Targeted Inhibition of the miR-199a/214 Cluster by CRISPR Interference Augments the Tumor Tropism of Human Induced Pluripotent Stem Cell-Derived Neural Stem Cells under Hypoxic Con... by Yumei Luo, Xuehu Xu, Xiuli An, Xiaofang Sun, Shu Wang, Detu Zhu

“…These findings suggest a novel application of CRISPRi in NSC-based tumor-targeted gene therapy.…”
Get full text

Clinical and molecular outcomes from the 5-Year natural history study of SSADH Deficiency, a model metabolic neurodevelopmental disorder by Itay Tokatly Latzer, Jean-Baptiste Roullet, Wardiya Afshar-Saber, Henry H. C. Lee, Mariarita Bertoldi, Gabrielle E. McGinty, Melissa L. DiBacco, Erland Arning, Melissa Tsuboyama, Alexander Rotenberg, Thomas Opladen, Kathrin Jeltsch, Àngels García-Cazorla, Natalia Juliá-Palacios, K. Michael Gibson, Mustafa Sahin, Phillip L. Pearl

“…This further enables the identification of biomarkers and changes throughout development that will be essential for upcoming targeted trials of enzyme replacement and gene therapy.…”
Get full text

HPV-Based Screening, Triage, Treatment, and Followup Strategies in the Management of Cervical Intraepithelial Neoplasia by Oscar Peralta-Zaragoza, Jessica Deas, Claudia Gómez-Cerón, Wendy Argelia García-Suastegui, Geny del Socorro Fierros-Zárate, Nadia Judith Jacobo-Herrera

“…Therefore, in this paper, we discuss the main diagnostic methods, management strategies, and followup of HPV-associated cervical lesions and review clinical trials applying gene therapy strategies against the development of cervical cancer.…”
Get full text

T cell receptors specific for an imatinib-induced mutation in BCR-ABL for adoptive T cell therapy by Meng-Tung Hsu, Gerald Willimsky, Gerald Willimsky, Gerald Willimsky, Leo Hansmann, Leo Hansmann, Leo Hansmann, Leo Hansmann, Thomas Blankenstein

Get full text

Local Overexpression of Interleukin-11 in the Central Nervous System Limits Demyelination and Enhances Remyelination by Anurag Maheshwari, Kris Janssens, Jeroen Bogie, Chris Van Den Haute, Tom Struys, Ivo Lambrichts, Veerle Baekelandt, Piet Stinissen, Jerome J. A. Hendriks, Helena Slaets, Niels Hellings

Get full text

Promoter Polymorphism of RGS2 Gene Is Associated with Change of Blood Pressure in Subjects with Antihypertensive Treatment: The Azelnidipine and Temocapril in Hypertensive Patients... by Ken Sugimoto, Tomohiro Katsuya, Kei Kamide, Tomomi Fujisawa, Izumi Shimaoka, Mitsuru Ohishi, Ryuichi Morishita, Toshio Ogihara, Hiromi Rakugi

Get full text

The combination of rAAV pseudo-lipid nanoparticle and triamcinolone acetonide enables multi-administration to liver by Chunmei Gan, Mi Leng, Yu Liu, Zhaoyue Zheng, Siwu He, Wen Qiao, Lin Xiao, Yao Xiao, Jingya Ye, Lixing Zhou, Jiao Zhou, Boduan Xiao, Wenxin Zhao, Jiamei Yang, Aohan Wu, Huiyuan Zhang, Hongbo Hu, Xiaobo Cen, Zhiyong Qian, Haohao Dong, C. Alexander Valencia, Lunzhi Dai, Hoi Yee Chow, Lei Zhang, Biao Dong

Get full text

New Strategies in the Treatment of Ta-T1 Transitional Cell Carcinoma (TCC) of the Bladder by Maurizio A. Brausi

“…Also in the pipeline are new approaches to treatment such as gene therapy, but these will need to be developed much more before they become part of routine practice.…”
Get full text

Analysis of Wave Solutions of an Adhenovirus-Tumor Cell System by Baba Issa Camara, Houda Mokrani

“…We discuss biological background and mathematical analysis of glioma gene therapy for contributing to cancer treatment. By a reaction-diffusion system, we model interactions between gliom cells and viruses. …”
Get full text

Oncolytic Immunotherapy: Where Are We Clinically? by Akseli Hemminki

Get full text

High power impulse magnetron sputtering deposited virucide and antibacterial CrN-Cu films and the durability of the antimicrobial activity by Santiago Domínguez-Meister, Janire Mediavilla, Anastasia Dianova, Noelia Álvarez, Sara Insausti, Edurne Rujas, Jose L. Nieva, Marta Brizuela, Iñigo Braceras

