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  1. Article ; Online: Boolean immunotherapy: reversal of fortune.

    Morgan, Richard A

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

    2014  Volume 22, Issue 6, Page(s) 1073–1074

    MeSH term(s) Animals ; Humans ; Interleukin-4 Receptor alpha Subunit/genetics ; Neoplasms, Experimental/immunology ; Neoplasms, Experimental/therapy ; Receptors, Interleukin-7/genetics ; T-Lymphocytes/immunology ; Tumor Microenvironment
    Chemical Substances Interleukin-4 Receptor alpha Subunit ; Receptors, Interleukin-7
    Language English
    Publishing date 2014-06-02
    Publishing country United States
    Document type Journal Article ; Comment
    ZDB-ID 2010592-7
    ISSN 1525-0024 ; 1525-0016
    ISSN (online) 1525-0024
    ISSN 1525-0016
    DOI 10.1038/mt.2014.75
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 5640: Show issues Location:
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  2. Article ; Online: Risky business: target choice in adoptive cell therapy.

    Morgan, Richard A

    Blood

    2013  Volume 122, Issue 20, Page(s) 3392–3394

    Abstract: In this issue of Blood, Casucci et al present an elegant study that describes a potential new target for adoptive cell transfer (ACT), in this case CD44 splice variant 6 (CD44v6), and detail why it may be a good target for ACT and how to manage expected ... ...

    Abstract In this issue of Blood, Casucci et al present an elegant study that describes a potential new target for adoptive cell transfer (ACT), in this case CD44 splice variant 6 (CD44v6), and detail why it may be a good target for ACT and how to manage expected off-tumor/on-target toxicities.
    MeSH term(s) Animals ; Antigens, Neoplasm/immunology ; Humans ; Hyaluronan Receptors/immunology ; Immunotherapy, Adoptive ; Leukemia, Myeloid, Acute/therapy ; Molecular Targeted Therapy ; Multiple Myeloma/therapy ; T-Lymphocyte Subsets/immunology
    Chemical Substances Antigens, Neoplasm ; CD44v6 antigen ; Hyaluronan Receptors
    Language English
    Publishing date 2013-11-13
    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-2013-09-527622
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  3. Article ; Online: Faster, cheaper, safer, T-cell engineering.

    Morgan, Richard A

    Journal of immunotherapy (Hagerstown, Md. : 1997)

    2012  Volume 36, Issue 1, Page(s) 1–2

    MeSH term(s) Humans ; Nerve Tissue Proteins/genetics ; T-Lymphocytes/metabolism ; Transgenes/genetics
    Chemical Substances Nerve Tissue Proteins ; PGBD1 protein, human
    Language English
    Publishing date 2012-12-01
    Publishing country United States
    Document type Journal Article ; Comment
    ZDB-ID 1064067-8
    ISSN 1537-4513 ; 1053-8550 ; 1524-9557
    ISSN (online) 1537-4513
    ISSN 1053-8550 ; 1524-9557
    DOI 10.1097/CJI.0b013e3182791257
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 1778: Show issues Location:
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  4. Article ; Online: Human tumor xenografts: the good, the bad, and the ugly.

    Morgan, Richard A

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

    2012  Volume 20, Issue 5, Page(s) 882–884

    MeSH term(s) Animals ; Crosses, Genetic ; Genetic Heterogeneity ; Humans ; Mice ; Mice, Inbred NOD ; Mice, Nude ; Mice, SCID ; Neoplasms/genetics ; Neoplasms/immunology ; Neoplasms, Experimental/genetics ; Neoplasms, Experimental/immunology ; Xenograft Model Antitumor Assays
    Language English
    Publishing date 2012-04-30
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2010592-7
    ISSN 1525-0024 ; 1525-0016
    ISSN (online) 1525-0024
    ISSN 1525-0016
    DOI 10.1038/mt.2012.73
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  5. Article ; Online: Live and let die: a new suicide gene therapy moves to the clinic.

    Morgan, Richard A

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

    2011  Volume 20, Issue 1, Page(s) 11–13

    MeSH term(s) Genes, Transgenic, Suicide ; Genetic Therapy ; Humans
    Language English
    Publishing date 2011-11-25
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2010592-7
    ISSN 1525-0024 ; 1525-0016
    ISSN (online) 1525-0024
    ISSN 1525-0016
    DOI 10.1038/mt.2011.273
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  6. Article: Genetic Modification of T Cells.

