Early Changes in Tumor Perfusion from T1-Weighted Dynamic Contrast-Enhanced MRI following Neural Stem Cell-Mediated Therapy of Recurrent High-Grade Glioma Correlate with Overall Su... by Prativa Sahoo, Paul Frankel, Julie Ressler, Margarita Gutova, Alexander J. Annala, Behnam Badie, Jana Portnow, Karen S. Aboody, Massimo D’Apuzzo, Russell C. Rockne

“…The aim of this study was to correlate T1-weighted dynamic contrast-enhanced MRI- (DCE-MRI-) derived perfusion parameters with overall survival of recurrent high-grade glioma patients who received neural stem cell- (NSC-) mediated enzyme/prodrug gene therapy. Methods. A total of 12 patients were included in this retrospective study. …”
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A genomic strategy for precision medicine in rare diseases: integrating customized algorithms into clinical practice by Cristina Méndez-Vidal, Nereida Bravo-Gil, Javier Pérez-Florido, Irene Marcos-Luque, Raquel M. Fernández, José Luis Fernández-Rueda, María González-del Pozo, Marta Martín-Sánchez, Elena Fernández-Suárez, Marcela Mena, Rosario Carmona, Joaquín Dopazo, Salud Borrego, Guillermo Antiñolo

“…These results led to over 5000 additional studies including carrier, prenatal and preimplantational tests or pharmacological and gene therapy treatments. Conclusion This strategy has shown promising improvements in the diagnostic rate, facilitating timely diagnosis and gradually expanding our services portfolio for rare diseases. …”
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Development of the inner ear and regeneration of hair cells after hearing impairment by Xin Bing, Chengcheng Liu, Xue Cao, Chengzhilin Li, Xiaochen Gao, Fangyuan Zhu, Xinhao Wu, Na Guo, Houyang Hu, Ming Xia, Miaoqing Zhao

“…In recent years, the protection of drug-related ototoxicity, inner ear stem cells, gene therapy, new materials, and signal regulation have become important ways to develop regeneration strategies of HCs. …”
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Integrative genomic analyses combined with molecular dynamics simulations reveal the impact of deleterious mutations of Bcl-2 gene on the apoptotic machinery and implications in ca... by Ghazi Elamin, Ghazi Elamin, Zhichao Zhang, Depika Dwarka, Kabange Kasumbwe, John Mellem, Nompumelelo P. Mkhwanazi, Paradise Madlala, Paradise Madlala, Mahmoud E. S. Soliman

“…Following 500 ns, MD simulations showed that these mutations caused a significant distortion in the protein conformational, protein-protein interactions (PPIs), and drug binding landscape compared to Bcl-2WT.ConclusionDespite being a predictive study, the findings presented in this report would offer a perspective insight for further experimental investigation, rational drug design, and cancer gene therapy.…”
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Systemic delivery of AAV5, AAV8, and AAV9 packaging a C5-12-microdystrophin-FLAG expression cassette in non-human primates by Mengping Liu, Erica Cook, Yanshan Dai, Erich Ehlert, Francois du Plessis, Jacek Lubelski, Bogdan G. Sleczka, Petia Shipkova, Zhuyin Li, Joshua Gamse, David Gordon, Leonard P. Adam, Paul C. Levesque, Glen B. Banks

“…Safely achieving therapeutic expression levels with adeno-associated virus (AAV) gene therapy is a significant challenge for treating the large muscle mass in humans. …”
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Fetal Spinal Cord Tissue in Mini-Guidance Channels Promotes Longitudinal Axonal Growth after Grafting into Hemisected Adult Rat Spinal Cords by Norman I. Bamber, Huaying Li, Patrick Aebischer, Xiao Ming Xu

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DNMT3A Mutations in Patients with Acute Myeloid Leukemia in South Brazil by Annelise Pezzi, Lauro Moraes, Vanessa Valim, Bruna Amorin, Gabriela Melchiades, Fernanda Oliveira, Maria Aparecida da Silva, Ursula Matte, Maria S. Pombo-de-Oliveira, Rosane Bittencourt, Liane Daudt, Lúcia Silla

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Peptide-based vaccine targeting IL17A attenuates experimental spondyloarthritis in HLA-B27 transgenic rats by Tetsuya Tomita, Hiromi Rakugi, Hiroki Hayashi, Ryuichi Morishita, Jiao Sun, Yuka Yanagida, Shota Yoshida, Satoshi Baba, Akiko Tenma, Masayoshi Toyoura, Sotaro Kawabata, Takako Ehara, Ryoko Asaki, Makoto Sakaguchi, Hideki Tomioka, Munehisa Shimamura, Hironori Nakagami

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CD94-driven in vitro expansion of highly functional adaptive NKG2C+ NKG2A- CD57+ NK cells from CMV+ healthy donors by Chiara Giordano, Simona Carlomagno, Simona Carlomagno, Michela Falco, Claudia Cantoni, Claudia Cantoni, Massimo Vitale, Ignazio Caruana, Johannes Dirks, Alberto Serio, Letizia Muccio, Giulia Bartalucci, Alessandra Bo, Franco Locatelli, Franco Locatelli, Cristina Bottino, Cristina Bottino, Simona Sivori, Simona Sivori, Mariella Della Chiesa, Mariella Della Chiesa

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High-accuracy crRNA array assembly strategy for multiplex CRISPR by Xiangtong Zhao, Lixian Yang, Peng Li, Zijing Cheng, Yongshi Jia, Limin Luo, Aihong Bi, Hanchu Xiong, Haibo Zhang, Hongen Xu, Jinrui Zhang, Yaodong Zhang

