Search Results

Targeting the metabolic pathway of human colon cancer overcomes resistance to TRAIL-induced apoptosis.

Colon cancer is a leading cause of cancer-related mortality for which targeted therapy is needed; however, trials using apoptosis-inducing ligand monotherapy to overcome resistance to apoptosis have not shown clinical responses. Since colon cancer...

Authors: Carr RM, Qiao G, Qin J, Jayaraman S, Prabhakar BS, Maker AV

Improved Expansion and In Vivo Function of Patient T Cells by a Serum-free Medium.

Improvements to T cell culture systems that promote long-term engraftment and function of adoptively transferred T cells will likely result in superior clinical benefit to more individuals. To this end, we recently developed a chemically...

Authors: Medvec AR, Ecker C, Kong H, Winters EA, Glover J, Varela-Rohena A, Riley JL.

SST Peer Reviewed

Reprogramming triggers endogenous L1 and Alu retrotransposition in human induced pluripotent stem cells.

Human induced pluripotent stem cells (hiPSCs) are capable of unlimited proliferation and can differentiate in vitro to generate derivatives of the three primary germ layers. Genetic and epigenetic abnormalities have been reported by Wissing and...

Authors: Klawitter S, Fuchs NV, Upton KR, Muñoz-Lopez M, Shukla R, Wang J, Garcia-Cañadas M, Lopez-Ruiz C,...

SST Peer Reviewed

Generation of inner ear organoids containing functional hair cells from human pluripotent stem cells.

The derivation of human inner ear tissue from pluripotent stem cells would enable in vitro screening of drug candidates for the treatment of hearing and balance dysfunction and may provide a source of cells for cell-based therapies of the inner ear....

Authors: Koehler KR, Nie J1, Longworth-Mills E, Liu XP, Lee J, Holt JR, Hashino E.

SST Peer Reviewed

Reprogramming human T cell function and specificity with non-viral genome targeting.

Decades of work have aimed to genetically reprogram T cells for therapeutic purposes1,2 using recombinant viral vectors, which do not target transgenes to specific genomic sites3,4. The need for viral vectors has slowed down research and clinical use...

Authors: Roth TL, Puig-Saus C, Yu R, Shifrut E, Carnevale J, Li PJ, Hiatt J, Saco J, Krystofinski P, Li H,...

SST Peer Reviewed

Generation of a human induced pluripotent stem cell line via CRISPR-Cas9 mediated integration of a site-specific homozygous mutation in CHMP2B.

Frontotemporal dementia (FTD) is an early onset neurodegenerative disease. Mutations in several genes cause familial FTD and one of them is charged multivesicular body protein 2B (CHMP2B) on chromosome 3 (FTD3), a component of the endosomal sorting...

Authors: Zhang Y, Schmid B, Nielsen TT, Nielsen JE, Clausen C, Hyttel P, Holst B, Freude KK.

SST Peer Reviewed

DNA methylation is extensively remodeled during mammalian gametogenesis and embryogenesis. Most transposons become hypomethylated, raising the question of their regulation in the absence of DNA methylation. To reproduce a rapid and extensive demethylation, we subjected mouse ES cells to chemically defined hypomethylating culture conditions. Surprisingly, we observed two phases of transposon regulation. After an initial burst of de-repression, various transposon families were efficiently re-silenced. This wa

DNA methylation is extensively remodeled during mammalian gametogenesis and embryogenesis. Most transposons become hypomethylated, raising the question of their regulation in the absence of DNA methylation. To reproduce a rapid and extensive...

Authors: Walter M, Teissandier A, Pérez-Palacios R, Bourc\\\'his D.

Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors

The Sleeping Beauty (SB) transposon system is a non-viral gene delivery platform that combines simplicity, inexpensive manufacture, and favorable safety features in the context of human applications. However, efficient correction of hematopoietic...

Authors: Marta Holstein, Cristina Mesa-Nuñez, Csaba Miskey, Elena Almarza, Valentina Poletti, Marco Schmeer,...

SST Peer Reviewed

Highly efficient Cas9-mediated transcriptional programming.

The RNA-guided nuclease Cas9 can be reengineered as a programmable transcription factor. However, modest levels of gene activation have limited potential applications. We describe an improved transcriptional regulator obtained through the rational...

Authors: Chavez A1, Scheiman J2, Vora S3, Pruitt BW4, Tuttle M4, P R Iyer E2, Lin S5, Kiani S6, Guzman CD4,...

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

Translation of the CRISPR-Cas9 system to human therapeutics holds high promise. However, specificity remains a concern especially when modifying stem cell populations. We show that existing rationally engineered Cas9 high-fidelity variants have...

Authors: Vakulskas CA, Dever DP, Rettig GR, Turk R, Jacobi AM, Collingwood MA, Bode NM, McNeill MS, Yan S,...

SST Peer Reviewed

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Targeting the metabolic pathway of human colon cancer overcomes resistance to TRAIL-induced apoptosis.

Improved Expansion and In Vivo Function of Patient T Cells by a Serum-free Medium.

Reprogramming triggers endogenous L1 and Alu retrotransposition in human induced pluripotent stem cells.

Generation of inner ear organoids containing functional hair cells from human pluripotent stem cells.

Reprogramming human T cell function and specificity with non-viral genome targeting.

Generation of a human induced pluripotent stem cell line via CRISPR-Cas9 mediated integration of a site-specific homozygous mutation in CHMP2B.

Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors

Highly efficient Cas9-mediated transcriptional programming.

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells