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Molecular Therapy: the Journal of the American Society of Gene Therapy

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https://www.readbyqxmd.com/read/30220614/sorting-out-the-best-enriching-hematopoietic-stem-cells-for-gene-therapy-and-editing
#1
EDITORIAL
Stefan Radtke, Olivier Humbert, Hans-Peter Kiem
No abstract text is available yet for this article.
September 13, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30217727/nasal-spray-to-the-rescue-of-neurons-in-stroke
#2
Christian Alzheimer
No abstract text is available yet for this article.
September 11, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30217731/complement-c3-targeted-gene-therapy-restricts-onset-and-progression-of-neurodegeneration-in-chronic-mouse-glaucoma
#3
Alejandra Bosco, Sarah R Anderson, Kevin T Breen, Cesar O Romero, Michael R Steele, Vince A Chiodo, Sanford L Boye, William W Hauswirth, Stephen Tomlinson, Monica L Vetter
Dysregulation of the complement system is implicated in neurodegeneration, including human and animal glaucoma. Optic nerve and retinal damage in glaucoma is preceded by local complement upregulation and activation, but whether targeting this early innate immune response could have therapeutic benefit remains undefined. Because complement signals through three pathways that intersect at complement C3 activation, here we targeted this step to restore complement balance in the glaucomatous retina and to determine its contribution to degeneration onset and/or progression...
August 24, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30217729/upregulation-of-lncrna-adamts9-as2-promotes-salivary-adenoid-cystic-carcinoma-metastasis-via-pi3k-akt-and-mek-erk-signaling
#4
Shule Xie, Xin Yu, Yingru Li, Hanyu Ma, Song Fan, Weixiong Chen, Guokai Pan, Weiwei Wang, Hanqing Zhang, Jinsong Li, Zhaoyu Lin
Neurotropic infiltrative growth and distant metastasis are the main causes of death in salivary adenoid cystic carcinoma (SACC) patients. Long noncoding RNAs (lncRNAs) are involved in many human neoplasms, however, their potential roles in SACC are unclear. In our study, we found that ADAM metallopeptidase with thrombospondin type 1 motif, 9 (ADAMTS9) antisense RNA 2 (ADAMTS9-AS2) was significantly upregulated in SACC patients with metastasis and SACC-lung metastasis (LM) cells. Moreover, ADAMTS9-AS2 expression was closely associated with the prognosis and distant metastasis in SACC patients...
August 24, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30217730/molecular-recalibration-of-pd-1-antigen-specific-t-cells-from-blood-and-liver
#5
Itziar Otano, David Escors, Anna Schurich, Harsimran Singh, Francis Robertson, Brian R Davidson, Giuseppe Fusai, Frederick A Vargas, Zhi M D Tan, Jia Y J Aw, Navjyot Hansi, Patrick T F Kennedy, Shao-An Xue, Hans J Stauss, Antonio Bertoletti, Andrea Pavesi, Mala K Maini
Checkpoint inhibitors and adoptive cell therapy provide promising options for treating solid cancers such as HBV-related HCC, but they have limitations. We tested the potential to combine advantages of each approach, genetically reprogramming T cells specific for viral tumor antigens to overcome exhaustion by down-modulating the co-inhibitory receptor PD-1. We developed a novel lentiviral transduction protocol to achieve preferential targeting of endogenous or TCR-redirected, antigen-specific CD8 T cells for shRNA knockdown of PD-1 and tested functional consequences for antitumor immunity...
August 16, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30217728/maturation-of-human-induced-pluripotent-stem-cell-derived-cardiomyocytes-by-soluble-factors-from-human-mesenchymal-stem-cells
#6
Shohei Yoshida, Shigeru Miyagawa, Satsuki Fukushima, Takuji Kawamura, Noriyuki Kashiyama, Fumiya Ohashi, Toshihiko Toyofuku, Koichi Toda, Yoshiki Sawa
In this study, we proposed that the functionality or phenotype of differentiated cardiomyocytes derived from human induced pluripotent stem cells (iPSC-CMs) might be modified by co-culture with mesenchymal stem cells (MSCs), resulting in an improved therapeutic potential for failing myocardial tissues. Structural, motility, electrophysiological, and metabolic analyses revealed that iPSC-CMs co-cultured with MSCs displayed aligned myofibrils with A-, H-, and I-bands that could contract and relax quickly, indicating the promotion of differentiation and the establishment of the iPSC-CM structural framework, and showed clear gap junctions and an electric pacing of >2 Hz, indicating enhanced cell-cell interactions...
