Search Results

Hepatocyte-intrinsic SMN deficiency drives metabolic dysfunction and liver steatosis in spinal muscular atrophy

Spinal Muscular Atrophy (SMA) is typically characterized as a motor neuron disease, but extra neuronal phenotypes are present in almost every organ in severely affected patients and animal models. Extra-neuronal phenotypes were previously...

Authors: Damien Meng-Kiat Leow , Yang Kai Ng , Loo Chien Wang , Hiromi Wl Koh , Tianyun Zhao , Zi Jian...

Mapping of mitogen and metabolic sensitivity in organoids defines requirements for human hepatocyte growth

Mechanisms underlying human hepatocyte growth in development and regeneration are incompletely understood. In vitro, human fetal hepatocytes (FH) can be robustly grown as organoids, while adult primary human hepatocyte (PHH) organoids remain...

Authors: Delilah Hendriks , Benedetta Artegiani , Thanasis Margaritis , Iris Zoutendijk , Susana Chuva de...

Mechanisms of 5-HT receptor antagonists in the regulation of fibrosis in a 3D human liver spheroid model

Non-alcoholic steatohepatitis (NASH) is a major health problem leading to liver fibrosis and hepatocellular carcinoma, among other diseases, and for which there is still no approved drug treatment. Previous studies in animal models and in LX-2 cells...

Authors: Sara Redenšek Trampuž, Sander van Riet, Åsa Nordling & Magnus Ingelman-Sundberg

Efficient expansion and CRISPR-Cas9-mediated gene correction of patient-derived hepatocytes for treatment of inherited liver diseases

Cell-based ex vivo gene therapy in solid organs, especially the liver, has proven technically challenging. Here, we report a feasible strategy for the clinical application of hepatocyte therapy. We first generated high-quality autologous hepatocytes...

Authors: Kun Zhang , Ping Wan , Liren Wang , Zhen Wang , Fangzhi Tan , Jie Li , Xiaolong Ma , Jin Cen , Xiang...

Non-viral expression of chimeric antigen receptors with multiplex gene editing in primary T cells

Efficient engineering of T cells to express exogenous tumor-targeting receptors such as chimeric antigen receptors (CARs) or T-cell receptors (TCRs) is a key requirement of effective adoptive cell therapy for cancer. Genome editing technologies, such...

Authors: Cappabianca Dan, Li Jingling, Zheng Yueting, Tran Cac, Kasparek Kassandra, Mendez Pedro, Thu Ricky,...

SST Peer Reviewed

The PP2A-Integrator-CDK9 axis fine-tunes transcription and can be targeted therapeutically in cancer

Gene expression by RNA polymerase II (RNAPII) is tightly controlled by cyclin-dependent kinases (CDKs) at discrete checkpoints during the transcription cycle. The pausing checkpoint following transcription initiation is primarily controlled by CDK9....

Authors: Vervoort SJ, Welsh SA, Devlin JR, Barbieri E, Knight DA, Offley S, Bjelosevic S, Costacurta M,...

SST Peer Reviewed

Engineering of potent CAR NK cells using non-viral Sleeping Beauty transposition from minimalistic DNA vectors

Natural Killer (NK) cells have high intrinsic cytotoxic capacity, and clinical trials have demonstrated their safety and efficacy for adoptive cancer therapy. Expression of chimeric antigen receptors (CARs) enhances NK cell target-specificity, with...

Authors: Bexte T, Botezatu L, Miskey C, Gierschek F, Moter A, Wendel P, Reindl LM, Campe J, Villena-Ossa JF,...

SST Peer Reviewed

Addressing the ADME Challenges of Compound Loss in a PDMS-Based Gut-on-Chip Microphysiological System

Microphysiological systems (MPSs) are promising in vitro technologies for physiologically relevant predictions of the human absorption, distribution, metabolism, and excretion (ADME) properties of drug candidates. However, polydimethylsiloxane...

Authors: Patrick Carius , Ferdinand Anton Weinelt , Chris Cantow , Markus Holstein , Aaron M...

Integration of ?-deficient CARs into the CD3-zeta gene conveys potent cytotoxicity in T and NK cells

Chimeric antigen receptor (CAR)-redirected immune cells hold significant therapeutic potential for oncology, autoimmune diseases, transplant medicine, and infections. All approved CAR-T therapies rely on personalized manufacturing using...

Authors: Kath J, Franke C, Drosdek V, Du W, Glaser V, Fuster-Garcia C, Stein M, Zittel T, Schulenberg S,...

SST Peer Reviewed

Structural surfaceomics reveals an AML-specific conformation of integrin ß2 as a CAR T cellular therapy target

Safely expanding indications for cellular therapies has been challenging given a lack of highly cancer-specific surface markers. Here we explore the hypothesis that tumor cells express cancer-specific surface protein conformations that are invisible...

Authors: Mandal K, Wicaksono G, Yu C, Adams JJ, Hoopmann MR, Temple WC, Izgutdina A, Escobar BP, Gorelik M,...

SST Peer Reviewed

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Hepatocyte-intrinsic SMN deficiency drives metabolic dysfunction and liver steatosis in spinal muscular atrophy

Mapping of mitogen and metabolic sensitivity in organoids defines requirements for human hepatocyte growth

Mechanisms of 5-HT receptor antagonists in the regulation of fibrosis in a 3D human liver spheroid model

Efficient expansion and CRISPR-Cas9-mediated gene correction of patient-derived hepatocytes for treatment of inherited liver diseases

Non-viral expression of chimeric antigen receptors with multiplex gene editing in primary T cells

The PP2A-Integrator-CDK9 axis fine-tunes transcription and can be targeted therapeutically in cancer

Engineering of potent CAR NK cells using non-viral Sleeping Beauty transposition from minimalistic DNA vectors

Addressing the ADME Challenges of Compound Loss in a PDMS-Based Gut-on-Chip Microphysiological System

Integration of ?-deficient CARs into the CD3-zeta gene conveys potent cytotoxicity in T and NK cells

Structural surfaceomics reveals an AML-specific conformation of integrin ß2 as a CAR T cellular therapy target