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Editorial
. 2023 Nov 1;108(11):2880-2882.
doi: 10.3324/haematol.2023.283462.

Gene therapy for congenital marrow failure syndromes - no longer grasping at straws?

Richard A Voit  1 , Seth J Corey  2
Affiliations
Editorial

Gene therapy for congenital marrow failure syndromes - no longer grasping at straws?

Richard A Voit et al. Haematologica. .
No abstract available

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Figures

Figure 1.
Figure 1.
Generation and rescue of primary human hematopoietic stem and progenitor cell model of Diamond-Blackfan anemia. Steps of model generation and gene therapy treatment. 1. Nanostraw delivery of Cas9 mRNA and RPS19 sgRNA ameliorates toxicity associated with other delivery methods. 2. Delivery of a homology-directed repair template with a gene fluorescent protein (GFP) cassette flanked by arms of homology to the RPS19 locus. 3. Integration of a GFP cassette at RPS19 generates trackable clones with RPS19 haploinsufficiency. 4. Delivery of EF1α-RPS19 by lentivirus. 5. RPS19 gene replacement improves erythroid differentiation and reverses many of the transcriptional consequences of RPS19 haploinsufficiency. AAV: adeno-associated virus.
See this image and copyright information in PMC

Comment on

References

    1. Liu Y, Schmiderer L, Hjort M, et al. . Engineered human Diamond-Blackfan anemia disease model confirms therapeutic effects of clinically applicable lentiviral vector at single-cell resolution. Haematologica 2023;108(11):3095-3109. - PMC - PubMed
    1. Kanter J, Walters MC, Krishnamurti L, et al. . Biologic and clinical efficacy of lentiglobin for sickle cell disease. N Engl J Med. 2022;386(7):617-628. - PubMed
    1. Esrick EB, Lehmann LE, Biffi A, et al. . Post-transcriptional genetic silencing of BCL11A to treat sickle cell disease. N Engl J Med. 2021;384(3):205-215. - PMC - PubMed
    1. Da Costa L, Leblanc T, Mohandas N. Diamond-Blackfan anemia. Blood. 2020;136(11):1262-1273. - PMC - PubMed
    1. Sieff C. Diamond-Blackfan anemia. In: Adam MP, Mirzaa GM, Pagon RA, et al.., eds. GeneReviews®. (WA): University of Washington, Seattle; 1993.