August 16, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30195725/pdgf-restores-the-defective-phenotype-of-adipose-derived-mesenchymal-stromal-cells-from-diabetic-patients
#7
Vivian Capilla-González, Javier López-Beas, Natalia Escacena, Yolanda Aguilera, Antonio de la Cuesta, Rafael Ruiz-Salmerón, Franz Martín, Abdelkrim Hmadcha, Bernat Soria
Diabetes is a chronic metabolic disorder that affects 415 million people worldwide. This pathology is often associated with long-term complications, such as critical limb ischemia (CLI), which increases the risk of limb loss and mortality. Mesenchymal stromal cells (MSCs) represent a promising option for the treatment of diabetes complications. Although MSCs are widely used in autologous cell-based therapy, their effects may be influenced by the constant crosstalk between the graft and the host, which could affect the MSC fate potential...
August 16, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30195724/crispr-induced-deletion-with-sacas9-restores-dystrophin-expression-in-dystrophic-models-in-vitro-and-in-vivo
#8
Benjamin L Duchêne, Khadija Cherif, Jean-Paul Iyombe-Engembe, Antoine Guyon, Joel Rousseau, Dominique L Ouellet, Xavier Barbeau, Patrick Lague, Jacques P Tremblay
Duchenne muscular dystrophy (DMD), a severe hereditary disease affecting 1 in 3,500 boys, mainly results from the deletion of exon(s), leading to a reading frameshift of the DMD gene that abrogates dystrophin protein synthesis. Pairs of sgRNAs for the Cas9 of Staphylococcus aureus were meticulously chosen to restore a normal reading frame and also produce a dystrophin protein with normally phased spectrin-like repeats (SLRs), which is not usually obtained by skipping or by deletion of complete exons. This can, however, be obtained in rare instances where the exon and intron borders of the beginning and the end of the complete deletion (patient deletion plus CRISPR-induced deletion) are at similar positions in the SLR...
August 16, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30166242/correction-of-the-marfan-syndrome-pathogenic-fbn1-mutation-by-base-editing-in-human-cells-and-heterozygous-embryos
#9
Yanting Zeng, Jianan Li, Guanglei Li, Shisheng Huang, Wenxia Yu, Yu Zhang, Dunjin Chen, Jia Chen, Jianqiao Liu, Xingxu Huang
There are urgent demands for efficient treatment of heritable genetic diseases. The base editing technology has displayed its efficiency and precision in base substitution in human embryos, providing a potential early-stage treatment for genetic diseases. Taking advantage of this technology, we corrected a Marfan syndrome pathogenic mutation, FBN1T7498C . We first tested the feasibility in mutant cells, then successfully achieved genetic correction in heterozygous human embryos. The results showed that the BE3 mediated perfect correction at the efficiency of about 89%...
August 14, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30143435/transvascular-delivery-of-hydrophobically-modified-sirnas-gene-silencing-in-the-rat-brain-upon-disruption-of-the-blood-brain-barrier
#10
Bruno M D C Godinho, Nils Henninger, James Bouley, Julia F Alterman, Reka A Haraszti, James W Gilbert, Ellen Sapp, Andrew H Coles, Annabelle Biscans, Mehran Nikan, Dimas Echeverria, Marian DiFiglia, Neil Aronin, Anastasia Khvorova
Effective transvascular delivery of therapeutic oligonucleotides to the brain presents a major hurdle to the development of gene silencing technologies for treatment of genetically defined neurological disorders. Distribution to the brain after systemic administrations is hampered by the low permeability of the blood-brain barrier (BBB) and the rapid clearance kinetics of these drugs from the blood. Here we show that transient osmotic disruption of the BBB enables transvascular delivery of hydrophobically modified small interfering RNA (hsiRNA) to the rat brain...
August 8, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30131302/disruption-of-chromosomal-architecture-of-cox2-locus-sensitizes-lung-cancer-cells-to-radiotherapy
#11
Yuxiang Sun, Hui Dai, Shaopeng Chen, Yajun Zhang, Tao Wu, Xianbin Cao, Guoping Zhao, An Xu, Jun Wang, Lijun Wu
Despite treatment of lung cancer with radiotherapy and chemotherapy, the survival rate of lung cancer patients remains poor. Previous studies demonstrated the importance of upregulation of inflammatory factors, such as cyclooxygenase 2 (cox2), in tumor tolerance. In the present study, we investigated the role of cox2 in radiosensitivity of lung cancer. Our results showed that the combination treatment of radiation with aspirin, an anti-inflammatory drug, induced a synergistic reduction of cell survival in A549 and H1299 lung cancer cells...
