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  1. Article ; Online: Towards improved yet regulated gene therapy for X-CGD.

    Kajaste-Rudnitski, Anna / Aiuti, Alessandro

    Blood

    2024  Volume 141, Issue 9, Page(s) 966–967

    MeSH term(s) Humans ; Granulomatous Disease, Chronic ; NADPH Oxidase 2
    Chemical Substances CYBB protein, human (EC 1.6.3.-) ; NADPH Oxidase 2 (EC 1.6.3.-)
    Language English
    Publishing date 2024-03-19
    Publishing country United States
    Document type Editorial ; Comment
    ZDB-ID 80069-7
    ISSN 1528-0020 ; 0006-4971
    ISSN (online) 1528-0020
    ISSN 0006-4971
    DOI 10.1182/blood.2022018800
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    Uh III Zs.140: Show issues Location:
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  2. Article ; Online: Newborn screening for severe combined immunodeficiency: changing the landscape of post-transplantation survival.

    Barzaghi, Federica / Aiuti, Alessandro

    Lancet (London, England)

    2023  Volume 402, Issue 10396, Page(s) 84–85

    MeSH term(s) Infant, Newborn ; Humans ; Severe Combined Immunodeficiency/diagnosis ; Neonatal Screening ; Hematopoietic Stem Cell Transplantation
    Language English
    Publishing date 2023-06-20
    Publishing country England
    Document type Journal Article ; Comment
    ZDB-ID 3306-6
    ISSN 1474-547X ; 0023-7507 ; 0140-6736
    ISSN (online) 1474-547X
    ISSN 0023-7507 ; 0140-6736
    DOI 10.1016/S0140-6736(23)01057-7
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  3. Article ; Online: Realizing the Potential of Gene Therapies for Rare and Ultra-Rare Inherited Diseases.

    Booth, Claire / Aiuti, Alessandro

    Human gene therapy

    2023  Volume 34, Issue 17-18, Page(s) 776–781

    Abstract: Rare and ultrarare diseases have been central to the field of gene therapy since its earliest stage, and we are now witnessing more and more effective treatments entering the clinical realm for patients in need. However, despite promising results across ... ...

    Abstract Rare and ultrarare diseases have been central to the field of gene therapy since its earliest stage, and we are now witnessing more and more effective treatments entering the clinical realm for patients in need. However, despite promising results across a range of rare diseases, transformative gene therapies may not be available and accessible to patients for nonmedical reasons. Traditional regulatory and commercialization pathways to licensed products seem to be prohibitive for ultrasmall patient populations. Here, we highlight some of the challenges of delivering gene therapies in rare diseases and discuss innovative solutions being proposed by the gene therapy community.
    MeSH term(s) Humans ; Rare Diseases/genetics ; Rare Diseases/therapy ; Genetic Therapy
    Language English
    Publishing date 2023-08-25
    Publishing country United States
    Document type Journal Article ; Review ; Research Support, Non-U.S. Gov't
    ZDB-ID 1028152-6
    ISSN 1557-7422 ; 1043-0342
    ISSN (online) 1557-7422
    ISSN 1043-0342
    DOI 10.1089/hum.2023.127
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  4. Article ; Online: Ensuring a future for gene therapy for rare diseases.

    Aiuti, Alessandro / Pasinelli, Francesca / Naldini, Luigi

    Nature medicine

    2022  Volume 28, Issue 10, Page(s) 1985–1988

    MeSH term(s) Genetic Therapy ; Humans ; Rare Diseases/genetics ; Rare Diseases/therapy
    Language English
    Publishing date 2022-08-12
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 1220066-9
    ISSN 1546-170X ; 1078-8956
    ISSN (online) 1546-170X
    ISSN 1078-8956
    DOI 10.1038/s41591-022-01934-9
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  5. Article: Editorial to the Special Issue "Clinical Immunology in Italy, with Special Emphasis to Primary and Acquired Immunodeficiencies: A Commemorative Issue in Honor of Prof. Fernando Aiuti".

    Aiuti, Alessandro / D'Amelio, Raffaele / Quinti, Isabella / Rossi, Paolo

    Biomedicines

    2023  Volume 11, Issue 12

    Abstract: Fernando Aiuti (Figure 1), born in Urbino on 8 June 1935, suddenly died on 9 January 2019, leaving ...

    Abstract Fernando Aiuti (Figure 1), born in Urbino on 8 June 1935, suddenly died on 9 January 2019, leaving a great void not only among his family members and those who knew him and appreciated his great humanity and acute intelligence, but in the entire immunological scientific community [...].
    Language English
    Publishing date 2023-11-30
    Publishing country Switzerland
    Document type Editorial
    ZDB-ID 2720867-9
    ISSN 2227-9059
    ISSN 2227-9059
    DOI 10.3390/biomedicines11123191
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  6. Article ; Online: Access to gene therapy for rare diseases when commercialization is not fit for purpose.

