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  1. Article ; Online: Hemophilia Gene Therapy: The End of the Beginning?

    De Wolf, Dries / Singh, Kshitiz / Chuah, Marinee K / VandenDriessche, Thierry

    Human gene therapy

    2023  Volume 34, Issue 17-18, Page(s) 782–792

    Abstract: Extensive preclinical research over the past 30 years has culminated in the recent regulatory approval of several gene therapy products for hemophilia. Based on the efficacy and safety data in a recently conducted phase III clinical trial, ...

    Abstract Extensive preclinical research over the past 30 years has culminated in the recent regulatory approval of several gene therapy products for hemophilia. Based on the efficacy and safety data in a recently conducted phase III clinical trial,
    MeSH term(s) United States ; Adult ; Child ; Humans ; Hemophilia A/genetics ; Hemophilia A/therapy ; Hemophilia B/genetics ; Hemophilia B/therapy ; DNA, Complementary ; Genetic Therapy
    Chemical Substances Valoctocogene Roxaparvovec ; DNA, Complementary
    Language English
    Publishing date 2023-09-06
    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.2023.112
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

    Zs.A 2988: Show issues Location:
    Je nach Verfügbarkeit (siehe Angabe bei Bestand)
    bis Jg. 1994: Bestellungen von Artikeln über das Online-Bestellformular
    Jg. 1995 - 2021: Lesesall (2.OG)
    ab Jg. 2022: Lesesaal (EG)

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  2. Article ; Online: Gene therapy for hemophilia B using CB 2679d-GT: a novel factor IX variant with higher potency than factor IX Padua.

    Nair, Nisha / De Wolf, Dries / Nguyen, Phuong Anh / Pham, Quang Hong / Samara-Kuko, Ermira / Landau, Jeff / Blouse, Grant E / Chuah, Marinee K / VandenDriessche, Thierry

    Blood

    2021  Volume 137, Issue 21, Page(s) 2902–2906

    Abstract: Sustained expression of therapeutic factor IX (FIX) levels has been achieved after adeno-associated viral (AAV) vector-based gene therapy in patients with hemophilia B. Nevertheless, patients are still at risk of vector dose-limiting toxicity, ... ...

    Abstract Sustained expression of therapeutic factor IX (FIX) levels has been achieved after adeno-associated viral (AAV) vector-based gene therapy in patients with hemophilia B. Nevertheless, patients are still at risk of vector dose-limiting toxicity, particularly liver inflammation, justifying the need for more efficient vectors and a lower dosing regimen. A novel increased potency FIX (designated as CB 2679d-GT), containing 3 amino acid substitutions (R318Y, R338E, T343R), significantly outperformed the R338L-Padua variant after gene therapy. CB 2679d-GT demonstrated a statistically significant approximately threefold improvement in clotting activity when compared with R338L-Padua after AAV-based gene therapy in hemophilic mice. Moreover, CB 2679d-GT gene therapy showed significantly reduced bleeding time (approximately fivefold to eightfold) and total blood loss volume (approximately fourfold) compared with mice treated with the R338L-Padua, thus achieving more rapid and robust hemostatic correction. FIX expression was sustained for at least 20 weeks with both CB 2679d-GT and R338L-Padua whereas immunogenicity was not significantly increased. This is a novel gene therapy study demonstrating the superiority of CB 2679d-GT, highlighting its potential to obtain higher FIX activity levels and superior hemostatic efficacy following AAV-directed gene therapy in hemophilia B patients than what is currently achievable with the R338L-Padua variant.
    MeSH term(s) Amino Acid Substitution ; Animals ; Bleeding Time ; Dependovirus/genetics ; Drug Evaluation, Preclinical ; Factor IX/chemistry ; Factor IX/genetics ; Factor IX/therapeutic use ; Gain of Function Mutation ; Gene Dosage ; Genetic Therapy ; Genetic Vectors/therapeutic use ; Hemophilia B/therapy ; Humans ; Liver/metabolism ; Mice ; Mice, Inbred C57BL ; Recombinant Proteins/therapeutic use
    Chemical Substances Recombinant Proteins ; factor IX-Padua ; Factor IX (9001-28-9)
    Language English
    Publishing date 2021-03-16
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 80069-7
    ISSN 1528-0020 ; 0006-4971
    ISSN (online) 1528-0020
    ISSN 0006-4971
    DOI 10.1182/blood.2020006005
    Database MEDical Literature Analysis and Retrieval System OnLINE

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