Work

Progress toward the challenging goal of HDR-based gene editing for hyper-IgM syndrome

Year: 2024

Type: article

Abstract: Allogeneic hematopoietic stem cell (HSC) transplantation from healthy donors has long been the standard therapy for treating monogenic inherited immunodeficiencies and blood cell disorders, such as Wiskott-Aldrich syndrome, Fanconi anemia, severe combined immunodeficiency, chronic granulomatous disease, and hyper-immunoglobulin M (hyper-IgM) syndromes.1 However, this approach is limited by the availability of donors and the risk of clinical complications, including infections, graft-versus-host disease, and engraftment failure.

Source: Molecular Therapy — Methods & Clinical Development

DOI: 10.1016/j.omtm.2024.101359

Authors: Luiz Fernando Bazzo Catto, Cynthia E. Dunbar

Institutions: National Institutes of Health, National Heart, Lung, and Blood Institute

Language: English

Topic: Immunodeficiency and Autoimmune Disorders

Subfield: Immunology

Field: Immunology and Microbiology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,900