Gene therapy clinical trials worldwide to 2017: An update

Cited by (903)

Items 101–200 of 903

1. Challenges of gene delivery to the central nervous system and the growing use of biomaterial vectors
   Devan L. Puhl, Anthony R. D’Amato, Ryan J. Gilbert · 2019 · Brain Research Bulletin
2. Pleiotropy or linkage? Their relative contributions to the genetic correlation of quantitative traits and detection by multitrait GWA studies
   Jobran Chebib, Frédéric Guillaume · 2021 · Genetics
3. Nucleic Acid-Based Approaches for Tumor Therapy
   Simone Hager, Frederic Julien Fittler, Ernst Wagner, Matthias Bros · 2020 · Cells
4. AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A
   Benoît Gautier, Hélène Hajjar, Sylvia Soares, Jade Berthelot, Marie Deck, Scarlette Abbou, Graham R. Campbell, María Ceprián, Sergio Gonzalez, Claire-Maëlle Fovet et al. · 2021 · Nature Communications
5. Cell and gene therapy for kidney disease
   Jennifer L. Peek, Matthew H. Wilson · 2023 · Nature Reviews Nephrology
6. Development of Large-Scale Downstream Processing for Lentiviral Vectors
   Anniina J. Valkama, Igor Oruetxebarria, Eevi M. Lipponen, Hanna M. Leinonen, Piia Käyhty, Heidi Hynynen, Vesa Turkki, Joonas Malinen, Tuukka Miinalainen, Tommi Heikura et al. · 2020 · Molecular Therapy — Methods & Clinical Development
7. Neuronal Ceroid Lipofuscinosis: Potential for Targeted Therapy
   Nicola Specchio, Alessandro Ferretti, Marina Trivisano, Nicola Pietrafusa, Chiara Pepi, Costanza Calabrese, Susanna Livadiotti, Alessandra Simonetti, Paolo Rossi, Paolo Curatolo et al. · 2020 · Drugs
8. Lipid-nucleic acid nanoparticles of novel ionizable lipids for systemic BMP-9 gene delivery to bone-marrow mesenchymal stem cells for osteoinduction
   Imran Vhora, Rohan Lalani, Priyanka Bhatt, Sushilkumar Patil, Ambikanandan R. Misra · 2019 · International Journal of Pharmaceutics
9. Lentiviral vector bioprocess economics for cell and gene therapy commercialization
   Ruxandra-Maria Comisel, Bo Kara, Frederick H. Fiesser, Suzanne S. Farid · 2020 · Biochemical Engineering Journal
10. Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases
    Della Grace Thomas Parambi, Khalid Saad Alharbi, Rajesh Kumar, Seetha Harilal, Gaber El‐Saber Batiha, Natália Alves Martins, Omnia Magdy Hendawy, Arafa Musa, Dibya Sundar Panda, Bijo Mathew · 2021 · Molecular Neurobiology
