Article

Autologous genome-edited hematopoietic stem cells correct Gaucher disease and establish a platform for clinical translation

2025-08-18

Abstract excerpt

<title>Abstract</title> <p>Gaucher disease type 1 is a lysosomal storage disorder caused by <italic>GBA1</italic> mutations that reduce glucocerebrosidase activity, leading to glycolipid buildup, particularly in macrophages. To develop a curative approach, we established a high-efficiency genome editing platform for human and murine hematopoietic stem-progenitor cells using CRISPR/Cas9, recombinant adeno-associat...

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Literature Corpus work: d421e7fa-158e-5eb6-808c-0db24efd0a06
DOI: 10.21203/rs.3.rs-7123212/v1

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