Get full text

Branched endosomal disruptor (BEND) lipids mediate delivery of mRNA and CRISPR-Cas9 ribonucleoprotein complex for hepatic gene editing and T cell engineering by Marshall S. Padilla, Kaitlin Mrksich, Yiming Wang, Rebecca M. Haley, Jacqueline J. Li, Emily L. Han, Rakan El-Mayta, Emily H. Kim, Sofia Dias, Ningqiang Gong, Sridatta V. Teerdhala, Xuexiang Han, Vivek Chowdhary, Lulu Xue, Zain Siddiqui, Hannah M. Yamagata, Dongyoon Kim, Il-Chul Yoon, James M. Wilson, Ravi Radhakrishnan, Michael J. Mitchell

Get full text

Development of a cell-penetrating peptide-based nanocomplex for long-term delivery of intact mitochondrial DNA into epithelial cells by Kyrie Wilson, Charles Holjencin, Hwaran Lee, Balasubramaniam Annamalai, Masaaki Ishii, Jeremy L. Gilbert, Andrew Jakymiw, Bärbel Rohrer

“…Gene therapy approaches for mitochondrial DNA (mtDNA)-associated damage/diseases have thus far been limited, and despite advancements in single gene therapy for mtDNA mutations and progress in mitochondrial transplantation, no method exists for restoring the entire mtDNA molecule in a clinically translatable manner. …”
Get full text

Nucleic Acid-Based Therapy Approaches for Huntington's Disease by Tatyana Vagner, Deborah Young, Alexandre Mouravlev

“…Since the mutation has multiple effects in the cell and the precise mechanism of the disease remains to be elucidated, gene therapy approaches have been developed that intervene in different aspects of the condition. …”
Get full text

A novel platform for engineered AAV-based vaccines by Sabrina Babutzka, Miranda Gehrke, Anastasia Papadopoulou, Maria Diedrichs-Möhring, Maria Giannaki, Lena Hennis, Bastian Föhr, Cale Kooyman, Andreas Osterman, Evangelia Yannaki, Gerhild Wildner, Hermann Ammer, Stylianos Michalakis

“…Engineering of adeno-associated virus (AAV) capsids allowed for the development of gene therapy vectors with improved tropism and enhanced transduction efficiency. …”
Get full text

Adoptive Cell Therapy with Tumor-Infiltrating Lymphocytes in Advanced Melanoma Patients by Mélanie Saint-Jean, Anne-Chantal Knol, Christelle Volteau, Gaëlle Quéreux, Lucie Peuvrel, Anabelle Brocard, Marie-Christine Pandolfino, Soraya Saiagh, Jean-Michel Nguyen, Christophe Bedane, Nicole Basset-Seguin, Amir Khammari, Brigitte Dréno

Get full text

Genetics of Cerebral Vasospasm by Travis R. Ladner, Scott L. Zuckerman, J Mocco

“…In our review, we summarize the most recent research involving key areas within the genetics and vasospasm discussion: (1) Prognostic role of genetics—risk stratification based on gene sequencing, biomarkers, and polymorphisms; (2) Signaling pathways—pinpointing key inflammatory molecules responsible for downstream cellular signaling and altering these mediators to provide therapeutic benefit; and (3) Gene therapy and gene delivery—using viral vectors or novel protein delivery methods to overexpress protective genes in the vasospasm cascade.…”
Get full text

Restenosis and Therapy by Laszlo Denes, Laszlo Entz, Veronika Jancsik

“…Those could be useful markers of carotid restenosis after stent implantation. Gene therapy and therapeutic angiogenesis is proposed for therapy in restenosis. …”
Get full text

The perspectives of autologous hepatocytes generation for the treatment of liver failure by M. N. Evseeva, A. F. Sheptulina, Yu. P. Rubtsov

“…This review presents alternative approaches for liver failure treatment: inhibition of epithelial-mesenchymal transformation, e.g. by induction of proliferation (gene therapy with HGF), and by influence on immune imbalance (T-regulatory lymphocytes application).Conclusion. …”
Get full text

Innovative IOP-Independent Neuroprotection and Neuroregeneration Strategies in the Pipeline for Glaucoma by James C. Tsai

“…These innovative strategies in the pipeline include investigation of neurotrophic factors, gene therapy, immune system modulation, and novel neuroregeneration pathways. …”
Get full text