Alternative splicing of its 5'-UTR limits CD20 mRNA translation and enables resistance to CD20-directed immunotherapies.


Journal

Blood
ISSN: 1528-0020
Titre abrégé: Blood
Pays: United States
ID NLM: 7603509

Informations de publication

Date de publication:
16 Nov 2023
Historique:
accepted: 19 08 2023
received: 08 03 2023
medline: 17 11 2023
pubmed: 8 9 2023
entrez: 8 9 2023
Statut: ppublish

Résumé

Aberrant skipping of coding exons in CD19 and CD22 compromises the response to immunotherapy in B-cell malignancies. Here, we showed that the MS4A1 gene encoding human CD20 also produces several messenger RNA (mRNA) isoforms with distinct 5' untranslated regions. Four variants (V1-4) were detected using RNA sequencing (RNA-seq) at distinct stages of normal B-cell differentiation and B-lymphoid malignancies, with V1 and V3 being the most abundant. During B-cell activation and Epstein-Barr virus infection, redirection of splicing from V1 to V3 coincided with increased CD20 positivity. Similarly, in diffuse large B-cell lymphoma, only V3, but not V1, correlated with CD20 protein levels, suggesting that V1 might be translation-deficient. Indeed, the longer V1 isoform contained upstream open reading frames and a stem-loop structure, which cooperatively inhibited polysome recruitment. By modulating CD20 isoforms with splice-switching morpholino oligomers, we enhanced CD20 expression and anti-CD20 antibody rituximab-mediated cytotoxicity in a panel of B-cell lines. Furthermore, reconstitution of CD20-knockout cells with V3 mRNA led to the recovery of CD20 positivity, whereas V1-reconstituted cells had undetectable levels of CD20 protein. Surprisingly, in vitro CD20-directed chimeric antigen receptor T cells were able to kill both V3- and V1-expressing cells, but the bispecific T-cell engager mosunetuzumab was only effective against V3-expressing cells. To determine whether CD20 splicing is involved in immunotherapy resistance, we performed RNA-seq on 4 postmosunetuzumab follicular lymphoma relapses and discovered that in 2 of them, the downregulation of CD20 was accompanied by a V3-to-V1 shift. Thus, splicing-mediated mechanisms of epitope loss extend to CD20-directed immunotherapies.

Identifiants

pubmed: 37683180
pii: 497824
doi: 10.1182/blood.2023020400
pmc: PMC10667349
doi:

Substances chimiques

RNA, Messenger 0
5' Untranslated Regions 0
Antigens, CD20 0
Protein Isoforms 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

1724-1739

Subventions

Organisme : NCI NIH HHS
ID : U01 CA232563
Pays : United States
Organisme : NCI NIH HHS
ID : P30 CA010815
Pays : United States
Organisme : NCI NIH HHS
ID : U01 CA243072
Pays : United States
Organisme : NCI NIH HHS
ID : T32 CA009615
Pays : United States
Organisme : NCI NIH HHS
ID : U01 CA232486
Pays : United States

Commentaires et corrections

Type : UpdateOf
Type : CommentIn

Informations de copyright

© 2023 by The American Society of Hematology. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.

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Auteurs

Zhiwei Ang (Z)

Division of Cancer Pathobiology, Children's Hospital of Philadelphia, Philadelphia, PA.

Luca Paruzzo (L)

Center for Cellular Immunotherapies, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.
Lymphoma Program, Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA.
Division of Hematology/Oncology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.

Katharina E Hayer (KE)

Department of Biomedical and Health Informatics, Children's Hospital of Philadelphia, Philadelphia, PA.

Carolin Schmidt (C)

Division of Cancer Pathobiology, Children's Hospital of Philadelphia, Philadelphia, PA.

Manuel Torres Diz (M)

Division of Cancer Pathobiology, Children's Hospital of Philadelphia, Philadelphia, PA.

Feng Xu (F)

Division of Genomic Diagnostic, Children's Hospital of Philadelphia, Philadelphia, PA.

Urvi Zankharia (U)

Gene Expression and Regulation Program, The Wistar Institute, Philadelphia, PA.

Yunlin Zhang (Y)

Center for Cellular Immunotherapies, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.

Samantha Soldan (S)

Gene Expression and Regulation Program, The Wistar Institute, Philadelphia, PA.

Sisi Zheng (S)

Division of Cancer Pathobiology, Children's Hospital of Philadelphia, Philadelphia, PA.

Catherine D Falkenstein (CD)

Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA.

Joseph P Loftus (JP)

Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA.

Scarlett Y Yang (SY)

Division of Cancer Pathobiology, Children's Hospital of Philadelphia, Philadelphia, PA.

Mukta Asnani (M)

Division of Cancer Pathobiology, Children's Hospital of Philadelphia, Philadelphia, PA.

Patricia King Sainos (P)

Department of Biology, University of Pennsylvania, Philadelphia, PA.

Vinodh Pillai (V)

Division of Hematopathology, Children's Hospital of Philadelphia, Philadelphia, PA.
Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, PA.
Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.

Emeline Chong (E)

Lymphoma Program, Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA.

Marilyn M Li (MM)

Division of Genomic Diagnostic, Children's Hospital of Philadelphia, Philadelphia, PA.
Division of Hematopathology, Children's Hospital of Philadelphia, Philadelphia, PA.
Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.

Sarah K Tasian (SK)

Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA.
Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, PA.
Department of Pediatrics, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.

Yoseph Barash (Y)

Department of Genetics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.

Paul M Lieberman (PM)

Gene Expression and Regulation Program, The Wistar Institute, Philadelphia, PA.

Marco Ruella (M)

Center for Cellular Immunotherapies, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.
Lymphoma Program, Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA.
Division of Hematology/Oncology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.

Stephen J Schuster (SJ)

Lymphoma Program, Abramson Cancer Center, University of Pennsylvania, Philadelphia, PA.
Division of Hematology/Oncology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.

Andrei Thomas-Tikhonenko (A)

Division of Cancer Pathobiology, Children's Hospital of Philadelphia, Philadelphia, PA.
Division of Oncology, Children's Hospital of Philadelphia, Philadelphia, PA.
Center for Childhood Cancer Research, Children's Hospital of Philadelphia, Philadelphia, PA.
Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.
Department of Pediatrics, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA.

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