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Engineering designer beta cells with a CRISPR-Cas9 conjugation platform

Genetically fusing protein domains to Cas9 has yielded several transformative technologies; however, the genetic modifications are limited to natural polypeptide chains at the Cas9 termini, which excludes a diverse array of molecules...

Authors: Lim D, Sreekanth V, Cox KJ, Law BK, Wagner BK, Karp JM

SST Peer Reviewed

An improved SNAP-ADAR tool enables efficient RNA base editing to interfere with post-translational protein modification

RNA base editing relies on the introduction of adenosine-to-inosine changes into target RNAs in a highly programmable manner in order to repair disease-causing mutations. Here, we propose that RNA base editing could be broadly applied to perturb...

Authors: Karthika Devi Kiran Kumar , Shubhangi Singh , Stella Maria Schmelzle , Paul Vogel , Carolin...

Virus-free CRISPR knockin of a chimeric antigen receptor into KLRC1 generates potent GD2-specific natural killer cells

Natural killer (NK) cells are an appealing off-the-shelf, allogeneic cellular therapy due to their cytotoxic profile. However, their activity against solid tumors remains suboptimal in part due to the upregulation of NK-inhibitory ligands, such as...

Authors: Shankar K, Zingler-Hoslet I, Tabima DM, Zima S, Shi L, Gimse K, Forsberg MH, Katta V, Davis SZ,...

SST Peer Reviewed

An hepatitis B and D virus infection model using human pluripotent stem cell-derived hepatocytes

Current culture systems available for studying hepatitis D virus (HDV) are suboptimal. In this study, we demonstrate that hepatocyte-like cells (HLCs) derived from human pluripotent stem cells (hPSCs) are fully permissive to HDV infection across...

Authors: Huanting Chi, Bingqian Qu, Angga Prawira, Talisa Richardt, Lars Maurer, Jungen Hu, Rebecca M Fu...

An efficient, non-viral arrayed CRISPR screening platform for iPSC-derived myeloid and microglia models

Here, we developed a CRISPR-Cas9 arrayed screen to investigate lipid handling pathways in human induced pluripotent stem cell (iPSC)- derived microglia.We established a robust method for the nucleofection of CRISPR-Cas9 ribonucleoprotein...

Authors: Meier S, Larsen ASG, Wanke F, Mercado N, Mei A, Takacs L, Mracsko ES, Collin L, Kampmann M,...

SST Peer Reviewed

BH3 mimetics augment cytotoxic T cell killing of acute myeloid leukemia via mitochondrial apoptotic mechanism

Adoptive cell therapy (ACT) can address an unmet clinical need for patients with relapsed/refractory acute myeloid leukemia (AML), but its effect is often modest in the setting of high tumor burden. In this study, we postulated that strategies...

Authors: Saxena K, Ryu E, Hung SH, Singh S, Zhang Q, Zeng Z, Wang Z, Konopleva M, Yee C

SST Peer Reviewed

A Drug Screen using Human iPSC-Derived Hepatocyte-like Cells Reveals Cardiac Glycosides as a Potential Treatment for Hypercholesterolemia

Efforts to identify pharmaceuticals to treat heritable metabolic liver diseases have been hampered by the lack of models. However, cells with hepatocyte characteristics can be produced from induced pluripotent stem cells (iPSCs). Here, we have used...

Authors: Max A Cayo , Sunil K Mallanna, Francesca Di Furio , Ran Jing , Lauren B Tolliver , Matthew...

An engineered human cardiac tissue model reveals contributions of systemic lupus erythematosus autoantibodies to myocardial injury

Systemic lupus erythematosus (SLE) is a heterogenous autoimmune disease that affects multiple organs, including the heart. The mechanisms of myocardial injury in SLE remain poorly understood. In this study, we engineered human cardiac tissues and...

Authors: Sharon Fleischer, Trevor R. Nash, Manuel A. Tamargo, Roberta I. Lock, Gabriela Venturini, Margaretha...

Modeling of Blood–Brain Barrier (BBB) Dysfunction and Immune Cell Migration Using Human BBB-on-a-Chip for Drug Discovery Research

Blood–brain barrier (BBB) dysfunction is a key feature in neuroimmunological and neurodegenerative diseases. In this study, we developed a microfluidic human BBB-on-a-chip to model barrier dysfunction and immune cell migration using immortalized TY10...

Authors: Masato Ohbuchi , Mayu Shibuta , Kazuhiro Tetsuka , Haruna Sasaki-Iwaoka , Masayo Oishi , Fumitaka...

EFFICIENT COST-EFFECTIVE MANUFACTURE OF A NON-VIRAL TRANSPOSON BASED NOVEL BAFF-CART FOR TREATMENT OF B-CELL CANCERS

CD19 CART cell therapies have shown remarkable efficacy in non-Hodgkin lymphoma. However, durable response has been observed in less than half of treated patients due to epitope loss and T cell exhaustion. To overcome this limitation, we developed a...

Authors: A.Lawrence, S.Kleinsorge-Block, L.Jonart, P.Caimi, R.Parameswaran, J.Reese

SST Peer Reviewed

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Engineering designer beta cells with a CRISPR-Cas9 conjugation platform

An improved SNAP-ADAR tool enables efficient RNA base editing to interfere with post-translational protein modification

Virus-free CRISPR knockin of a chimeric antigen receptor into KLRC1 generates potent GD2-specific natural killer cells

An hepatitis B and D virus infection model using human pluripotent stem cell-derived hepatocytes

An efficient, non-viral arrayed CRISPR screening platform for iPSC-derived myeloid and microglia models

BH3 mimetics augment cytotoxic T cell killing of acute myeloid leukemia via mitochondrial apoptotic mechanism

A Drug Screen using Human iPSC-Derived Hepatocyte-like Cells Reveals Cardiac Glycosides as a Potential Treatment for Hypercholesterolemia

An engineered human cardiac tissue model reveals contributions of systemic lupus erythematosus autoantibodies to myocardial injury

Modeling of Blood–Brain Barrier (BBB) Dysfunction and Immune Cell Migration Using Human BBB-on-a-Chip for Drug Discovery Research

EFFICIENT COST-EFFECTIVE MANUFACTURE OF A NON-VIRAL TRANSPOSON BASED NOVEL BAFF-CART FOR TREATMENT OF B-CELL CANCERS