Gene therapy clinical trials worldwide to 2017: An update

Ginn, Samantha L., Amaya, Anais K., Alexander, Ian E., Edelstein, Michael, Abedi, Mohammad R.·2018·The Journal of Gene Medicine

Cited by (903)

Items 301–400 of 903

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    Irina Alekseevna Nedorubova, Tatiana Borisovna Bukharova, Victoria Mokrousova, Maria A. Khvorostina, Andrey Vyacheslavovich Vasilyev, Andrey Anatolevich Nedorubov, Timofei Evgenevich Grigoriev, Yu. D. Zagoskin, С. Н. Чвалун, Sergey Ivanovich Kutsev et al. · 2022 · International Journal of Molecular Sciences
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  4. Revisiting gene delivery to the brain: silencing and editing
    João Conniot, Sepehr Talebian, Susana M. N. Simões, Lino Ferreira, João Conde · 2020 · Biomaterials Science
  5. Understanding In Vivo Fate of Nucleic Acid and Gene Medicines for the Rational Design of Drugs
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  6. Transdermal gene delivery
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    Sachin R. Chaugule, Christodoulos K. Constantinou, Aijaz Ahmad John, Dimitra Micha, Elisabeth M. W. Eekhoff, Ellen M. Gravallese, Guangping Gao, Jae‐Hyuck Shim · 2025 · Human Gene Therapy
  8. Gene Delivery by PAMAM Dendrimer Conjugated with the Nuclear Localization Signal Peptide Derived from Influenza B Virus Nucleoprotein
    Jeil Lee, Seulgi Lee, Yong‐Eun Kwon, Youn‐Joong Kim, Joon Sig Choi · 2019 · Macromolecular Research
  9. National guidelines for gene therapy product (2019)
    Kiruthika Sivagourounadin, Mirunalini Ravichandran, Priyadharsini Rajendran · 2021 · Perspectives in Clinical Research
  10. Targeting the lung epithelium after intravenous delivery by directed evolution of underexplored sites on the AAV capsid
    David Goertsen, Nick Goeden, Nicholas C. Flytzanis, Viviana Gradinaru · 2022 · Molecular Therapy — Methods & Clinical Development
  11. Unravelling Receptor and RGD Motif Dependence of Retargeted Adenoviral Vectors using Advanced Tumor Model Systems
    Mariya Chernyavska, Markus Schmid, Patrick C. Freitag, Valentina Palacio-Castañeda, Aigars Piruska, Wilhelm T. S. Huck, Andreas Plückthun, Wouter P. R. Verdurmen · 2019 · Scientific Reports
  12. Polyamidoamine Dendrimer Grafted with an Acid-Responsive Charge-Reversal Layer for Improved Gene Delivery
    Juan Wang, Remy C. Cooper, Hu Yang · 2020 · Biomacromolecules
  13. CRISPR-Associated (CAS) Effectors Delivery via Microfluidic Cell-Deformation Chip
    Noshad Peyravian, Maziar Malekzadeh Kebria, Jafar Kiani, Peiman Brouki Milan, Masoud Mozafari · 2021 · Materials
  14. Selective sodium iodide symporter (NIS) gene therapy of glioblastoma mediated by EGFR-targeted lipopolyplexes
    Rebekka Spellerberg, Teoman Benli‐Hoppe, Carolin Kitzberger, Simone Berger, Kathrin A. Schmohl, Nathalie Schwenk, Hsi-Yu Yen, Christian Zach, Franz Schilling, Wolfgang Weber et al. · 2021 · Molecular Therapy — Oncolytics
  15. A Decade of Progress in Gene Targeted Therapeutic Strategies in Duchenne Muscular Dystrophy: A Systematic Review
    Lam Chung Liang, Nadiah S Sulaiman, Muhammad Dain Yazid · 2022 · Frontiers in Bioengineering and Biotechnology
  16. Gene Therapy for Retinal Diseases
    N. Samiy · 2014 · Journal of Ophthalmic & Vision Research
  17. Antibody-targeted chromatin enables effective intracellular delivery and functionality of CRISPR/Cas9 expression plasmids