August 8, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30131301/a-novel-anti-lilrb4-car-t-cell-for-the-treatment-of-monocytic-aml
#12
Samuel John, Heyu Chen, Mi Deng, Xun Gui, Guojin Wu, Weina Chen, Zunling Li, Ningyan Zhang, Zhiqiang An, Cheng Cheng Zhang
To effectively improve treatment for acute myeloid leukemia (AML), new molecular targets and therapeutic approaches need to be identified. Chimeric antigen receptor (CAR)-modified T cells targeting tumor-associated antigens have shown promise in the treatment of some malignancies. However, CAR-T cell development for AML has been limited by lack of an antigen with high specificity for AML cells that is not present on normal hematopoietic stem cells, and thus will not result in myelotoxicity. Here we demonstrate that leukocyte immunoglobulin-like receptor-B4 (LILRB4) is a tumor-associated antigen highly expressed on monocytic AML cells...
August 7, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30122422/crispr-cas9-mediated-in-situ-correction-of-lamb3-gene-in-keratinocytes-derived-from-a-junctional-epidermolysis-bullosa-patient
#13
Daniela Benati, Francesca Miselli, Fabienne Cocchiarella, Clarissa Patrizi, Marta Carretero, Samantha Baldassarri, Virginia Ammendola, Cristina Has, Stefano Colloca, Marcela Del Rio, Fernando Larcher, Alessandra Recchia
Deficiency of basement membrane heterotrimeric laminin 332 component, coded by LAMA3, LAMB3, and LAMC2 genes, causes junctional epidermolysis bullosa (JEB), a severe skin adhesion defect. Herein, we report the first application of CRISPR/Cas9-mediated homology direct repair (HDR) to in situ restore LAMB3 expression in JEB keratinocytes in vitro and in immunodeficient mice transplanted with genetically corrected skin equivalents. We packaged an adenovector carrying Cas9/guide RNA (gRNA) tailored to the intron 2 of LAMB3 gene and an integration defective lentiviral vector bearing a promoterless quasi-complete LAMB3 cDNA downstream a splice acceptor site and flanked by homology arms...
August 4, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30120059/exosome-derived-mir-130a-activates-angiogenesis-in-gastric-cancer-by-targeting-c-myb-in-vascular-endothelial-cells
#14
Haiou Yang, Haiyang Zhang, Shaohua Ge, Tao Ning, Ming Bai, Jialu Li, Shuang Li, Wu Sun, Ting Deng, Le Zhang, Guoguang Ying, Yi Ba
Metastasis is a crucial reason for the poor prognosis of gastric cancer. Angiogenesis is closely associated with tumor invasion and metastasis. Cancer-derived exosomes play an important role in the establishment of the tumor microenvironment. In this study, exosomes were isolated by sequential differential centrifugation, and they were verified by transmission electron microscopy. Changes in the biological behavior of human umbilical vein endothelial cells were evaluated with downstream cellular functional experiments...
August 4, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30078765/a-nanostructured-lipid-carrier-for-delivery-of-a-replicating-viral-rna-provides-single-low-dose-protection-against-zika
#15
Jesse H Erasmus, Amit P Khandhar, Jeff Guderian, Brian Granger, Jacob Archer, Michelle Archer, Emily Gage, Jasmine Fuerte-Stone, Elise Larson, Susan Lin, Ryan Kramer, Rhea N Coler, Christopher B Fox, Dan T Stinchcomb, Steven G Reed, Neal Van Hoeven
Since the first demonstration of in vivo gene expression from an injected RNA molecule almost two decades ago,1 the field of RNA-based therapeutics is now taking significant strides, with many cancer and infectious disease targets entering clinical trials.2 Critical to this success has been advances in the knowledge and application of delivery formulations. Currently, various lipid nanoparticle (LNP) platforms are at the forefront,3 but the encapsulation approach underpinning LNP formulations offsets the synthetic and rapid-response nature of RNA vaccines...
