Results 41 to 50 of about 1,204,526 (212)

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

Gene Therapy [PDF]

Internal Medicine, 1993
F, Levine, T, Friedmann
  +8 more sources

Early induction of cytokine release syndrome by rapidly generated CAR T cells in preclinical models

EMBO Molecular Medicine
Cytokine release syndrome (CRS) is a significant side-effect of conventional chimeric antigen receptor (CAR) T-cell therapy. To facilitate patient accessibility, short-term (st) CAR T cells, which are administered to patients only 24 h after vector ...
Arezoo Jamali, Naphang Ho, Angela Braun, Elham Adabi, Frederic B Thalheimer, Christian J Buchholz   +5 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

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

Correction to: 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
An amendment to this paper has been published and can be accessed via the original article.
Camilo Breton, Peter M. Clark, Lili Wang, Jenny A. Greig, James M. Wilson   +4 more
doaj   +1 more source

Gene therapy for hemophilia [PDF]

Frontiers in Bioscience, 2015
Hemophilia is an X-linked inherited bleeding disorder consisting of two classifications, hemophilia A and hemophilia B, depending on the underlying mutation. Although the disease is currently treatable with intravenous delivery of replacement recombinant clotting factor, this approach represents a significant cost both monetarily and in terms of ...
Geoffrey L, Rogers, Roland W, Herzog
openaire   +2 more sources

Involvement of hepatic macrophages in the antifibrotic effect of IGF-I-overexpressing mesenchymal stromal cells

Stem Cell Research & Therapy, 2016
Background Cirrhosis is a major health problem worldwide and new therapies are needed. Hepatic macrophages (hMø) have a pivotal role in liver fibrosis, being able to act in both its promotion and its resolution.
Esteban Fiore, Mariana Malvicini, Juan Bayo, Estanislao Peixoto, Catalina Atorrasagasti, Romina Sierra, Marcelo Rodríguez, Sofia Gómez Bustillo, Mariana G. García, Jorge B. Aquino, Guillermo Mazzolini   +10 more
doaj   +1 more source

Identifying microRNA determinants of human myelopoiesis

Scientific Reports, 2018
Myelopoiesis involves differentiation of hematopoietic stem cells to cellular populations that are restricted in their self-renewal capacity, beginning with the common myeloid progenitor (CMP) and leading to mature cells including monocytes and ...
Megha Rajasekhar, Ulf Schmitz, Stephane Flamant, Justin J.-L. Wong, Charles G. Bailey, William Ritchie, Jeff Holst, John E. J. Rasko   +7 more
doaj   +1 more source

Drug-Inducible Gene Therapy Effectively Reduces Spontaneous Seizures in Kindled Rats but Creates Off-Target Side Effects in Inhibitory Neurons [PDF]

, 2023
Kyle A. Sullivan, Iuliia Vitko, K. L. Blair, Ronald P. Gaykema, Madison J. Failor, Jennifer M. San Pietro, Deblina Dey, John Williamson, Ruth L. Stornetta, Jaideep Kapur, Edward Perez‐Reyes   +10 more
openalex   +1 more source