Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome

Pille, Melissa; Avila, John M.; Park, So Hyun; Le, Cuong Q.; Xue, Haipeng; Haerynck, Filomeen; Saxena, Lavanya; Lee, Ciaran; Shpall, Elizabeth J.; Bao, Gang; Vandekerckhove, Bart; Davis, Brian R.

doi:10.1016/j.omtm.2024.101208

Molecular Therapy - Methods & Clinical Development

2024

DOI: 10.1016/j.omtm.2024.101208

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Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome

Melissa Pille,

John M. Avila,

So Hyun Park

et al.

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2024

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Cited by 2 publications

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It’s all about location: Targeting the right spot for Wiskott-Aldrich syndrome

Naseem,

Cavazza

2024

Molecular Therapy - Methods & Clinical Development

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It’s all about location: Targeting the right spot for Wiskott-Aldrich syndrome

Naseem,

Cavazza

2024

Molecular Therapy - Methods & Clinical Development

View full text Add to dashboard Cite

The potency of hematopoietic stem cell reprogramming for changing immune tone

Daman,

Cheong,

Berneking

et al. 2024

Immunological Reviews

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SummaryInnate immune memory endows innate immune cells with antigen independent heightened responsiveness to subsequent challenges. The durability of this response can be mediated by inflammation induced epigenetic and metabolic reprogramming in hematopoietic stem and progenitor cells (HSPCs) that are maintained through differentiation to mature immune progeny. Understanding the mechanisms and extent of trained immunity induction by pathogens and vaccines, such as BCG, in HSPC remains a critical area of exploration with important implications for health and disease. Here we review these concepts and present new analysis to highlight how inflammatory reprogramming of HSPC can potently alter immune tone, including to enhance specific anti‐tumor responses. New findings in the field pave the way for novel HSPC targeting therapeutic strategies in cancer and other contexts of immune modulation. Future studies are expected to unravel diverse and extensive effects of infections, vaccines, microbiota, and sterile inflammation on hematopoietic progenitor cells and begin to illuminate the broad spectrum of immunologic tuning that can be established through altering HSPC phenotypes. The purpose of this review is to draw attention to emerging and speculative topics in this field where we posit that focused study of HSPC in the framework of trained immunity holds significant promise.

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Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome

Cited by 2 publications

References 39 publications

It’s all about location: Targeting the right spot for Wiskott-Aldrich syndrome

It’s all about location: Targeting the right spot for Wiskott-Aldrich syndrome

The potency of hematopoietic stem cell reprogramming for changing immune tone

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