11. Gene Therapy Targeting p53 and KRAS for Colorectal Cancer Treatment: A Myth or the Way Forward?
    Hidayati Husainy Hasbullah, Marahaini Musa · 2021 · International Journal of Molecular Sciences
12. Exploring modified chitosan-based gene delivery technologies for therapeutic advancements
    Amol Dilip Gholap, Harshad S. Kapare, Sakshi Pagar, Pallavi A. Kamandar, Deblina D. Bhowmik, Nikhar Vishwakarma, Sarjana Raikwar, Atul Garkal, Tejal A Mehta, Satish V. Rojekar et al. · 2024 · International Journal of Biological Macromolecules
13. Nonviral Gene Therapy for Cancer: A Review
    Chiaki Hidai, Hisataka Kitano · 2018 · Diseases
14. Bacteria and cancer: Different sides of the same coin
    Ghazaleh Laliani, Shokufeh Ghasemian Sorboni, Roya Lari, Atieh Yaghoubi, Saman Soleimanpour, Majid Khazaei, Seyed Mahdi Hasanian, Amir Avan · 2020 · Life Sciences
15. Bioluminescence Imaging Applications in Cancer: A Comprehensive Review
    Nour AlSawaftah, Afifa Farooq, Salam Dhou, Amin F. Majdalawieh · 2020 · IEEE Reviews in Biomedical Engineering
16. Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB
    Seigo Kimura, Hideyoshi Harashima · 2020 · Pharmaceutics
17. New Insights into Cell Culture Technology
    Sivakumar JT Gowder · 2017 · InTech eBooks
18. Preparation, characterization, and transfection efficiency of low molecular weight polyethylenimine-based nanoparticles for delivery of the plasmid encoding CD200 gene
    Fatemeh Nouri, Hossein Sadeghpour, Reza Heidari, Ali Dehshahri · 2017 · International Journal of Nanomedicine
19. MnCaCs-Biomineralized Oncolytic Virus for Bimodal Imaging-Guided and Synergistically Enhanced Anticancer Therapy
    Lili Huang, Xue Li, Jinfeng Zhang, Qian Ru Zhao, Ming Jing Zhang, An‐An Liu, Dai‐Wen Pang, Hai‐Yan Xie · 2019 · Nano Letters
20. Cellular pathways of recombinant adeno-associated virus production for gene therapy
    Sha, Sha, Maloney, Andrew J., Katsikis, Georgios, Nguyen, Tam N.T., Neufeld, Caleb, Wolfrum, Jacqueline, Barone, Paul W., Springs, Stacy L., Manalis, Scott R., Sinskey, Anthony J. et al. · 2021 · Biotechnology Advances
21. Advances in RNA-based therapeutics: current breakthroughs, clinical translation, and future perspectives
    Sarbjeet Kaur Makkar · 2025 · Frontiers in Genetics
22. Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?
    Wagner, Dimitrios Laurin, Peter, Lena, Schmueck-Henneresse, Michael · 2021 · Gene Therapy
23. The era of gene therapy: From preclinical development to clinical application
    Nabil A. Alhakamy, David T. Curiel, Cory J. Berkland · 2021 · Drug Discovery Today
24. Chitosan applications in studying and managing osteosarcoma
    Parisa Maleki Dana, Jamal Hallajzadeh, Zatollah Asemi, Mohammad Alì Mansournia, Bahman Yousefi · 2020 · International Journal of Biological Macromolecules
25. An Update on the Application of CRISPR Technology in Clinical Practice
    Firouzeh Morshedzadeh, Mahmoud Ghanei, Malihe Lotfi, Morteza Ghasemi, Mohsen Ahmadi, Parisa Najari-Hanjani, Samaneh Sharif, S. Mozaffari-Jovin, M. Peymani, M. Abbaszadegan · 2023 · Molecular Biotechnology
26. Advances and prospects of RNA delivery nanoplatforms for cancer therapy
    Mohamed S. Attia, Gregor Stefan Kijanka, Nam‐Trung Nguyen, Jun Zhang, Hongjie An · 2024 · Acta Pharmaceutica Sinica B
27. Novel, rare and common pathogenic variants in the CFTR gene screened by high-throughput sequencing technology and predicted by in silico tools
