Results 91 to 100 of about 626,396 (286)

Targeted Gene Delivery through the Respiratory System: Rationale for Intratracheal Gene Transfer

Journal of Cardiovascular Development and Disease, 2019
Advances in DNA- and RNA-based technologies have made gene therapy suitable for many lung diseases, especially those that are hereditary. The main objective of gene therapy is to deliver an adequate amount of gene construct to the intended target cell ...
Michael G. Katz, Anthony S. Fargnoli, Sarah M. Gubara, Kenneth Fish, Thomas Weber, Charles R. Bridges, Roger J. Hajjar, Kiyotake Ishikawa   +7 more
doaj   +1 more source

CRISPR-Cas9 screens in human cells and primary neurons identify modifiers of C9ORF72 dipeptide-repeat-protein toxicity. [PDF]

, 2018
Hexanucleotide-repeat expansions in the C9ORF72 gene are the most common cause of amyotrophic lateral sclerosis and frontotemporal dementia (c9ALS/FTD).
A Berson, A Jovičić, AA Dominguez, Aaron D. Gitler, AC Elden, AD Gitler, AD Gitler, AD Gitler, AE Renton, AM Jablonski, Amy Li, Ana Jovičić, AS Ng, BD Freibaum, C Sidrauski, C Sidrauski, C. Kimberly Tsui, D Ron, David W. Morgens, DW Morgens, DW Morgens, E Yeger-Lotem, EM Lynes, EY Son, FB Gao, Gregor Bieri, H Koike-Yusa, HJ Kim, HJ Kim, I Kwon, IR Mackenzie, IR Mackenzie, J Couthouis, J Joung, James Ousey, JC Christianson, JG Wideman, JP Taylor, Julien Couthouis, Justin K. Ichida, K Kanekura, K Mori, K Zhang, KH Lee, KM Green, KY Shi, L Ellgaard, L Huang, LA Becker, LA Gilbert, LA Gilbert, M Armakola, M DeJesus-Hernandez, M Neumann, M Prudencio, MA Horlbeck, Marc Tessier-Lavigne, MC Jonikas, MH Schludi, MI Love, Michael C. Bassik, Michael S. Haney, NF Liachko, Nicholas J. Kramer, Nicholas T. Hertz, O Shalem, OM Peters, PE Ash, Q Zhou, R Dafinca, R Lopez-Gonzalez, R Seijffers, RJ Platt, RM Deans, Rosanna Ma, S Boeynaems, S Boeynaems, S Ju, S Konermann, S Mizielinska, S Treusch, SC Ling, SJ Zhang, T Wang, T Westergard, T Zu, TF Gendron, W Cheng, X Wen, Y Lin, Y Zhou, Yingxiao Shi, YJ Zhang, YS Davidson, Z Shevtsova, Z Sun   +95 more
core   +1 more source

Predictors of response and rational combinations for the novel MCL‐1 inhibitor MIK665 in acute myeloid leukemia

Molecular Oncology, EarlyView.
This study characterizes the responses of primary acute myeloid leukemia (AML) patient samples to the MCL‐1 inhibitor MIK665. The results revealed that monocytic differentiation is associated with MIK665 sensitivity. Conversely, elevated ABCB1 expression is a potential biomarker of resistance to the treatment, which can be overcome by the combination ...
Joseph Saad, Rhiannon Newman, Elmira Khabusheva, Sofia Aakko, Eric Durand, Mahesh Tambe, Heikki Kuusanmäki, Alun Parsons, Juho J. Miettinen, Komal Kumar Javarappa, Ezgi June Olgac, Nemo Ikonen, Mika Kontro, Kimmo Porkka, Heiko Maacke, Janghee Woo, Ensar Halilovic, Caroline A. Heckman   +17 more
wiley   +1 more source

An efficient method for the transduction of primary pediatric glioma neurospheres

MethodsX, 2018
Pediatric high grade glioma (pHGG) and diffuse intrinsic pontine glioma (DIPG) are rare, but rapidly fatal malignancies of the central nervous system (CNS), and the leading cause of cancer-related death in children.
Michaël H. Meel, Dennis S. Metselaar, Piotr Waranecki, Gertjan J.L. Kaspers, Esther Hulleman   +4 more
doaj   +1 more source