August 2, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30093305/post-injury-nose-to-brain-delivery-of-activin-a-and-serpinb2-reduces-brain-damage-in-a-mouse-stroke-model
#16
Bettina Buchthal, Ursula Weiss, Hilmar Bading
Synaptic NMDA receptors activating nuclear calcium-driven adaptogenomics control a potent body-own neuroprotective mechanism, referred to as acquired neuroprotection. Viral vector-mediated gene transfer in conjunction with stereotactic surgery has previously demonstrated the proficiency of several nuclear calcium-regulated genes to protect in vivo against brain damage caused by toxic extrasynaptic NMDA receptor signaling following seizures or stroke. Here we used noninvasive nose-to-brain administration of Activin A and SerpinB2, two secreted nuclear calcium-regulated neuroprotectants, for post-injury treatment of brain damage following middle cerebral artery occlusion (MCAO) in C57BL/6N mice...
July 23, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30078764/a-drug-tunable-gene-therapy-for-broad-spectrum-protection-against-retinal-degeneration
#17
Clayton P Santiago, Casey J Keuthan, Sanford L Boye, Shannon E Boye, Aisha A Imam, John D Ash
Retinal degenerations are a large cluster of diseases characterized by the irreversible loss of light-sensitive photoreceptors that impairs the vision of 9.1 million people in the US. An attractive treatment option is to use gene therapy to deliver broad-spectrum neuroprotective factors. However, this approach has had limited clinical translation because of the inability to control transgene expression. To address this problem, we generated an adeno-associated virus vector named RPF2 that was engineered to express domains of leukemia inhibitory factor fused to the destabilization domain of bacterial dihydrofolate reductase...
July 19, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30093306/systemic-aav-micro-dystrophin-gene-therapy-for-duchenne-muscular-dystrophy
#18
REVIEW
Dongsheng Duan
Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene mutation. Conceptually, replacing the mutated gene with a normal one would cure the disease. However, this task has encountered significant challenges due to the enormous size of the gene and the distribution of muscle throughout the body. The former creates a hurdle for viral vector packaging and the latter begs for whole-body therapy. To address these obstacles, investigators have invented the highly abbreviated micro-dystrophin gene and developed body-wide systemic gene transfer with adeno-associated virus (AAV)...
July 17, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30078766/longitudinal-in-vivo-monitoring-of-the-cns-demonstrates-the-efficacy-of-gene-therapy-in-a-sheep-model-of-cln5-batten-disease
#19
Nadia L Mitchell, Katharina N Russell, Martin P Wellby, Hollie E Wicky, Lucia Schoderboeck, Graham K Barrell, Tracy R Melzer, Steven J Gray, Stephanie M Hughes, David N Palmer
Neuronal ceroid lipofuscinoses (NCLs; Batten disease) are neurodegenerative lysosomal storage diseases predominantly affecting children. Single administration of brain-directed lentiviral or recombinant single-stranded adeno-associated virus 9 (ssAAV9) vectors expressing ovine CLN5 into six pre-clinically affected sheep with a naturally occurring CLN5 NCL resulted in long-term disease attenuation. Treatment efficacy was demonstrated by non-invasive longitudinal in vivo monitoring developed to align with assessments used in human medicine...
July 17, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/30077612/blocking-macrophage-migration-inhibitory-factor-protects-against-cisplatin-induced-acute-kidney-injury-in-mice
#20
Jinhong Li, Ying Tang, Patrick M K Tang, Jun Lv, Xiao-Ru Huang, Christine Carlsson-Skwirut, Lydie Da Costa, Anna Aspesi, Suada Fröhlich, Pawel Szczęśniak, Philipp Lacher, Jörg Klug, Andreas Meinhardt, Günter Fingerle-Rowson, Rujun Gong, Zhihua Zheng, Anping Xu, Hui-Yao Lan
Macrophage migration inhibitory factor (MIF) is elevated in patients with acute kidney injury (AKI) and is suggested as a potential predictor for renal replacement therapy in AKI. In this study, we found that MIF also plays a pathogenic role and is a therapeutic target for AKI. In a cisplatin-induced AKI mouse model, elevated plasma MIF correlated with increased serum creatinine and the severity of renal inflammation and tubular necrosis, whereas deletion of MIF protected the kidney from cisplatin-induced AKI by largely improving renal functional and histological injury, and suppressing renal inflammation including upregulation of cytokines such as interleukin (IL)-1β, tumor necrosis factor-alpha (TNF-α), IL-6, inducible nitric oxide synthase (iNOS), MCP-1, IL-8, and infiltration of macrophages, neutrophils, and T cells...
July 17, 2018: Molecular Therapy: the Journal of the American Society of Gene Therapy
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