    Tobias Killian, Annette Buntz, Teresa Herlet, Heike Seul, Olaf Mundigl, Gernot Längst, Ulrich Brinkmann · 2019 · Nucleic Acids Research
  18. Increased gene delivery efficiency and specificity of a lipid-based nanosystem incorporating a glycolipid
    Henrique Faneca, Dina Farinha, Maria C. Pedroso de Lima, Mariana Magalhães · 2014 · International Journal of Nanomedicine
  19. The role of small molecules in cell and gene therapy
    Lewis L. Brayshaw, Carlos Martinez-Fleites, Takis Athanasopoulos, Thomas D. Southgate, Laurent S. Jespers, Christopher Herring · 2020 · RSC Medicinal Chemistry
  20. MicroRNAs as therapeutic targets in breast cancer metastasis
    Fahima Danesh Pouya, Yousef Rasmi, Maria Gazouli, Eleni Zografos, Mohadeseh Nemati · 2021 · Drug Delivery and Translational Research
  21. Current therapies and therapeutic decision making for childhood‐onset movement disorders
    Shekeeb S. Mohammad, Simon Paget, Russell Clive Dale · 2019 · Movement Disorders
  22. Advances in Targeted Gene Delivery
    Anjuman Ara Begum, István Tóth, Waleed M. Hussein, Peter Michael Moyle · 2019 · Current Drug Delivery
  23. Versatile cationic liposomes for RIP3 overexpression in colon cancer therapy and RIP3 downregulation in acute pancreatitis therapy
    Lijing Zhang, Simeng Liu, Huimin Liu, Chengli Yang, Ai‐Ling Jiang, Heng Wei, Dan Sun, Zheng Cai, Yu Zheng · 2019 · Journal of drug targeting
  24. Tuning the Topological Landscape of DNA–Cyclodextrin Nanocomplexes by Molecular Design
    Tania Neva, Ana I. Carbajo‐Gordillo, Juan Manuel Benito, Hugo Lana, Gema Marcelo, Carmen Ortiz Mellet, Conchita Tros de Ilarduya, Francisco Mendicuti, José Manuel García Fernández · 2020 · Chemistry - A European Journal
  25. Development of Theranostic Cationic Liposomes Designed for Image-Guided Delivery of Nucleic Acid
    Hai Doan Do, Christine Ménager, Aude Michel, Johanne Séguin, Tawba Korichi, Hélène Dhôtel, Corinne Marie, Bich‐Thuy Doan, Nathalie Mignet · 2020 · Pharmaceutics
  26. Could gene therapy cure HIV?
    Mohsen Sheykhhasan, Aidin Foroutan, Hamed Manoochehri, Saeideh Gholamzadeh Khoei, Naresh Poondla, Massoud Saidijam · 2021 · Life Sciences
  27. Genome-based therapeutic interventions for β-type hemoglobinopathies
    Kariofyllis Karamperis, Maria T. Tsoumpeli, Fotios Kounelis, Maria Koromina, Christina Mitropoulou, Cátia Moutinho, George P. Patrinos · 2021 · Human Genomics
  28. A high-content image-based drug screen of clinical compounds against cell transmission of adenovirus
    Fanny Georgi, Fabien Kuttler, Luca Murer, Vardan Andriasyan, Robert Witte, Artur Yakimovich, Gerardo Turcatti, Urs F. Greber · 2020 · Scientific Data
  29. Unleashing the potential of mRNA therapeutics for inherited neurological diseases
    Edoardo Monfrini, Giacomo Baso, Dario Ronchi, Megi Meneri, Delia Gagliardi, Lorenzo Quetti, Federico Verde, Nicola Ticozzi, Antonia Ratti, Alessio Di Fonzo et al. · 2024 · Brain
  30. Characterization of iRGD-Ligand Modified Arginine-Histidine-Rich Peptides for Nucleic Acid Therapeutics Delivery to αvβ3 Integrin-Expressing Cancer Cells
    Anna Egorova, A. V. Selutin, Marianna A. Maretina, S. А. Selkov, В. С. Баранов, Anton Kiselev · 2020 · Pharmaceuticals
  31. Peptides-based therapy and diagnosis. Strategies for non-invasive therapies in cancer
    Corina Ciobănaşu · 2021 · Journal of drug targeting
  32. Nonreplicating Adenoviral Vectors: Improving Tropism and Delivery of Cancer Gene Therapy
