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Highly efficient induced pluripotent stem cell reprogramming of cryopreserved lymphoblastoid cell lines

Tissue culture based in-vitro experimental modeling of human inherited disorders provides insight into the cellular and molecular mechanisms involved and the underlying genetic component influencing the disease phenotype. The breakthrough development...

Authors: Satish Kumar , Joanne E Curran , Erika C Espinosa , David C Glahn , John Blangero

SST Peer Reviewed

High-efficiency nonviral CRISPR/Cas9-mediated gene editing of human T cells using plasmid donor DNA

Genome engineering of T lymphocytes, the main effectors of antitumor adaptive immune responses, has the potential to uncover unique insights into their functions and enable the development of next-generation adoptive T cell therapies. Viral gene...

Authors: Oh SA, Senger K, Madireddi S, Akhmetzyanova I, Ishizuka IE, Tarighat S, Lo JH, Shaw D, Haley B, Rutz...

SST Peer Reviewed

Functional Profiling of a Plasmodium Genome Reveals an Abundance of Essential Genes

The genomes of malaria parasites contain many genes of unknown function. To assist drug development through the identification of essential genes and pathways, we have measured competitive growth rates in mice of 2,578 barcoded Plasmodium berghei...

Authors: Bushell E, Gomes AR, Sanderson T, Anar B, Girling G, Herd C, Metcalf T, Modrzynska K, Schwach F,...

Human Pluripotent Stem Cell-Derived Organoids as Models of Liver Disease

We cultured H1 human embryonic stem cells (WA-01, passage 27-40) and induced pluripotent stem cells (GM23338) with a series of chemically defined and serum-free media to induce formation of posterior foregut cells, which were differentiated in...

Authors: Bin Ramli MN, Lim YS, Koe CT, Demircioglu D, Tng W, Uy Gonzales KA, Tan CP, Szczerbinska I, Liang H,...

SST Peer Reviewed

Efficient generation of isogenic primary human myeloid cells using CRISPR-Cas9 ribonucleoproteins

Genome engineering of primary human cells with CRISPR-Cas9 has revolutionized experimental and therapeutic approaches to cell biology, but human myeloid-lineage cells have remained largely genetically intrac table. We present a method for the...

Authors: Hiatt J, Cavero DA, McGregor MJ, Zheng W, Budzik JM, Roth TL, Haas KM, Wu D, Rathore U, Meyer-Franke...

SST Peer Reviewed

Assembly and Function of a Bioengineered Human Liver for Transplantation Generated Solely from Induced Pluripotent Stem Cells

The availability of an autologous transplantable auxiliary liver would dramatically affect the treatment of liver disease. Assembly and function in vivo of a bioengineered human liver derived from induced pluripotent stem cells (iPSCs) has not been...

Authors: Kazuki Takeishi, Alexandra Collin de l’Hortet, Yang Wang, Kan Handa, Jorge Guzman-Lepe, Kentaro...

SST Peer Reviewed

Electroporation-mediated Delivery of Cas9Ribonucleoproteins Results in High Levels of Gene Editing in Primary Hepatocytes

Adeno-associated virus vectors are the most used delivery method for liver-directed gene editing. Still, they are associated with significant disadvantages that can compromise the safety and efficacy of therapies. Here, we investigate the effects of...

Authors: Tanner Rathbone , Ilayda Ates , Lawrence Fernando , Ethan Addlestone , Ciaran M Lee , Vincent P...

TALEN mediated gene editing in a mouse model of Fanconi anemia

The promising ability to genetically modify hematopoietic stem and progenitor cells by precise gene editing remains challenging due to their sensitivity to in vitro manipulations and poor efficiencies of homologous recombination. This study...

Authors: Maria José Pino-Barrio , Yari Giménez, Mariela Villanueva , Marcus Hildenbeutel , Rebeca...

SST Peer Reviewed

dCas9-based gene editing for cleavage-free genomic knock-in of long sequences

Gene editing is a powerful tool for genome and cell engineering. Exemplified by CRISPR–Cas, gene editing could cause DNA damage and trigger DNA repair processes that are often error-prone. Such unwanted mutations and safety concerns can be...

Authors: Wang C, Qu Y, Cheng JKW, Hughes NW, Zhang Q, Wang M, Cong L.

Induced expression of CCL19 promotes the anti-tumor ability of CAR-T cells by increasing their infiltration ability

Background: Chimeric antigen receptor-engineered T cell (CAR-T) therapy has shown promising potential for anti-cancer treatment. However, for pancreatic ductal adenocarcinoma (PDAC), the lack of infiltrative ability of these CAR-T cells leads to...

Authors: Jian-Fei Hu , Zu-Wei Wang , Cheng-Yu Liao , Zhi-Wen Chen , Feng-Ping Kang , Cai-Feng Lin ,...

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Highly efficient induced pluripotent stem cell reprogramming of cryopreserved lymphoblastoid cell lines

High-efficiency nonviral CRISPR/Cas9-mediated gene editing of human T cells using plasmid donor DNA

Functional Profiling of a Plasmodium Genome Reveals an Abundance of Essential Genes

Human Pluripotent Stem Cell-Derived Organoids as Models of Liver Disease

Efficient generation of isogenic primary human myeloid cells using CRISPR-Cas9 ribonucleoproteins

Assembly and Function of a Bioengineered Human Liver for Transplantation Generated Solely from Induced Pluripotent Stem Cells

Electroporation-mediated Delivery of Cas9Ribonucleoproteins Results in High Levels of Gene Editing in Primary Hepatocytes

TALEN mediated gene editing in a mouse model of Fanconi anemia

dCas9-based gene editing for cleavage-free genomic knock-in of long sequences

Induced expression of CCL19 promotes the anti-tumor ability of CAR-T cells by increasing their infiltration ability