{"database":"GEO","file_versions":[{"headers":{"Content-Type":["application/json"]},"body":{"files":{"Other":["ftp://ftp.ncbi.nlm.nih.gov/geo/series/GSE317nnn/GSE317101/"]},"type":"primary"},"statusCode":"OK","statusCodeValue":200}],"scores":null,"additional":{"omics_type":["Transcriptomics"],"species":["Homo sapiens"],"gds_type":[" Other","Expression profiling by high throughput sequencing"],"full_dataset_link":["https://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE317101"],"repository":["GEO"],"entry_type":["GSE"],"additional_accession":[]},"is_claimable":false,"name":"A hematopoietic stem cell shield for AML-directed immunotherapy","description":"One major challenge in the development of successful chimeric antigen receptor (CAR) T cells for patients with acute myeloid leukemia (AML) has been that most well-studied AML-associated antigens are also expressed on vital normal hematopoietic stem cells (HSCs) or mature myeloid cells. This challenge in optimal antigen selection leads to deleterious on-target toxicity, particularly in the context of CAR T cell persistence. CAR T cells targeting CD371, a type II transmembrane glycoprotein, have shown potent anti-leukemic activity in phase I trials to date; however, CD371 is wildly expressed throughout the normal myeloid compartment. Here we identify that CD371 is dispensable for normal human HSC and myeloid cell function, map the epitope of a CD371 CAR T cell currently in phase I trials, develop a base-editing strategy to shield normal hematopoiesis from CD371-directed immunotherapy, and describe an off-the-shelf approach to target AML while sparing normal hematopoiesis through the use of CD371 CAR T cells.","dates":{"publication":"2026/04/08"},"accession":"GSE317101","cross_references":{"GSM":["GSM9465239","GSM9465238","GSM9465240","GSM9465242","GSM9465241","GSM9465243"],"GPL":["24676"],"GSE":["317101"],"taxon":["Homo sapiens"]}}