    Morgan, Richard A / Boyerinas, Benjamin

    Biomedicines

    2016  Volume 4, Issue 2

    Abstract: Gene transfer technology and its application to human gene therapy greatly expanded in the last decade. One area of investigation that appears particularly promising is the transfer of new genetic material into T cells for the potential treatment of ... ...

    Abstract Gene transfer technology and its application to human gene therapy greatly expanded in the last decade. One area of investigation that appears particularly promising is the transfer of new genetic material into T cells for the potential treatment of cancer. Herein, we describe several core technologies that now yield high-efficiency gene transfer into primary human T cells. These gene transfer techniques include viral-based gene transfer methods based on modified
    Language English
    Publishing date 2016-04-20
    Publishing country Switzerland
    Document type Journal Article ; Review
    ZDB-ID 2720867-9
    ISSN 2227-9059
    ISSN 2227-9059
    DOI 10.3390/biomedicines4020009
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  7. Article ; Online: Salient Features of Endonuclease Platforms for Therapeutic Genome Editing.

    Certo, Michael T / Morgan, Richard A

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

    2016  Volume 24, Issue 3, Page(s) 422–429

    Abstract: Emerging gene-editing technologies are nearing a revolutionary phase in genetic medicine: precisely modifying or repairing causal genetic defects. This may include any number of DNA sequence manipulations, such as knocking out a deleterious gene, ... ...

    Abstract Emerging gene-editing technologies are nearing a revolutionary phase in genetic medicine: precisely modifying or repairing causal genetic defects. This may include any number of DNA sequence manipulations, such as knocking out a deleterious gene, introducing a particular mutation, or directly repairing a defective sequence by site-specific recombination. All of these edits can currently be achieved via programmable rare-cutting endonucleases to create targeted DNA breaks that can engage and exploit endogenous DNA repair pathways to impart site-specific genetic changes. Over the past decade, several distinct technologies for introducing site-specific DNA breaks have been developed, yet the different biological origins of these gene-editing technologies bring along inherent differences in parameters that impact clinical implementation. This review aims to provide an accessible overview of the various endonuclease-based gene-editing platforms, highlighting the strengths and weakness of each with respect to therapeutic applications.
    MeSH term(s) Animals ; Endonucleases/metabolism ; Gene Editing/history ; Gene Editing/methods ; Gene Transfer Techniques ; Genetic Engineering/history ; Genetic Engineering/methods ; Genetic Therapy/methods ; Genetic Vectors/genetics ; Genome ; History, 20th Century ; Humans ; Transduction, Genetic
    Chemical Substances Endonucleases (EC 3.1.-)
    Language English
    Publishing date 2016-03
    Publishing country United States
    Document type Historical Article ; Journal Article ; Review
    ZDB-ID 2010592-7
    ISSN 1525-0024 ; 1525-0016
    ISSN (online) 1525-0024
    ISSN 1525-0016
    DOI 10.1038/mt.2016.21
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  8. Article ; Online: Genetic modification of T cells.

    Morgan, Richard A / Kakarla, Sunitha

    Cancer journal (Sudbury, Mass.)

    2014  Volume 20, Issue 2, Page(s) 145–150

    Abstract: Gene transfer technology has advanced rapidly from simple physical-chemical laboratory methods in the 1970s and 1980s to the sophisticated viral and nonviral methods currently in clinical practice. Herein, we review 4 gene transfer methodologies applied ... ...

    Abstract Gene transfer technology has advanced rapidly from simple physical-chemical laboratory methods in the 1970s and 1980s to the sophisticated viral and nonviral methods currently in clinical practice. Herein, we review 4 gene transfer methodologies applied in human gene therapy clinical trials transferring chimeric antigen receptors into T cells for the treatment of B-cell malignancies. The 4 methods include 2 viral vector gene transfer technologies, gamma retroviral vectors and lentiviral vectors, and 2 nonviral methods, transposons and mRNA electroporation.
    MeSH term(s) DNA Transposable Elements/genetics ; Electroporation/methods ; Gene Transfer Techniques ; Genetic Vectors ; Humans ; Lentivirus/genetics ; RNA, Messenger/genetics ; Retroviridae/genetics
    Chemical Substances DNA Transposable Elements ; RNA, Messenger
    Language English
    Publishing date 2014-03
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 2018400-1
    ISSN 1540-336X ; 1528-9117 ; 1081-4442
    ISSN (online) 1540-336X
    ISSN 1528-9117 ; 1081-4442
    DOI 10.1097/PPO.0000000000000033
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    Zs.A 4470: Show issues Location:
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    Zs.MO 163: Show issues

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  9. Article ; Online: Lentiviral gene therapy for X-linked chronic granulomatous disease recapitulates endogenous CYBB regulation and expression.

