Results 41 to 50 of about 1,173,119 (313)

ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing

BMC Genomics, 2020
Background Identifying nuclease-induced double-stranded breaks in DNA on a genome-wide scale is critical for assessing the safety and efficacy of genome editing therapies.
Camilo Breton, Peter M. Clark, Lili Wang, Jenny A. Greig, James M. Wilson   +4 more
doaj   +1 more source

Therapeutic potential of highly functional codon-optimized microutrophin for muscle-specific expression

Scientific Reports, 2022
High expectations have been set on gene therapy with an AAV-delivered shortened version of dystrophin (µDys) for Duchenne muscular dystrophy (DMD), with several drug candidates currently undergoing clinical trials.
Anna V. Starikova, Victoria V. Skopenkova, Anna V. Polikarpova, Denis A. Reshetov, Svetlana G. Vassilieva, Oleg A. Velyaev, Anna A. Shmidt, Irina M. Savchenko, Vladislav O. Soldatov, Tatiana V. Egorova, Maryana V. Bardina   +10 more
doaj   +1 more source

Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model

Molecular Therapy: Methods & Clinical Development, 2023
Limb-girdle muscular dystrophy (LGMD) type 2C/R5 results from mutations in the γ-sarcoglycan (SGCG) gene and is characterized by muscle weakness and progressive wasting.
Young-Eun Seo, Stephen H. Baine, Amber N. Kempton, Oliver C. Rogers, Sarah Lewis, Kaitlin Adegboye, Alex Haile, Danielle A. Griffin, Ellyn L. Peterson, Eric R. Pozsgai, Rachael A. Potter, Louise R. Rodino-Klapac   +11 more
doaj   +1 more source

Biological and technical complexities in analyzing extracellular vesicle immune interactions in B‐cell malignancies

FEBS Letters, EarlyView.
Extracellular vesicles (EVs) mediate intercellular communication in tumor immune microenvironments. However, their role in B‐cell malignancies remains poorly defined, owing to biological complexity and technical challenges in EV isolation and analysis.
Daniel Bachurski, Michael Hallek
wiley   +1 more source

FGF21 gene therapy as treatment for obesity and insulin resistance

EMBO Molecular Medicine, 2018
Prevalence of type 2 diabetes (T2D) and obesity is increasing worldwide. Currently available therapies are not suited for all patients in the heterogeneous obese/T2D population, hence the need for novel treatments.
Veronica Jimenez, Claudia Jambrina, Estefania Casana, Victor Sacristan, Sergio Muñoz, Sara Darriba, Jordi Rodó, Cristina Mallol, Miquel Garcia, Xavier León, Sara Marcó, Albert Ribera, Ivet Elias, Alba Casellas, Ignasi Grass, Gemma Elias, Tura Ferré, Sandra Motas, Sylvie Franckhauser, Francisca Mulero, Marc Navarro, Virginia Haurigot, Jesus Ruberte, Fatima Bosch   +23 more
doaj   +1 more source

Application of Raman Spectroscopy and Univariate Modelling As a Process Analytical Technology for Cell Therapy Bioprocessing

Frontiers in Medicine, 2018
Cell therapies offer unquestionable promises for the treatment, and in some cases even the cure, of complex diseases. As we start to see more of these therapies gaining market authorization, attention is turning to the bioprocesses used for their ...
Marc-Olivier Baradez, Daniela Biziato, Enas Hassan, Damian Marshall   +3 more
doaj   +1 more source

Gene therapy [PDF]

Oral Diseases, 2013
Applications of gene therapy have been evaluated in virtually every oral tissue, and many of these have proved successful at least in animal models. While gene therapy will not be used routinely in the next decade, practitioners of oral medicine should be aware of the potential of this novel type of treatment that doubtless will benefit many patients ...
openaire   +2 more sources

The epithelial barrier theory proposes a comprehensive explanation for the origins of allergic and other chronic noncommunicable diseases

FEBS Letters, EarlyView.
Exposure to common noxious agents (1), including allergens, pollutants, and micro‐nanoplastics, can cause epithelial barrier damage (2) in our body's protective linings. This may trigger an immune response to our microbiome (3). The epithelial barrier theory explains how this process can lead to chronic noncommunicable diseases (4) affecting organs ...
Can Zeyneloglu, Huseyn Babayev, Ismail Ogulur, Sena Ardicli, Yagiz Pat, Duygu Yazici, Bingjie Zhao, Lihong Chang, Xiaoqing Liu, Paolo D'Avino, Manru Li, Ceren Biçer, Fatma Hacer Kurtoğlu Babayev, Raja Dhir, Kari C. Nadeau, Marie‐Charlotte Brüggen, Mubeccel Akdis, Cezmi A. Akdis   +17 more
wiley   +1 more source

Therapeutic efficacy of thrombin-preconditioned mesenchymal stromal cell-derived extracellular vesicles on Escherichia coli-induced acute lung injury in mice

Respiratory Research
Background Acute lung injury (ALI) following pneumonia involves uncontrolled inflammation and tissue injury, leading to high mortality. We previously confirmed the significantly increased cargo content and extracellular vesicle (EV) production in ...
Yuna Bang, Sein Hwang, Young Eun Kim, Dong Kyung Sung, Misun Yang, So Yoon Ahn, Se In Sung, Kyeung Min Joo, Yun Sil Chang   +8 more
doaj   +1 more source

Expanding CAR T cells in human platelet lysate renders T cells with in vivo longevity

Journal for ImmunoTherapy of Cancer, 2019
Background Pre-clinical and clinical studies have shown that the infusion of CAR T cells with a naive-like (TN) and central memory (TCM) phenotype is associated with prolonged in vivo T cell persistence and superior anti-tumor effects.
Alejandro Torres Chavez, Mary Kathryn McKenna, Emanuele Canestrari, Christina T. Dann, Carlos A. Ramos, Premal Lulla, Ann M. Leen, Juan F. Vera, Norihiro Watanabe   +8 more
doaj   +1 more source