    Fox, Thomas / Bueren, Juan / Candotti, Fabio / Fischer, Alain / Aiuti, Alessandro / Lankester, Arjan / Booth, Claire

    Nature medicine

    2023  Volume 29, Issue 3, Page(s) 518–519

    MeSH term(s) Humans ; Rare Diseases/genetics ; Rare Diseases/therapy ; Genetic Therapy
    Language English
    Publishing date 2023-02-13
    Publishing country United States
    Document type Letter
    ZDB-ID 1220066-9
    ISSN 1546-170X ; 1078-8956
    ISSN (online) 1546-170X
    ISSN 1078-8956
    DOI 10.1038/s41591-023-02208-8
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    Zs.MG 39: Show issues

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  7. Article ; Online: Editorial to the Special Issue “Clinical Immunology in Italy, with Special Emphasis to Primary and Acquired Immunodeficiencies

    Alessandro Aiuti / Raffaele D’Amelio / Isabella Quinti / Paolo Rossi

    Biomedicines, Vol 11, Iss 12, p

    A Commemorative Issue in Honor of Prof. Fernando Aiuti”

    2023  Volume 3191

    Abstract: Fernando Aiuti (Figure 1), born in Urbino on 8 June 1935, suddenly died on 9 January 2019, leaving ...

    Abstract Fernando Aiuti (Figure 1), born in Urbino on 8 June 1935, suddenly died on 9 January 2019, leaving a great void not only among his family members and those who knew him and appreciated his great humanity and acute intelligence, but in the entire immunological scientific community [.]
    Keywords n/a ; Biology (General) ; QH301-705.5
    Language English
    Publishing date 2023-11-01T00:00:00Z
    Publisher MDPI AG
    Document type Article ; Online
    Database BASE - Bielefeld Academic Search Engine (life sciences selection)

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  8. Article ; Online: New perspectives in gene therapy for inherited disorders.

    Cicalese, Maria Pia / Aiuti, Alessandro

    Pediatric allergy and immunology : official publication of the European Society of Pediatric Allergy and Immunology

    2020  Volume 31 Suppl 24, Page(s) 5–7

    Abstract: Gene therapy has become promising in many fields of medicine, as a single treatment could allow long-lasting and curative benefits. New medicines based on cell gene correction are expected to occur in upcoming years and will be hopefully part of the ... ...

    Abstract Gene therapy has become promising in many fields of medicine, as a single treatment could allow long-lasting and curative benefits. New medicines based on cell gene correction are expected to occur in upcoming years and will be hopefully part of the therapeutic armamentarium for inherited and acquired diseases. Issues related to the costs of these new therapies and access to care for all patients, and procedures and expertise needed to facilitate their application worldwide require to be addressed, together with long-term safety and efficacy monitoring.
    MeSH term(s) Animals ; CRISPR-Cas Systems ; Gene Editing ; Genetic Diseases, Inborn/genetics ; Genetic Diseases, Inborn/therapy ; Genetic Therapy/methods ; Genetic Therapy/trends ; Genetic Vectors ; Heredodegenerative Disorders, Nervous System/genetics ; Heredodegenerative Disorders, Nervous System/therapy ; Humans ; Immune System Diseases/genetics ; Immune System Diseases/therapy ; Zinc Finger Nucleases/genetics ; Zinc Finger Nucleases/metabolism
    Chemical Substances Zinc Finger Nucleases (EC 3.1.-)
    Language English
    Publishing date 2020-02-11
    Publishing country England
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 1057059-7
    ISSN 1399-3038 ; 0905-6157 ; 0906-5784
    ISSN (online) 1399-3038
    ISSN 0905-6157 ; 0906-5784
    DOI 10.1111/pai.13149
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    Zs.A 3096: Show issues Location:
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  9. Article ; Online: Intrathymic delivery a new route for adenoviral-associated vector gene therapy.

    Silvia, Gregori / Alessandro, Aiuti

    The Journal of allergy and clinical immunology

    2019  Volume 145, Issue 2, Page(s) 499–501

    MeSH term(s) Dependovirus ; Genetic Therapy ; Genetic Vectors ; T-Lymphocytes
    Language English
    Publishing date 2019-12-09
    Publishing country United States
    Document type Editorial ; Comment
    ZDB-ID 121011-7
    ISSN 1097-6825 ; 1085-8725 ; 0091-6749
    ISSN (online) 1097-6825 ; 1085-8725
    ISSN 0091-6749
    DOI 10.1016/j.jaci.2019.11.037
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  10. Article ; Online: Overhydrated hereditary stomatocytosis: A rare cause of familiar persistent macrocytosis due to SLC4A1 variants.

    Marcello, Anna Paola / Visconti, Camilla / Vercellati, Cristina / Zaninoni, Anna / Bianchi, Paola / Barcellini, Wilma / Aiuti, Alessandro / Fermo, Elisa / Ferrua, Francesca

    Pediatric blood & cancer

    2023  , Page(s) e30344

    Language English
    Publishing date 2023-04-14
    Publishing country United States
    Document type Letter
    ZDB-ID 2131448-2
    ISSN 1545-5017 ; 1545-5009
    ISSN (online) 1545-5017
    ISSN 1545-5009
    DOI 10.1002/pbc.30344
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