    Stéphanie Villa-Nova Pereira, José Dirceu Ribeiro, Antônio Fernando Ribeiro, Carmen Sílvia Bertuzzo, Fernando Augusto Lima Marson · 2019 · Scientific Reports
28. The Horizon of Gene Therapy in Modern Medicine: Advances and Challenges
    Babak Arjmand, Bagher A. Larijani, Motahareh Sheikh Hosseini, Moloud Payab, Kambiz Gilany, Parisa Goodarzi, Peyvand Parhizkar Roudsari, Mobina Amanollahi, Negin Sadat Hosseini Mohammadi · 2019 · Advances in experimental medicine and biology
29. Achieving Spatial and Molecular Specificity with Ultrasound-Targeted Biomolecular Nanotherapeutics
    Jerzy O. Szablowski, Avinoam Bar‐Zion, Mikhail G. Shapiro · 2019 · Accounts of Chemical Research
30. Investigating the Impact of Delivery System Design on the Efficacy of Self-Amplifying RNA Vaccines
    Giulia Anderluzzi, Gustavo Lou, Simona Gallorini, Michela Brazzoli, Russell Johnson, Derek T. O’Hagan, Barbara C. Baudner, Yvonne Perrie · 2020 · Vaccines
31. Synthetically Engineered Adeno-Associated Virus for Efficient, Safe, and Versatile Gene Therapy Applications
    Margaret L. Lugin, Rebecca T. Lee, Young Jik Kwon · 2020 · ACS Nano
32. Non-Viral Vector-Mediated Gene Therapy for ALS: Challenges and Future Perspectives
    Gayathri R. Ediriweera, Liyu Chen, Justin J. Yerbury, Kristofer J. Thurecht, Kara L. Vine · 2021 · Molecular Pharmaceutics
33. Lipid-Nucleic Acid Complexes: Physicochemical Aspects and Prospects for Cancer Treatment
    Ricardo Gaspar, Filipe Coelho, Bruno F. B. Silva · 2020 · Molecules
34. Poly(ethylene glycol)–Poly(beta-amino ester)-Based Nanoparticles for Suicide Gene Therapy Enhance Brain Penetration and Extend Survival in a Preclinical Human Glioblastoma Orthotopic Xenograft Model
    Jayoung Kim, Sujan Kumar Mondal, Stephany Yi Tzeng, Yuan Rui, Rawan Al‐kharboosh, Kristen Kozielski, Adip Guruprasad Bhargav, César A. Garcia, Alfredo Quiñones‐Hinojosa, Jordan J. Green · 2020 · ACS Biomaterials Science & Engineering
35. Hydrogel-Based Localized Nonviral Gene Delivery in Regenerative Medicine Approaches—An Overview
    Natalia Carballo-Pedrares, Isaac Manuel Fuentes-Boquete, Silvia Díaz‐Prado, Ana Rey‐Rico · 2020 · Pharmaceutics
36. Nanofabrication based on DNA nanotechnology
    Yan Zhao, Xinpei Dai, Fei Wang, Xueli Zhang, Chunhai Fan, Xiaoguo Liu · 2019 · Nano Today
37. Novel insights into gene therapy in the cornea
    Rajiv R. Mohan, Lynn M. Martin, Nishant R. Sinha · 2020 · Experimental Eye Research
38. The Human Gene Editing Debate
    John H. Evans · 2020
39. Perspectives of Rare Disease Experts on Newborn Genome Sequencing
    Nina B. Gold, Sophia M. Adelson, Nidhi D Shah, Shardae Williams, Sarah L. Bick, Emilie S. Zoltick, Jessica I. Gold, Alanna Strong, Rebecca D. Ganetzky, Amy E. Roberts et al. · 2023 · JAMA Network Open
40. Harnessing the Therapeutic Potential of Biomacromolecules through Intracellular Delivery of Nucleic Acids, Peptides, and Proteins
    Yu Ping Tian, Matthew Tirrell, James L. LaBelle · 2022 · Advanced Healthcare Materials
41. History and current status of clinical studies using human pluripotent stem cells
    Sabine Kobold, Nils Bultjer, Glyn Stacey, Sabine C. Mueller, Armin Kurtz, Nancy Mah · 2023 · Stem Cell Reports
42. Gene-Editing Technologies Paired With Viral Vectors for Translational Research Into Neurodegenerative Diseases
