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6404 results sorted by

Efficient CRISPR/Cas9-Mediated Gene Knockin in Mouse Hematopoietic Stem and Progenitor Cells

Mutations accumulating in hematopoietic stem and progenitor cells (HSPCs) during development can cause severe hematological disorders. Modeling these mutations in mice is essential for understanding their functional consequences. Here, we describe an...
Authors: Ngoc Tung Tran , Thomas Sommermann , Robin Graf , Janine Trombke , Jenniffer Pempe , Kerstin Petsch...
SST Peer Reviewed

Hepatitis Delta Virus Acts as an Immunogenic Adjuvant in Hepatitis B Virus-Infected Hepatocytes

Hepatitis delta virus (HDV) requires hepatitis B virus (HBV) to complete its infection cycle and causes severe hepatitis, with limited therapeutic options. To determine the prospect of T cell therapy in HBV/HDV co-infection, we study the impact of...
Authors: Christine Y.L. Tham, Janine Kah, Anthony T. Tan, ..., Marc L€utgehetmann, Maura Dandri, Antonio...

A time-resolved, multi-symbol molecular recorder via sequential genome editing

DNA is naturally well suited to serve as a digital medium for in vivo molecular recording. However, contemporary DNA-based memory devices are constrained in terms of the number of distinct 'symbols' that can be concurrently recorded and/or by a...
Authors: Choi J, Chen W, Minkina A, Chardon FM, Suiter CC, Regalado SG, Domcke S, Hamazaki N, Lee C, Martin...

Modulation of hepatitis B virus infection by epidermal growth factor secreted from liver sinusoidal endothelial cells

Hepatocytes derived from human iPSCs are useful to study hepatitis B virus (HBV) infection, however infection efficiency is rather poor. In order to improve the efficiency of HBV infection to iPSC-derived hepatocytes, we set a co-culture of...
Authors: Shin-Wei C hen, Misao Himeno, Yuta Koui, Masaya Sugiyama, Hironori Nishitsuji, Masashi Mizokami,...

CRISPR-Mediated Non-Viral Site-Specific Gene Integration and Expression in T Cells: Protocol and Application for T-Cell Therapy

T cells engineered with chimeric antigen receptors (CARs) show great promise in the treatment of some cancers. Modifying T cells to express CARs generally relies on T-cell transduction using viral vectors carrying a transgene, resulting in...
Authors: Zelda Odé , Jose Condori , Nicolas Peterson , Sheng Zhou , Giedre Krenciute 
SST Peer Reviewed

Abrogation of HLA surface expression using CRISPR/Cas9 genome editing: a step toward universal T cell therapy

As recent advancements in the chimeric antigen receptor-T cells have revolutionized the way blood cancers are handled, potential benefits from producing off-the-shelf, standardized immune cells entail the need for development of allogeneic immune...
Authors: Lee J, Sheen JH, Lim O, Lee Y, Ryu J, Shin D, Kim YY, Kim M.

Pharmacological interventions enhance virus-free generation of TRAC-replaced CAR T cells

Chimeric antigen receptor (CAR) redirected T cells are potent therapeutic options against hematological malignancies. The current dominant manufacturing approach for CAR T cells depends on retroviral transduction. With the advent of gene...
Authors: Kath J, Du W, Pruene A, Braun T, Thommandru B, Turk R, Sturgeon ML, Kurgan GL, Amini L, Stein M,...

Isogenic cell models of cystic fibrosis-causing variants in natively expressing pulmonary epithelial cells.

Background: Assessment of approved drugs and developmental drug candidates for rare cystic fibrosis (CF)-causing variants of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) requires abundant material from relevant models.Methods:...
Authors: Valley HC, Bukis KM, Bell A, Cheng Y, Wong E, Jordan NJ, Allaire NE, Sivachenko A, Liang F, Bihler...
SST Peer Reviewed

NFAM1 Promotes Pro-Inflammatory Cytokine Production in Mouse and Human Monocytes

NFAT activating protein with ITAM motif 1 (NFAM1) is an ITAM bearing-transmembrane receptor that has been reported to play a role in B cell signaling and development. We performed expression analysis of NFAM1 using publicly available gene expression...
Authors: Juchem KW, Gounder AP, Gao JP, Seccareccia E, Yeddula N, Huffmaster NJ, Côté-Martin A, Fogal SE,...

CRISPR-Cas9 based gene editing of the immune checkpoint NKG2A enhances NK cell mediated cytotoxicity against multiple myeloma

Natural Killer (NK) cells are known for their high intrinsic cytotoxic capacity, and the possibility to be applied as ‘off-the-shelf’ product makes them highly attractive for cell-based immunotherapies. In patients with multiple myeloma (MM), an...
Authors: Bexte T, Alzubi J, Reindl LM, Wendel P, Schubert R, Salzmann-Manrique E, von Metzler I, Cathomen T,...
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