Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Editorial
. 2023 Sep 12;7(10):e953.
doi: 10.1097/HS9.0000000000000953. eCollection 2023 Oct.

Current and Future Perspective in Hematopoietic Stem Progenitor Cell-gene Therapy for Inborn Errors of Metabolism

Francesca Tucci  1   2 , Giulia Consiglieri  1   2   3 , Matilde Cossutta  1   4 , Maria Ester Bernardo  1   2   5
Affiliations
Editorial

Current and Future Perspective in Hematopoietic Stem Progenitor Cell-gene Therapy for Inborn Errors of Metabolism

Francesca Tucci et al. Hemasphere. .
No abstract available

PubMed Disclaimer

Conflict of interest statement

The authors have no conflicts of interest to disclose.

Figures

Figure 1.
Figure 1.
HSPC-GT for IEM. Autologous HSPC are collected from the patient through bone marrow harvest or peripheral blood stem cell mobilization, and CD34+ cells are immunomagnetically selected and cultivated with the therapeutic vector. After the missing gene has been transferred to the HSPC, transduced cells are usually cryopreserved to wait for quality control release. Thereafter, the cells are thawed and reinfused into the patient after a chemotherapy-based preparative regimen. Once engrafted, the genetically modified HSPC will give rise to a corrected progeny, which will circulate and deliver the missing enzyme to different tissues and organs via the cross-correction mechanism. GT = gene therapy; HSPC = hematopoietic stem cells; IEM = inborn errors of metabolism.
See this image and copyright information in PMC

References

    1. Ezgu F. Inborn errors of metabolism. Adv Clin Chem. 2016;73:195–250. - PubMed
    1. Boustany RM. Lysosomal storage diseases--the horizon expands. Nat Rev Neurol. 2013;9:583–598. - PubMed
    1. Platt FM, Jeyakumar M. Substrate reduction therapy. Acta Paediatr. 2008;97:88–93. - PubMed
    1. Fernandez-Pereira C, San Millán-Tejado B, Gallardo-Gómez M, et al. . Therapeutic approaches in lysosomal storage diseases. Biomolecules. 2021;11:1775. - PMC - PubMed
    1. Broomfield A, Jones SA, Hughes SM, et al. . The impact of the immune system on the safety and efficiency of enzyme replacement therapy in lysosomal storage disorders. J Inherit Metab Dis. 2016;39:499–512. - PubMed

Publication types

LinkOut - more resources