    Joseph E. Rittiner, Malik Moncalvo, Ornit Chiba‐Falek, Boris Kantor · 2020 · Frontiers in Molecular Neuroscience
43. Multifunctional Natural Polymer Nanoparticles as Antifibrotic Gene Carriers for CKD Therapy
    Adam C. Midgley, Yongzhen Wei, Dashuai Zhu, Fangli Gao, Hongyu Yan, Anila Khalique, Wenya Luo, Huan Jiang, Xiangsheng Liu, Jiasen Guo et al. · 2020 · Journal of the American Society of Nephrology
44. Barriers and Opportunities for Implementation of Outcome-Based Spread Payments for High-Cost, One-Shot Curative Therapies
    Sissel Michelsen, Salma Nachi, Walter Van Dyck, Steven Simoens, Isabelle Huys · 2020 · Frontiers in Pharmacology
45. Effect of molecular weight of chitosan on the physicochemical, morphological, and biological properties of polyplex nanoparticles intended for gene delivery
    María Eugenia Aranda-Barradas, Saul E. Trejo-López, Alicia Del Real, Samuel Álvarez-Almazán, Abraham Méndez‐Albores, Carlos Gerardo García-Tovar, Francisco Rodolfo González-Díaz, Susana Patricia Miranda-Castro · 2022 · Carbohydrate Polymer Technologies and Applications
46. Design Strategies for Novel Lipid Nanoparticle for mRNA Vaccine and Therapeutics: Current Understandings and Future Perspectives
    Xiaochi Li, Junli Li, Jiazheng Wei, Weixin Du, Cheng Su, Xiaobin Shen, Aihua Zhao, Miao Xu · 2025 · MedComm
47. Recent advances in gene therapy for lysosomal storage disorders
    Andrea Amalfitano, David P. W. Rastall · 2015 · The Application of Clinical Genetics
48. Gene Replacement Therapy: A Primer for the Health-system Pharmacist
    John Petrich, Dominic Marchese, Chris Jenkins, Michael Storey, Jill E Blind · 2019 · Journal of Pharmacy Practice
49. Recent CPP-based applications in medicine
    Kaido Kurrikoff, Ülo Langel · 2019 · Expert Opinion on Drug Delivery
50. Genetic Hearing Loss and Gene Therapy
    Nathaniel T. Carpena, Min Young Lee · 2018 · Genomics & Informatics
51. Scaffold‐Based Delivery of Nucleic Acid Therapeutics for Enhanced Bone and Cartilage Repair
    Domhnall C. Kelly, Rosanne M. Raftery, Caroline M. Curtin, Caitríona M. O’Driscoll, Fergal J. O’Brien · 2019 · Journal of Orthopaedic Research®
52. Optimization of chitosan-based polyelectrolyte nanoparticles for gene delivery, using design of experiment: in vitro and in vivo study
    Masoumeh Baghaei, Farnaz Sadat Mirzazadeh Tekie, Mohammad Reza Khoshayand, Reyhaneh Varshochian, Maliheh Hajiramezanali, Marziye Javaheri Kachousangi, Rassoul Dinarvand, Fatemeh Atyabi · 2020 · Materials Science and Engineering C
53. Revisiting the role of pulsed electric fields in overcoming the barriers to in vivo gene electrotransfer
    Shaurya Sachdev, Tjaša Potočnik, Lea Rems, Damijan Miklavčič A · 2021 · Bioelectrochemistry
54. The Potential of Cell-Penetrating Peptides for mRNA Delivery to Cancer Cells
    Yelee Kim, Hyo‐Suk Kim, Eun Hye Kim, Hochung Jang, Yeongji Jang, Sung-Gil Chi, Yoosoo Yang, Sun Hwa Kim · 2022 · Pharmaceutics
55. Clinical applications of CRISPR‐based genome editing and diagnostics
    Dana Vera Foss, Megan L. Hochstrasser, Ross C. Wilson · 2019 · Transfusion
56. Roads Forward for European GMO Policy—Uncertainties in Wake of ECJ Judgment Have to be Mitigated by Regulatory Reform
    Martin S. Wasmer · 2019 · Frontiers in Bioengineering and Biotechnology
57. Cancer Chemotherapy via Natural Bioactive Compounds
