N-of-1 medicine by Peter Wang, Qiao Ying Leong, Ni Yin Lau, Wei Ying Ng, Siong Peng Kwek, Lester Tan, Shang-Wei Song, Kui You, Li Ming Chong, Isaiah Zhuang, Yoong Hun Ong, Nigel Foo, Xavier Tadeo, Kirthika Senthil Kumar, Smrithi Vijayakumar, Yoann Sapanel, Marlena Natalia Raczkowska, Alexandria Remus, Agata Blasiak, Dean Ho

“…Examples include genome/molecular alteration-guided drug selection, single-patient gene therapy design and synergy-based drug combination development, and these approaches can yield substantially diverse recommendations. …”
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DNGR-1 limits Flt3L-mediated antitumor immunity by restraining tumor-infiltrating type I conventional dendritic cells by Ignacio Melero, Ruth Conde-Garrosa, David Sancho, Santos Mañes, Alfonso R Sanchez-Paulete, Francisco J Cueto, Carlos del Fresno, Paola Brandi, Alexis J. Combes, Elena Hernández-García, Michel Enamorado, Christian P Bromley, Manuel J Gomez, Santiago Zelenay, Salvador Iborra, Matthew F. Krummel

“…To overexpress Flt3L systemically, we performed gene therapy through the hydrodynamic injection of an Flt3L-encoding plasmid. …”
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Hydrogel-based nanoparticles: revolutionizing brain tumor treatment and paving the way for future innovations by Alireza Shadab, Simin Farokhi, Arshia Fakouri, Neda Mohagheghzadeh, Ali Noroozi, Zahra Sadat Razavi, Arian Karimi Rouzbahani, Hamidreza Zalpoor, Mohamad Mahjoor

“…This includes investigating tailored hydrogels, hybrid systems, computational modeling, and the integration of gene therapy and immunotherapy techniques. The study also addresses the need for enhanced synthesis techniques, stability, scalability, and cost-cutting measures to overcome obstacles and advance the clinical use of hydrogel-based nanoparticles in treating brain tumors. …”
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PET imaging of AAV9 and AAVBR1 trafficking in normal mice by Aditya Bansal, Shalini Sharma, Manasa Kethamreddy, Mukesh K. Pandey

“…Abstract Adeno-associated virus (AAV) mediated gene therapy is advancing and needs a noninvasive imaging tool to evaluate its effective targeting, biodistribution and clearance for precise use in humans. …”
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Divergence in photoreceptor cell death and neuroinflammation in transvitreal and transscleral subretinal delivery in mice by Daniel E. Maidana, Sara Pastor Puente, Catherine Wang, Shivam Chandra, Lucia Gonzalez-Buendia, Eleftherios Paschalis Ilios, Andrius Kazlauskas, Demetrios G. Vavvas

“…Abstract Subretinal injections provide direct access to photoreceptors and RPE, which is crucial for the delivery of gene therapy and neuroprotective approaches. To access the subretinal space, transvitreal (TV) and transscleral (TS) subretinal injections have been widely used in humans and animal models. …”
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Synergistic strategies for glioblastoma treatment: CRISPR-based multigene editing combined with immune checkpoint blockade by Xiaolin Liu, Xiao Liu, Xiaonan Luo, Maorong Zhu, Nannan Liu, Juan Li, Qi Zhang, Cheng Zou, Yuxin Wu, Zhengcong Cao, Shuangxin Ma, Weizhong Wang, Guangzhao Yang, Jintao Gu, Wei Liu, Meng Li, Anan Yin, Yalong He, Wei Lin

“…Furthermore, the EVs contained Cas9 protein and sgRNA for precise and minimally invasive gene therapy, which addressing the key barriers associated with in vivo CRISPR‒Cas9 gene editing treatment. …”
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Virus-inspired biogenic delivery system for advancing cancer therapy by Di Sun, Hao Liang, Qianwen Mu, Chengchao Chu, Gang Liu, Chao Liu

“…Virus-inspired particles have been utilized in various applications, including vaccination, gene therapy, drug therapy, and diagnostics. Biogenic delivery systems imitating the natural structure of viruses are regarded as innovative nanoplatforms used to deliver drug compounds to related sites and target cells in organisms. …”
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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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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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