“…The study provides a flexible and powerful tool for the convenient implementation of multiplex CRISPR across DNA and RNA, facilitating the dissection of sophisticated cellular networks and the future realization of multi-target gene therapy.…”
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Mesenchymal Progenitor Cells and Their Orthopedic Applications: Forging a Path towards Clinical Trials by Deana S. Shenaq, Farbod Rastegar, Djuro Petkovic, Bing-Qiang Zhang, Bai-Cheng He, Liang Chen, Guo-Wei Zuo, Qing Luo, Qiong Shi, Eric R. Wagner, Enyi Huang, Yanhong Gao, Jian-Li Gao, Stephanie H. Kim, Ke Yang, Yang Bi, Yuxi Su, Gaohui Zhu, Jinyong Luo, Xiaoji Luo, Jiaqiang Qin, Russell R. Reid, Hue H. Luu, Rex C. Haydon, Tong-Chuan He

“…Thus, engineering such cells to maximize therapeutic potential has been the focus of cell/gene therapy to date. Here, we discuss the path towards the development of clinical trials utilizing BMSCs for orthopaedic applications. …”
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Comparable outcomes after busulfan- or treosulfan-based conditioning for allo-HSCT in children with ALL: results of FORUM by Krzysztof Kalwak, Laura M. Moser, Ulrike Pötschger, Peter Bader, Katharina Kleinschmidt, Roland Meisel, Jean-Hugues Dalle, Akif Yesilipek, Adriana Balduzzi, Gergely Krivan, Evgenios Goussetis, Raquel Staciuk, Petr Sedlacek, Herbert Pichler, Peter Svec, Melissa Gabriel, Tayfun Güngör, Ernest Bilic, Jochen Buechner, Marleen Renard, Kim Vettenranta, Marianne Ifversen, Cristina Diaz-de-Heredia, Jerry Stein, Jacek Toporski, Marc Bierings, Christina Peters, Marc Ansari, Franco Locatelli

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Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group by Alessia Cavazza, Francisco J. Molina-Estévez, Álvaro Plaza Reyes, Victor Ronco, Asma Naseem, Špela Malenšek, Peter Pečan, Annalisa Santini, Paula Heredia, Araceli Aguilar-González, Houria Boulaiz, Qianqian Ni, Marina Cortijo-Gutierrez, Kristina Pavlovic, Inmaculada Herrera, Berta de la Cerda, Emilio M. Garcia-Tenorio, Eva Richard, Sergio Granados-Principal, Arístides López-Márquez, Mariana Köber, Marijana Stojanovic, Melita Vidaković, Irene Santos-Garcia, Lorea Blázquez, Emily Haughton, Dongnan Yan, Rosario María Sánchez-Martín, Loubna Mazini, Gloria Gonzalez Aseguinolaza, Annarita Miccio, Paula Rio, Lourdes R. Desviat, Manuel A.F.V. Gonçalves, Ling Peng, Cecilia Jiménez-Mallebrera, Francisco Martin Molina, Dhanu Gupta, Duško Lainšček, Yonglun Luo, Karim Benabdellah

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Current Progress on MicroRNA-Based Gene Delivery in the Treatment of Osteoporosis and Osteoporotic Fracture by Xi Sun, Qi Guo, Wenhua Wei, Stephen Robertson, Ying Yuan, Xianghang Luo

“…Undoubtedly, microRNA-based gene therapies show great potential to become novel approaches against bone-related diseases, including osteoporosis and associated fractures. …”
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Anderson–Fabry Disease: From Endothelial Dysfunction to Emerging Therapies by Cosimo A. Stamerra, Rita Del Pinto, Paolo di Giosia, Claudio Ferri, Amirhossein Sahebkar

“…Substrate reduction, messenger ribonucleic acid (mRNA)-based, and gene therapies are also on the horizon. In this review, the clinical scenario and molecular aspects of Anderson–Fabry disease are described, along with updates on disease mechanisms and emerging therapies.…”
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Genetic Correction of Sickle Cell Anemia and β-Thalassemia: Progress and New Perspective by Ajay Perumbeti, Punam Malik

“…Gene therapy for β-globinopathies, particularly β-thalassemia and sickle cell anemia, holds promise for the future as a definitive corrective approach for these common and debilitating disorders. …”
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Spinal Fusion in the Next Generation: Gene and Cell Therapy Approaches by Marta Barba, Claudia Cicione, Camilla Bernardini, Vincenzo Campana, Ernesto Pagano, Fabrizio Michetti, Giandomenico Logroscino, Wanda Lattanzi

“…In recent years, cell and gene therapies have attracted great interest from the scientific community. …”
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Guiding the Differentiation Direction of Pancreatic Islet-Derived Stem Cells by Glycated Collagen by Gokhan Duruksu, Aysegul Aciksari

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Investigating Ultrafiltration Membranes and Operation Modes for Improved Lentiviral Vector Processing by Jennifer J. Labisch, Maria Evangelopoulou, Tobias Schleuß, Andreas Pickl

“…ABSTRACT The demand for lentiviral vectors (LVs) as tools for ex vivo gene therapies is ever‐increasing. Despite their promising applications, challenges in LV production remain largely due to the fragile envelope, which challenges the maintenance of vector stability. …”
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Pyruvate Kinase Deficiency Causing Priapism by Vinay Hanyalu Shankar, Bharadwaj Adithya-Sateesh, Nicole Gousy, Girma Ayele, Freyr Petursson, Rediet Atalay, Miriam Michael

“…Management strategies vary from full splenectomies to hematopoietic stem cell transplants with gene therapies with transfusions and administration of PK-activators coming in between. …”
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