    Kalyani Pathak, Manash Pratim Pathak, Riya Saikia, Urvashee Gogoi, Jon Jyoti Sahariah, James H. Zothantluanga, Abhishek Samanta, Aparoop Das · 2022 · Current Drug Discovery Technologies
58. Nanoplatform‐Based In Vivo Gene Delivery Systems for Cancer Therapy
    Rui Luo, Hao Le, Qinjie Wu, Changyang Gong · 2024 · Small
59. Influence of cationic lipid concentration on properties of lipid–polymer hybrid nanospheres for gene delivery
    Hansoo Park, Soo‐Hong Lee, Jong Chan Ahn, Jagathesh Chandra Bose Rajendiran, Yoshie Arai · 2015 · International Journal of Nanomedicine
60. Smart dendrimers: Synergizing the targeting of anticancer bioactives
    Vikrant Saluja, Abhinav Mankoo, Gaurav Kant Saraogi, Murtaza M. Tambuwala, Vijay Kumar Mishra · 2019 · Journal of Drug Delivery Science and Technology
61. Graphene oxide as a 2D platform for complexation and intracellular delivery of siRNA
    Irene de Lázaro, Sandra Vranic, Domenico Marson, Artur Filipe Rodrigues, Maurizio Buggio, Adrián Esteban-Arranz, Mariarosa Mazza, Paola Posocco, Kostas Kostarelos · 2019 · Nanoscale
62. Clinical translation of gene medicine
    Fazhan Wang, Zhou Qin, Hansi Lu, Siyan He, Jing Cong Luo, Chaohui Jin, Xiangrong Song · 2019 · The Journal of Gene Medicine
63. Synthetic Biology: Emerging Concepts to Design and Advance Adeno‐Associated Viral Vectors for Gene Therapy
    Hanna J. Wagner, Wilfried Weber, Martin Fussenegger · 2021 · Advanced Science
64. The effect of genetically modified platelet-derived growth factor-BB over-expressing mesenchymal stromal cells during core decompression for steroid-associated osteonecrosis of the femoral head in rabbits
    Roberto Alfonso Guzman, Masahiro Maruyama, Seyedsina Moeinzadeh, Elaine Lui, Ning Zhang, Hunter W. Storaci, Kaysie Tam, Ejun Elijah Huang, Takeshi Utsunomiya, Claire Rhee et al. · 2021 · Stem Cell Research & Therapy
65. Gene therapy for cystic fibrosis: Challenges and prospects
    Hongshu Sui, Xinghua Xu, Yanping Su, Zhaoqing Gong, Minhua Yao, Xiaocui Liu, Ting Zhang, Ziyao Jiang, Tianhao Bai, Junzuo Wang et al. · 2022 · Frontiers in Pharmacology
66. Advances on non-invasive physically triggered nucleic acid delivery from nanocarriers
    Hai Doan Do, Brice Martin Couillaud, Bich‐Thuy Doan, Yohann Corvis, Nathalie Mignet · 2018 · Advanced Drug Delivery Reviews
67. Interactions in DNA Condensation: An Important Factor for Improving the Efficacy of Gene Transfection
    Xuan Nie, Ze Zhang, Changhui Wang, Yunshan Fan, Qing-Yong Meng, Ye‐Zi You · 2018 · Bioconjugate Chemistry
68. Intellectual Disability and Potassium Channelopathies: A Systematic Review
    Miriam Kessi, Baiyu Chen, Jing Peng, Yulin Tang, Eleonore Olatoutou, Fang Ping He, Lifen Yang, Fei Yin · 2020 · Frontiers in Genetics
69. The Impact of Product and Process Related Critical Quality Attributes on Immunogenicity and Adverse Immunological Effects of Biotherapeutics
    Yi Wen, Vibha Jawa · 2020 · Journal of Pharmaceutical Sciences
70. Nano based-oncolytic viruses for cancer therapy
    Mobarakeh Ajam-Hosseini, Fatemeh Akhoondi, Mohammad Doroudian · 2023 · Critical Reviews in Oncology/Hematology
71. CRISPR in Public Health: The Health Equity Implications and Role of Community in Gene-Editing Research and Applications
    Andrew M. Subica · 2023 · American Journal of Public Health
72. Powerful anti-colon cancer effect of modified nanoparticle-mediated IL-15 immunogene therapy through activation of the host immune system