    Wong, Ryan L / Sackey, Sarah / Brown, Devin / Senadheera, Shantha / Masiuk, Katelyn / Quintos, Jason P / Colindres, Nicole / Riggan, Luke / Morgan, Richard A / Malech, Harry L / Hollis, Roger P / Kohn, Donald B

    Blood

    2023  Volume 141, Issue 9, Page(s) 1007–1022

    Abstract: X-linked chronic granulomatous disease (X-CGD) is a primary immunodeficiency caused by mutations in the CYBB gene, resulting in the inability of phagocytic cells to eliminate infections. To design a lentiviral vector (LV) capable of recapitulating the ... ...

    Abstract X-linked chronic granulomatous disease (X-CGD) is a primary immunodeficiency caused by mutations in the CYBB gene, resulting in the inability of phagocytic cells to eliminate infections. To design a lentiviral vector (LV) capable of recapitulating the endogenous regulation and expression of CYBB, a bioinformatics-guided approach was used to elucidate the cognate enhancer elements regulating the native CYBB gene. Using this approach, we analyzed a 600-kilobase topologically associated domain of the CYBB gene and identified endogenous enhancer elements to supplement the CYBB promoter to develop MyeloVec, a physiologically regulated LV for the treatment of X-CGD. When compared with an LV currently in clinical trials for X-CGD, MyeloVec showed improved expression, superior gene transfer to hematopoietic stem and progenitor cells (HSPCs), corrected an X-CGD mouse model leading to complete protection against Burkholderia cepacia infection, and restored healthy donor levels of antimicrobial oxidase activity in neutrophils derived from HSPCs from patients with X-CGD. Our findings validate the bioinformatics-guided design approach and have yielded a novel LV with clinical promise for the treatment of X-CGD.
    MeSH term(s) Animals ; Mice ; Granulomatous Disease, Chronic/genetics ; Granulomatous Disease, Chronic/therapy ; NADPH Oxidases/genetics ; NADPH Oxidases/metabolism ; NADPH Oxidase 2/genetics ; Genetic Therapy/methods ; Mutation
    Chemical Substances NADPH Oxidases (EC 1.6.3.-) ; NADPH Oxidase 2 (EC 1.6.3.-) ; Cybb protein, mouse (EC 1.6.3.-)
    Language English
    Publishing date 2023-03-02
    Publishing country United States
    Document type Journal Article
    ZDB-ID 80069-7
    ISSN 1528-0020 ; 0006-4971
    ISSN (online) 1528-0020
    ISSN 0006-4971
    DOI 10.1182/blood.2022016074
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  10. Article ; Online: Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned.

    Morgan, Richard A / Gray, David / Lomova, Anastasia / Kohn, Donald B

    Cell stem cell

    2018  Volume 21, Issue 5, Page(s) 574–590

    Abstract: The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases has become a clinical standard but is limited by the availability of suitable matched donors and potential immunologic complications. Gene therapy using autologous ...

    Abstract The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases has become a clinical standard but is limited by the availability of suitable matched donors and potential immunologic complications. Gene therapy using autologous HSCs should avoid these limitations and thus may be safer. Progressive improvements in techniques for genetic correction of HSCs, by either vector gene addition or gene editing, are facilitating successful treatments for an increasing number of diseases. We highlight the progress, successes, and remaining challenges toward the development of HSC gene therapies and discuss lessons they provide for the development of future clinical stem cell therapies.
    MeSH term(s) Clinical Trials as Topic ; Gene Editing ; Gene Transfer Techniques ; Genetic Therapy ; Genetic Vectors/metabolism ; Hematopoietic Stem Cell Transplantation/adverse effects ; Hematopoietic Stem Cells/cytology ; Humans
    Language English
    Publishing date 2018-01-01
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 2375354-7
    ISSN 1875-9777 ; 1934-5909
    ISSN (online) 1875-9777
    ISSN 1934-5909
    DOI 10.1016/j.stem.2017.10.010
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    Zs.MG 75: Show issues

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