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In vivo human T cell engineering with enveloped delivery vehicles

Viruses and virally derived particles have the intrinsic capacity to deliver molecules to cells, but the difficulty of readily altering cell-type selectivity has hindered their use for therapeutic delivery. Here, we show that cell...

Authors: Hamilton JR, Chen E, Perez BS, Sandoval Espinoza CR, Kang MH, Trinidad M, Ngo W, Doudna JA

SST Peer Reviewed

CRISPR/Cas9 editing of NKG2A improves the efficacy of primary CD33-directed chimeric antigen receptor natural killer cells

Chimeric antigen receptor (CAR)-modified natural killer (NK) cells show antileukemic activity against acute myeloid leukemia (AML) in vivo. However, NK cell-mediated tumor killing is often impaired by the interaction between human leukocyte antigen...

Authors: Bexte T, Albinger N, Al Ajami A, Wendel P, Buchinger L, Gessner A, Alzubi J, Särchen V, Vogler M,...

SST Peer Reviewed

The role of sinusoidal endothelial cells and TIMP1 in the regulation of fibrosis in a novel human liver 3D NASH model

Background: The prevalence of NAFLD is rapidly increasing. NAFLD can progress to NASH, fibrosis, cirrhosis, and HCC, which will soon become the main causes of liver transplantation. To date, no effective drug for NASH has been approved by the...

Authors: van Riet, Sander; Julien, Anais; Atanasov, Andrea Nordling, Åsa; Ingelman-Sundberg, Magnus

Single-stranded HDR templates with truncated Cas12a binding sequences improve knock-in efficiencies in primary human T cells

Non-viral gene editing via CRISPR-Cas12a offers an alternative to Cas9-based methods, providing better targeting of AT-rich regions, simplified guide RNA manufacturing, and high specificity. However, the efficacy of editing outcomes is...

Authors: Ana-Maria Nitulescu (1,2), Weijie Du (1,2,#), Viktor Glaser (1,2), Jonas Kath (1,2), Robert...

SST Peer Reviewed

Genome Engineering of Primary and Pluripotent Stem Cell-Derived Hepatocytes for Modeling Liver Tumor Formation

Genome editing has demonstrated its utility in generating isogenic cell-based disease models, enabling the precise introduction of genetic alterations into wild-type cells to mimic disease phenotypes and explore underlying mechanisms. However, its...

Authors: Lulu Zhang , Xunting Wang , Xuelian Yang , Yijia Chi , Yihang Chu , Yi Zhang , Yufan Gong , Fei...

Universal allogeneic CAR T cells engineered with Sleeping Beauty transposons and CRISPR-CAS9 for cancer immunotherapy

Allogeneic CD19-specific chimeric antigen receptor (CAR) T cells with inactivated donor T cell receptor (TCR) expression can be used as an "off-the-shelf" therapeutic modality for lymphoid malignancies, thus offering an attractive alternative to...

Authors: Tipanee J, Samara-Kuko E, Gevaert T, Chuah MK, Vanden Driessche T

SST Peer Reviewed

Hypoimmunogenic Human Pluripotent Stem Cells as a Powerful Tool for Liver Regenerative Medicine

Induced pluripotent stem cells (iPSC) have huge potential as cell therapy for various diseases, given their potential for unlimited self-renewal and capability to differentiate into a wide range of cell types. Although autologous iPSCs represents the...

Authors: Piera Trionfini , Elena Romano , Marco Varinelli , Lorena Longaretti , Paola Rizzo , Roberta...

Chemical manipulation of an activation/inhibition switch in the nuclear receptor PXR

Nuclear receptors are ligand-activated transcription factors that can often be useful drug targets. Unfortunately, ligand promiscuity leads to two-thirds of receptors remaining clinically untargeted. PXR is a nuclear receptor that can be activated by...

Authors: Efren Garcia-Maldonado , Andrew D Huber , Sergio C Chai , Stanley Nithianantham , Yongtao Li , Jing...

Efficient prime editing in mouse brain, liver and heart with dual AAVs

Realizing the promise of prime editing for the study and treatment of genetic disorders requires efficient methods for delivering prime editors (PEs) in vivo. Here we describe the identification of bottlenecks limiting adeno-associated virus...

Authors: Jessie R. Davis, Samagya Banskota, Jonathan M. Levy, Gregory A. Newby, Xiao Wang, Andrew V....

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...

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In vivo human T cell engineering with enveloped delivery vehicles

CRISPR/Cas9 editing of NKG2A improves the efficacy of primary CD33-directed chimeric antigen receptor natural killer cells

The role of sinusoidal endothelial cells and TIMP1 in the regulation of fibrosis in a novel human liver 3D NASH model

Single-stranded HDR templates with truncated Cas12a binding sequences improve knock-in efficiencies in primary human T cells

Genome Engineering of Primary and Pluripotent Stem Cell-Derived Hepatocytes for Modeling Liver Tumor Formation

Universal allogeneic CAR T cells engineered with Sleeping Beauty transposons and CRISPR-CAS9 for cancer immunotherapy

Hypoimmunogenic Human Pluripotent Stem Cells as a Powerful Tool for Liver Regenerative Medicine

Chemical manipulation of an activation/inhibition switch in the nuclear receptor PXR

Efficient prime editing in mouse brain, liver and heart with dual AAVs

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