    Xiaoxiao Liu, Yanyan Li, Xiaodong Sun, Yagmur Muftuoglu, Bilan Wang, Ting Yu, Yuzhu Hu, Lu Ma, Mingli Xiang, Gang Feng Guo et al. · 2018 · Theranostics
73. Collagen scaffold-mediated delivery of NLC/siRNA as wound healing materials
    Özgül Tezgel, Nicholas Distasio, Valentina Laghezza Masci, Annarita Taddei, Anna Szarpak, Rachel Auzély‐Velty, Fabrice P. Navarro, Isabelle Texier · 2019 · Journal of Drug Delivery Science and Technology
74. Gene therapy targeting cardiac phosphoinositide 3-kinase (p110α) attenuates cardiac remodeling in type 2 diabetes
    Darnel Prakoso, Miles J. De Blasio, Mitchel Tate, Helen Kiriazis, Daniel G. Donner, Hongwei Qian, David M. Nash, Minh Deo, Kate L. Weeks, Laura J. Parry et al. · 2020 · American Journal of Physiology-Heart and Circulatory Physiology
75. Cancer gene therapy goes viral: viral vector platforms come of age
    Urban Bezeljak · 2022 · Radiology and Oncology
76. Chondroitin Sulfate Capsule System for Efficient and Secure Gene Delivery
    Tomoaki Kurosaki, Takashi Kitahara, Shintaro Fumoto, Koyo Nishida, Kayo Yamamoto, Hiroo Nakagawa, Yukinobu Kodama, Norihide Higuchi, Tadahiro Nakamura, Hitoshi Sasaki · 2010 · Journal of Pharmacy & Pharmaceutical Sciences
77. Positron emission tomography imaging of novel AAV capsids maps rapid brain accumulation
    Jai Woong Seo, Elizabeth S. Ingham, Lisa M. Mahakian, Spencer K. Tumbale, Bo Wu, Sadaf Aghevlian, Shahin Shams, Mo Baikoghli, Poorva Jain, Xiaozhe Ding et al. · 2020 · Nature Communications
78. Perspectives of using microRNA-loaded nanocarriers for epigenetic reprogramming of drug resistant colorectal cancers
    Olga A. Sukocheva, Junqi Liu, Неганова Маргарита Евгеньевна, Narasimha Murthy Beeraka, Yulia R. Aleksandrova, Prasath Manogaran, Ekaterina Grigorevskikh, Vladimir N. Chubarev, Ruitai Fan · 2022 · Seminars in Cancer Biology
79. Copolymer micelles function as pH-responsive nanocarriers to enhance the cytotoxicity of a HER2 aptamer in HER2-positive breast cancer cells
    Yinxing Shen, Junqi Zhang, Weiju Hao, Tong Wang, Jing Liu, Youhua Xie, Shouhong Xu, Honglai Liu · 2018 · International Journal of Nanomedicine
80. Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity
    Mickaël Guilbaud, Marie Devaux, Celia Couzinié, Johanne Le Duff, Alice Toromanoff, Céline Vandamme, Nicolas Jaulin, Gwladys Gernoux, Thibaut Larcher, Philippe Moullier et al. · 2019 · Human Gene Therapy
81. Gene Therapy for Liver Cancers: Current Status from Basic to Clinics
    Kenya Kamimura, Takeshi Yokoo, Hiroyuki Abé, Shuji Terai · 2019 · Cancers
82. The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous System
    Françoise Piguet, Timothée De Saint-Denis, Emilie Audouard, Kévin Beccaria, Arthur André, Guillaume Wurtz, Raphael Schatz, Sandro Alves, Caroline Sevin, Michel Zérah et al. · 2020 · Human Gene Therapy
83. HIV-based lentiviral vectors: Origin and sequence differences
    Nathan M. Johnson, Anna Francesca Alvarado, Trey N. Moffatt, Joshua M. Edavettal, Tarun A. Swaminathan, Stephen E. Braun · 2021 · Molecular Therapy — Methods & Clinical Development
84. Advances in the treatment of neuronal ceroid lipofuscinosis
    Jonathan B. Rosenberg, Alvin Chen, Stephen M. Kaminsky, Ronald G. Crystal, Dolan Sondhi · 2019 · Expert Opinion on Orphan Drugs
85. Ligand targeting and peptide functionalized polymers as non-viral carriers for gene therapy
