and progenitor cells (HSPCs). We discuss the early phase in human trials of genome editing-based therapies for hemoglobinopathies.
    MeSH term(s) CRISPR-Cas Systems ; DNA ; Gene Editing ; Genetic Vectors ; Hematopoietic Stem Cells ; Humans ; Immune System
    Chemical Substances DNA (9007-49-2)
    Language English
    Publishing date 2022-06-27
    Publishing country United States
    Document type Journal Article ; Review ; Research Support, N.I.H., Intramural
    ZDB-ID 93115-9
    ISSN 1558-1977 ; 0889-8588
    ISSN (online) 1558-1977
    ISSN 0889-8588
    DOI 10.1016/j.hoc.2022.05.002
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  4. Article: Genome Editing for the β-Hemoglobinopathies.

    Porteus, Matthew H

    Advances in experimental medicine and biology

    2017  Volume 1013, Page(s) 203–217

    Abstract: The β-hemoglobinopathies are diverse set of disorders caused by mutations in the β-globin (HBB) gene. Because HBB protein is a critical component (along with α-globin, heme, and iron) of hemoglobin, the molecule essential for oxygen delivery to tissues, ... ...

    Abstract The β-hemoglobinopathies are diverse set of disorders caused by mutations in the β-globin (HBB) gene. Because HBB protein is a critical component (along with α-globin, heme, and iron) of hemoglobin, the molecule essential for oxygen delivery to tissues, mutations in HBB can result in lethal diseases or diseases with multi-organ dysfunction. HBB mutations can be roughly divided into two categories: those that cause a dysfunctional protein (such as sickle cell disease but also including varied diseases caused by high-affinity hemoglobins, low-affinity hemoglobins, and methemoglobinemia) and those that cause the insufficient production of HBB protein (β-thalassemia). Sickle cell disease and β-thalassemia are both the most prevalent and the most devastating of the β-hemoglobinopathies.
    Language English
    Publishing date 2017
    Publishing country United States
    Document type Journal Article
    ISSN 2214-8019 ; 0065-2598
    ISSN (online) 2214-8019
    ISSN 0065-2598
    DOI 10.1007/978-1-4939-7299-9_8
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  5. Article ; Online: Knock-in editing: it functionally corrects!

    Porteus, Matthew H

    Blood

    2016  Volume 127, Issue 21, Page(s) 2507–2509

    MeSH term(s) CRISPR-Cas Systems ; Gene Editing ; Humans ; RNA Editing
    Language English
    Publishing date 2016-05-26
    Publishing country United States
    Document type Journal Article ; Comment
    ZDB-ID 80069-7
    ISSN 1528-0020 ; 0006-4971
    ISSN (online) 1528-0020
    ISSN 0006-4971
    DOI 10.1182/blood-2016-03-703181
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  6. Article: Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease.

    Xu, Liwen / Lahiri, Premanjali / Skowronski, Jason / Bhatia, Neehar / Lattanzi, Annalisa / Porteus, Matthew H

    Molecular therapy. Methods & clinical development

    2023  Volume 30, Page(s) 317–331

    Abstract: ... Ex ... ...

    Abstract Ex vivo
    Language English
    Publishing date 2023-07-27
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2872938-9
    ISSN 2329-0501 ; 2329-0501
    ISSN (online) 2329-0501
    ISSN 2329-0501
    DOI 10.1016/j.omtm.2023.07.009
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  7. Article ; Online: Reply to "Efficient Nuclease-free HR by Clade F AAV Requires High MOIs with High Quality Vectors".

    Dudek, Amanda M / Porteus, Matthew H

    Molecular therapy : the journal of the American Society of Gene Therapy

    2019  Volume 27, Issue 12, Page(s) 2063

    MeSH term(s) Dependovirus/genetics ; Gene Editing ; Genetic Vectors ; Homologous Recombination ; Humans
    Language English
    Publishing date 2019-11-11
    Publishing country United States
    Document type Letter ; Comment
    ZDB-ID 2010592-7
    ISSN 1525-0024 ; 1525-0016
    ISSN (online) 1525-0024
    ISSN 1525-0016
    DOI 10.1016/j.ymthe.2019.11.004
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  8. Article ; Online: AAV6 Is Superior to Clade F AAVs in Stimulating Homologous Recombination-Based Genome Editing in Human HSPCs.

    Dudek, Amanda M / Porteus, Matthew H

    Molecular therapy : the journal of the American Society of Gene Therapy

    2019  Volume 27, Issue 10, Page(s) 1701–1705

    MeSH term(s) DNA Breaks ; Dependovirus/genetics ; Gene Editing ; Genome, Human ; Homologous Recombination ; Humans
    Language English
    Publishing date 2019-09-12
    Publishing country United States
    Document type Letter ; Comment
    ZDB-ID 2010592-7
    ISSN 1525-0024 ; 1525-0016
    ISSN (online) 1525-0024
    ISSN 1525-0016
    DOI 10.1016/j.ymthe.2019.09.005
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  9. Article ; Online: Towards a new era in medicine: therapeutic genome editing.

    Porteus, Matthew H

    Genome biology

    2015  Volume 16, Page(s) 286

    Abstract: Genome editing is the process of precisely modifying the nucleotide sequence of the genome. It has provided a powerful approach to research questions but, with the development of a new set of tools, it is now possible to achieve frequencies of genome ... ...

    Abstract Genome editing is the process of precisely modifying the nucleotide sequence of the genome. It has provided a powerful approach to research questions but, with the development of a new set of tools, it is now possible to achieve frequencies of genome editing that are high enough to be useful therapeutically. Genome editing is being developed to treat not only monogenic diseases but also infectious diseases and diseases that have both a genetic and an environmental component.
    MeSH term(s) Animals ; Gene Targeting/methods ; Genetic Therapy/methods ; Genome ; Humans
    Language English
    Publishing date 2015-12-22
    Publishing country England
    Document type Journal Article ; Research Support, N.I.H., Extramural ; Review
    ZDB-ID 2040529-7
    ISSN 1474-760X ; 1465-6914 ; 1465-6906
    ISSN (online) 1474-760X ; 1465-6914
    ISSN 1465-6906
    DOI 10.1186/s13059-015-0859-y
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  10. Article: Genome Editing of the Blood: Opportunities and Challenges.

    Porteus, Matthew H

    Current stem cell reports

    2015  Volume 1, Issue 1, Page(s) 23–30

    Abstract: The ability to remove blood cells, including hematopoietic stem cells (HSCs), from a person and then re-transplant them (hematopoietic stem cell transplantation (HSCT) is a well-established treatment paradigm that can be used in both the autologous ... ...

    Abstract The ability to remove blood cells, including hematopoietic stem cells (HSCs), from a person and then re-transplant them (hematopoietic stem cell transplantation (HSCT) is a well-established treatment paradigm that can be used in both the autologous setting or in the allogeneic setting. Using allogeneic HSCT can cure different genetic diseases of the blood but has significant limitations. An alternative to allogeneic HSCT is to transplant genetically modified HSCs instead. A powerful approach to the precision modification of HSCs is to use genome editing whereby the genome is modified with spatial precision (at an exact location) in the genome and sometimes with nucleotide precision (the exact nucleotide changes are introduced). The progress and challenges of genome editing of blood are discussed.
    Language English
    Publishing date 2015-05-06
    Publishing country Switzerland
    Document type Journal Article
    ZDB-ID 2808623-5
    ISSN 2198-7866
    ISSN 2198-7866
    DOI 10.1007/s40778-014-0003-z
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