Advancements in the Treatment of Mucopolysaccharidoses: From Established Therapies to Gene Therapy by Joanna Wanat, Wojciech Homa, Aleksandra Warunek, Gabriela Gronowicz, Izabela Dzikowska, Agata Siejka, Daria Stefaniak, Weronika Zielińska, Michał Chról

Subjects: “…Gene therapy…”
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Gene therapy by hepatocyte growth factor results in regression of experimental liver fibrosis by N. A. Dzhoyashvili, N. I. Kalinina, I. B. Beloglazova, Z. I. Tsokolayeva, P. I. Makarevich, Yu. L. Perov, Ye. V. Parfenova, V. A. Tkachuk

“…Obtained results allow to consider gene therapy by hepatocyte growth factor in composition of non-viral vectors as one of perspective methods of treatment of chronic liver diseases.…”
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Gene Therapy: A step toward advanced orthodontics – A narrative review by Monika Shukla, Shalu Jain, C. Munish Reddy, Pradeep Raghav, Kumar Amit, Rinchin Yangzom, Shivika Garg, Aastha Kamrani

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Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations by Lucas Tricoli, Sunetra Sase, Julia L. Hacker, Vi Pham, Maxwell Chappell, Laura Breda, Stephanie N. Hurwitz, Naoto Tanaka, Carlo Castruccio Castracani, Amaliris Guerra, Zhongqi Hou, Lars Schlotawa, Karthikeyan Radhakrishnan, Matthew Hogenauer, Aoife Roche, John Everett, Frederic Bushman, Peter Kurre, Rebecca Ahrens-Nicklas, Laura A. Adang, Adeline L. Vanderver, Stefano Rivella

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Engineering a bacterial toxin deaminase from the DYW-family into a novel cytosine base editor for plants and mammalian cells by Dingbo Zhang, Fiona Parth, Laura Matos da Silva, Teng-Cheong Ha, Axel Schambach, Jens Boch

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Addressing the limitations of the regulatory landscape in South Africa regarding advanced cell and gene therapies and related sectors involving human cells, tissues and organs by I M Viljoen, M S Pepper

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Therapeutics in paediatric genetic diseases: current and future landscape by Ai Ling Koh, Saumya Shekhar Jamuar

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From bench to bedside: Developing CRISPR/Cas-based therapy for ocular diseases by Qing Zhao, Linxin Wei, Youxin Chen

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AAV-based vectors for human diseases modeling in laboratory animals by Timur I. Aliev, Dmitry V. Yudkin

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Recombinant AAV batch profiling by nanopore sequencing elucidates product-related DNA impurities and vector genome length distribution by Florian Dunker-Seidler, Kathrin Breunig, Magdalena Haubner, Florian Sonntag, Markus Hörer, Rebecca C. Feiner

Subjects: “…nanopore sequencing, gene therapy…”
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Genetic advancements in breast cancer treatment: a review by Marzieh Shokoohi, Sadaf Sedaghatshoar, Homaira Arian, Milad Mokarami, Fatemeh Habibi, Fatemeh Bamarinejad

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Clinically meaningful improvements after gene therapy for aromatic L-amino acid decarboxylase deficiency (AADCd) in the Peabody Developmental Motor Scale, Second Edition (PDMS-2) and correlation with Bayley-III scores and motor milestones by Wuh-Liang Hwu, Hui-Min Lee, John Devin Peipert, Rongrong Zhang, Christian Werner, J. Rafael Sierra, Thomas O’Connell, Jonathan J. Woolley, Marjorie Crowell, Antonia Wang, Ioannis Tomazos

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NPRL2 gene therapy induces effective antitumor immunity in KRAS/STK11 mutant anti-PD1 resistant metastatic non-small cell lung cancer (NSCLC) in a humanized mouse model by Ismail M Meraz, Mourad Majidi, Renduo Song, Feng Meng, Lihui Gao, Qi Wang, Jing Wang, Elizabeth J Shpall, Jack A Roth

Subjects: “…nprl2 gene therapy…”
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The Genetic Basis of Congenital Cataracts: Advances in Diagnostics and Therapeutics by Agnieszka Starzyk, Piotr Charzewski

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Therapeutic landscape for inherited ocular diseases: current and emerging therapies by Hwei Wuen Chan, Jaslyn Oh, Bart Leroy

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The oncolytic adenovirus TILT-123 with pembrolizumab in platinum resistant or refractory ovarian cancer: the phase 1a PROTA trial by Matthew Stephen Block, James Hugo Armstrong Clubb, Johanna Mäenpää, Santeri Pakola, Dafne Carolina Alves Quixabeira, Tatiana Kudling, Elise Jirovec, Lyna Haybout, Mirte van der Heijden, Sanae Zahraoui, Susanna Grönberg-Vähä-Koskela, Sini Raatikainen, Victor Arias, Saru Basnet, Nea Ojala, Teijo Pellinen, Annabrita Hemmes, Katja Välimäki, Annukka Pasanen, Tuomo Alanko, Daniel Adamo, Susan Ramadan, Jorma Sormunen, Juha Kononen, Julia Wanda Cohen, Michael Jon Chisamore, John Goldfinch, Suvi Sorsa, Riikka Havunen, Claudia Kistler, Aino Kalervo, Víctor Cervera-Carrascon, João Manuel dos Santos, Akseli Hemminki

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Ex vivo delivery of recombinant IL-10 to human donor lungs by Jonathan C. Yeung, Terumoto Koike, Dirk Wagnetz, Tiago N. Machuca, Riccardo Bonato, Mingyao Liu, Stephen Juvet, Marcelo Cypel, Shaf Keshavjee

“…However, the very short half-life of IL-10 in vivo (∼2 hours) has necessitated the use of gene therapy in almost all animal models of lung transplantation. …”
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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. …”
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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. …”
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