    Khan Muhammad, Jing Zhao, Ihsan Ullah, Jintang Guo, Xiang‐Kui Ren, Yakai Feng · 2019 · Biomaterials Science
86. A few ethical issues in translational research for gene and cell therapy
    Luciana Riva, Carlo Petrini · 2019 · Journal of Translational Medicine
87. Cancer Nanomedicines in an Evolving Oncology Landscape
    Peng Guo, Jing Huang, Marsha A. Moses · 2020 · Trends in Pharmacological Sciences
88. Orthopaedic Gene Therapy
    Christopher H. Evans, Steve C. Ghivizzani, Paul D. Robbins · 2021 · JBJS Reviews
89. Gene Therapy for Cystic Fibrosis Lung Disease: Overcoming the Barriers to Translation to the Clinic
    Martin Donnelley, David Parsons · 2018 · Frontiers in Pharmacology
90. Filling Adeno-Associated Virus Capsids: Estimating Success by Cryo-Electron Microscopy
    Subramanian, Suriyasri, Maurer, Anna C., Bator, Carol M., Makhov, Alexander M., Conway, James F., Turner, Kevin B., Marden, James H., Vandenberghe, Luk H., Hafenstein, Susan L. · 2019 · Human Gene Therapy
91. Emerging therapeutic targets for cerebral edema
    Ruchira Menka Jha, Sudhanshu P. Raikwar, Sandra Mihaljević, Amanda M. Casabella, Joshua S. Catapano, Anupama Rani, Shashvat M. Desai, Volodymyr Gerzanich, J. Marc Simard · 2021 · Expert Opinion on Therapeutic Targets
92. Optimizing Modified mRNA In Vitro Synthesis Protocol for Heart Gene Therapy
    Yoav Hadas, Nishat Sultana, Elias Youssef, Mohammad Tofael Kabir Sharkar, Keerat Kaur, Elena Chepurko, Lior Zangi · 2019 · Molecular Therapy — Methods & Clinical Development
93. Cationic dynamic covalent polymers for gene transfection
    Dandan Su, Maëva Coste, Andrei Diaconu, Mihail Dumitru Barboiu, Sébastien Ulrich · 2020 · Journal of Materials Chemistry B
94. Solely aqueous formulation of hydrophobic cationic polymers for efficient gene delivery
    Jana I. Solomun, Gizem Cinar, Prosper P. Mapfumo, Friederike Richter, Elisabeth Moek, Franziska Hausig, Liam Martin, Stephanie Hoeppener, Ivo Nischang, Anja Traeger · 2020 · International Journal of Pharmaceutics
95. Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates
    Douglas Ballon, Jonathan B. Rosenberg, Edward K. Fung, Anastasia Nikolopoulou, Paresh J. Kothari, Bishnu P. De, Bin He, Alvin Chen, Linda Heier, Dolan Sondhi et al. · 2020 · Human Gene Therapy
96. AAV and Compacted DNA Nanoparticles for the Treatment of Retinal Disorders: Challenges and Future Prospects
    Zongchao Han, Shannon M. Conley, Muna I. Naash · 2011 · Investigative Ophthalmology & Visual Science
97. Scaffold-Based Gene Therapeutics for Osteochondral Tissue Engineering
    Xin Yan, You‐Rong Chen, Yifan Song, Meng Yang, Jing Ye, Gang Zhou, Jia‐Kuo Yu · 2020 · Frontiers in Pharmacology
98. Peptides as a Material Platform for Gene Delivery: Emerging Concepts and Converging Technologies
    Morgan A. Urello, Wei‐Hsin Hsu, R. James Christie · 2020 · Acta Biomaterialia
99. The optimized drug delivery systems of treating cancer bone metastatic osteolysis with nanomaterials
    Xi Cheng, Jinrong Wei, Qi Ge, Danlei Xing, Xuefeng Zhou, Yunzhu Qian, Guoqin Jiang · 2020 · Drug Delivery
100. Functional tissue-engineered bone-like graft made of a fibrin scaffold and TG2 gene-modified EMSCs for bone defect repair
     Wentao Shi, Yunduan Que, Xuan Zhang, Lu Bian, Xuejian Yu, Xue Ming TANG, Guofeng Yang, Yao Dai, Shiqi Bi, Demin Lv et al. · 2021 · NPG Asia Materials