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6399 results sorted by

Targeted gene knock-in by homology-directed genome editing using Cas9 ribonucleoprotein and AAV donor delivery.

Realizing the full potential of genome editing requires the development of efficient and broadly applicable methods for delivering programmable nucleases and donor templates for homology-directed repair (HDR). The RNA-guided Cas9 endonuclease can be...
Authors: Gaj T, Staahl BT, Rodrigues GM, Limsirichai P, Ekman FK, Doudna JA, Schaffer DV.
SST Peer Reviewed

Retinal toxicity, in vivo and in vitro, associated with inhibition of nicotinamide phosphoribosyltransferase

Nicotinamide phosphoribosyltransferase (NAMPT) is a pleiotropic protein with intra- and extra-cellular functions as an enzyme, cytokine, growth factor, and hormone. NAMPT is of interest for oncology, because it catalyzes the rate-limiting step in the...
Authors: Zabka TS, Singh J, Dhawan P, Liederer BM, Oeh J, Kauss MA, Xiao Y, Zak M, Lin T, McCray B, La N,...
SST Peer Reviewed

The p53–Mdm2 interaction and the E3 ligase activity of Mdm2/Mdm4 are conserved from lampreys to humans

The extant jawless vertebrates, represented by lampreys and hagfish, are the oldest group of vertebrates and provide an interesting genomic evolutionary pivot point between invertebrates and jawed vertebrates. Through genome analysis of one of these...
Authors: Cynthia R. Coffill, Alison P. Lee, Jia Wei Siau, Sharon M. Chee, Thomas L. Joseph, Yaw Sing Tan,...

Going non-viral: the Sleeping Beauty transposon system breaks on through to the clinical side.

Molecular medicine has entered a high-tech age that provides curative treatments of complex genetic diseases through genetically engineered cellular medicinal products. Their clinical implementation requires the ability to stably integrate genetic...
Authors: Hudecek M1, Izsvák Z2, Johnen S3, Renner M4, Thumann G5, Ivics Z4.
SST Peer Reviewed

Inhibition of SGLT1 abrogates preconditioning-induced cardioprotection against ischemia-reperfusion injury

BACKGROUND: Recently, we reported Na+/glucose co-transporter (SGLT1) expression in mouse and human heart. We speculated that SGLT1 might play an important role in ischemic preconditioning-induced cardioprotection. Therefore, the present study was...
Authors: Kanwal A, Nizami HL, Mallapudi S, Putcha UK, Mohan GK, Banerjee SK
SST Peer Reviewed

Engineering a vascularised 3D in vitro model of cancer progression

The hallmark of tumours is the ability of cancerous cells to promote vascular growth, to disseminate and invade to distant organs. The metastatic process is heavily influenced by the extracellular matrix (ECM) density and composition of the...
Authors: Tarig Magdeldin, Víctor López-Dávila, Judith Pape, Grant W. W. Cameron, Mark Emberton, Marilena...

Immunotherapy of acute leukemia by chimeric antigen receptor-modified lymphocytes using an improved Sleeping Beauty transposon platform.

Chimeric antigen receptor (CAR)-modified T-cell adoptive immunotherapy is a remarkable therapeutic option proven effective in the treatment of hematological malignancies. In order to optimize cell manufacturing, we sought to develop a novel...
Authors: Magnani CF1, Turazzi N1, Benedicenti F2, Calabria A2, Tenderini E2, Tettamanti S1, Giordano...
SST Peer Reviewed

Long-term electromagnetic exposure of developing neuronal networks: A flexible experimental setup

Neuronal networks in vitro are considered one of the most promising targets of research to assess potential electromagnetic field induced effects on neuronal functionality. A few exposure studies revealed there is currently no evidence of any adverse...
Authors: Oster S, Daus AW, Erbes C, Goldhammer M, Bochtler U, Thielemann C
SST Peer Reviewed

Inheritable Silencing of Endogenous Genes by Hit-and-Run Targeted Epigenetic Editing.

Gene silencing is instrumental to interrogate gene function and holds promise for therapeutic applications. Here, we repurpose the endogenous retroviruses' silencing machinery of embryonic stem cells to stably silence three highly expressed genes in...
Authors: Amabile A, Migliara A, Capasso P, Biffi M, Cittaro D, Naldini L, Lombardo A.
SST Peer Reviewed

Marker-free coselection for CRISPR-driven genome editing in human cells.

Targeted genome editing enables the creation of bona fide cellular models for biological research and may be applied to human cell-based therapies. Therefore, broadly applicable and versatile methods for increasing its efficacy in cell populations...
Authors: Agudelo D, Duringer A, Bozoyan L, Huard CC, Carter S, Loehr J, Synodinou D, Drouin M, Salsman J,...
SST Peer Reviewed
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