Rosuvastatin Enhances VSV-G Lentiviral Transduction of NK Cells via Upregulation of the Low-Density Lipoprotein Receptor

Molecular Therapy: Methods & Clinical Development, 2020
Adoptive natural killer (NK) cell therapy is attaining promising clinical outcomes in recent years, but improvements are needed. Genetic modification of NK cells with a tumor antigen-specific receptor on their surface coupled to intracellular signaling ...
Ying Gong, Roel G.J. Klein Wolterink, Ian Janssen, Arjan J. Groot, Gerard M.J. Bos, Wilfred T.V. Germeraad   +5 more
doaj   +1 more source

Signal transducer and activator of transcription 2 deficiency is a novel disorder of mitochondrial fission [PDF]

, 2015
Defects of mitochondrial dynamics are emerging causes of neurological disease. In two children presenting with severe neurological deterioration following viral infection we identified a novel homozygous STAT2 mutation, c.1836C4A (p.Cys612Ter), using ...
Anderson, G, Cale, CM, Chalasani, A, Chan, E, Duchen, MR, Gilmour, KC, Hambleton, S, Jacques, S, Osellame, LD, Plagnol, V, Qasim, W, Rahman, S, Shahni, R, Taanman, JW, Wadatilake, Y   +14 more
core   +1 more source

The neural crest‐associated gene ERRFI1 is involved in melanoma progression and resistance toward targeted therapy

Molecular Oncology, EarlyView.
ERRFI1, a neural crest (NC)‐associated gene, was upregulated in melanoma and negatively correlated with the expression of melanocytic differentiation markers and the susceptibility of melanoma cells toward BRAF inhibitors (BRAFi). Knocking down ERRFI1 significantly increased the sensitivity of melanoma cells to BRAFi.
Nina Wang, Qian Sun, Daniel Novak, Lei Zhu, Juliane Poelchen, Tamara Steinfass, Yiman Wang, Viktor Umansky, Jochen Utikal   +8 more
wiley   +1 more source

Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization. [PDF]

, 2018
Lentiviral vector mobilization following HIV-1 infection of vector-transduced cells poses biosafety risks to vector-treated patients and their communities.
Bi, Yanmin, Fry, Nate J, Hu, Peirong, Kafri, Tal, Kohn, Donald B, Ma, Hong, Suwanmanee, Thipparat, Zeithaml, Brian   +7 more
core  

Intein‐based modular chimeric antigen receptor platform for specific CD19/CD20 co‐targeting

Molecular Oncology, EarlyView.
CARtein is a modular CAR platform that uses split inteins to splice antigen‐recognition modules onto a universal signaling backbone, enabling precise, scarless assembly without re‐engineering signaling domains. Deployed here against CD19 and CD20 in B‐cell malignancies, the design supports flexible multi‐antigen targeting to boost T‐cell activation and
Pablo Gonzalez‐Garcia, Noelia Moares, Wenjie Yi‐He, Rosa Luna‐Espejo, Ricardo Fernandez‐Cisnal, Javier Ocaña‐Cuesta, Juan P. Muñoz‐Miranda, Antonio Gabucio, Cecilia M. Fernandez‐Ponce, Francisco Garcia‐Cozar   +9 more
wiley   +1 more source

Improving PARP inhibitor efficacy in bladder cancer without genetic BRCAness by combination with PLX51107

Molecular Oncology, EarlyView.
Clinical trials on PARP inhibitors in urothelial carcinoma (UC) showed limited efficacy and a lack of predictive biomarkers. We propose SLFN5, SLFN11, and OAS1 as UC‐specific response predictors. We suggest Talazoparib as the better PARP inhibitor for UC than Olaparib.
Jutta Schmitz, Anna L. Bartkowiak, Michael Rose, Nora Kolks, Patrick Petzsch, Vandana Solanki, Anne Stoffel, Bianca Faßbender, Leandra Lepping, Julka Volkamer, Karl Köhrer, Marc Seifert, Tokameh Mahmoudi, Tahlita C. M. Zuiverloon, Günter Niegisch, Michèle J. Hoffmann   +15 more
wiley   +1 more source