    Nayara Gusmão Tessarollo, Ana Carolina Martins Domingues, Fernanda Antunes, Jean Carlos dos Santos da Luz, Otavio Augusto Rodrigues, Otto Luiz Dutra Cerqueira, Bryan Eric Strauss · 2021 · Cancers
  33. Controlling CRISPR with small molecule regulation for somatic cell genome editing
    Namita Khajanchi, Krishanu Saha · 2021 · Molecular Therapy
  34. The use of melittin to enhance transgene expression mediated by recombinant adeno-associated virus serotype 2 vectors both in vitro and in vivo
    Yilin Xie, Jiyao Wang, Yun He, Xiao-min Yu, Qingyun Zheng, Chen Ling, Xi-lin Feng, Liqing Zhu · 2022 · Journal of Chinese Integrative Medicine
  35. Peptide-Based Nanoparticles for αvβ3 Integrin-Targeted DNA Delivery to Cancer and Uterine Leiomyoma Cells
    Anna Egorova, A. V. Selutin, Marianna A. Maretina, S. А. Selkov, Anton Kiselev · 2022 · Molecules
  36. Delivery of CRISPR/Cas9 Plasmid DNA by Hyperbranched Polymeric Nanoparticles Enables Efficient Gene Editing
    Kemao Xiu, Laura Saunders, Luan Wen, Jinxue Ruan, Ruonan Dong, Jun Whi Song, Dongshan Yang, Jifeng Zhang, Jie Xu, Y. Eugene Chen et al. · 2022 · Cells
  37. Therapeutic Controlled Release Strategies for Human Osteoarthritis
    Dan Wang, Wei Liu, J. Venkatesan, H. Madry, M. Cucchiarini · 2024 · Advanced Healthcare Materials
  38. Strategies for Modifying Adenoviral Vectors for Gene Therapy
    Anna Muravyeva, Svetlana Smirnikhina · 2024 · International Journal of Molecular Sciences
  39. Human artificial chromosomes for pluripotent stem cell-based tissue replacement therapy
    Sergey A. Sinenko, Sergey V. Ponomartsev, Alexey N. Tomilin · 2020 · Experimental Cell Research
  40. Transferrin Receptor Targeted Polyplexes Completely Comprised of Sequence‐Defined Components
    Teoman Benli‐Hoppe, Şurhan Göl Öztürk, Özgür Öztürk, Simone Berger, Ernst Wagner, Mina Yazdi · 2021 · Macromolecular Rapid Communications
  41. Non-Viral Targeted Nucleic Acid Delivery: Apply Sequences for Optimization
    Yanfang Wang, Ernst Wagner · 2020 · Pharmaceutics
  42. Stem cell-based therapy for hirschsprung disease, do we have the guts to treat?
    Ali Fouad Alhawaj · 2021 · Gene Therapy
  43. Bacteria-Based Microdevices for the Oral Delivery of Macromolecules
    Zhenping Cao, Sisi Lin, Jinyao Liu · 2021 · Pharmaceutics
  44. An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy
    Yueyi Li, Hongyu Zeng, Yuhao Wei, Xuelei Ma, Zhiyao He · 2023 · Human Gene Therapy
  45. Synthetic cytotoxic drugs as cancer chemotherapeutic agents
    Ranjit Singh, Anjleena Malhotra, Ranju Bansal · 2023 · Elsevier eBooks
  46. Viral Vectors in Gene Replacement Therapy
    Ekaterina Minskaia, Alima Galieva, Alexander D. Egorov, Roman A. Ivanov, Alexander V. Karabelsky · 2023 · Biochemistry (Moscow)
  47. Effect of Diphtheria Toxin-Based Gene Therapy for Hepatocellular Carcinoma
    Kenya Kamimura, Takeshi Yokoo, Hiroyuki Abé, Norihiro Sakai, Takuro Nagoya, Yuji Kobayashi, Masato Ohtsuka, Hiromi Miura, Akira Sakamaki, Hiroteru Kamimura et al. · 2020 · Cancers
  48. Harnessing the Natural Biology of Adeno-Associated Virus to Enhance the Efficacy of Cancer Gene Therapy
    J. Bower, Liu-Jiang Song, P. Bastola, Matthew L. Hirsch · 2021 · Viruses
  49. The audiogram: Detection of pure-tone stimuli in ototoxicity monitoring and assessments of investigational medicines for the inner ear
    Colleen G. Le Prell, Carmen C. Brewer, Kathleen C. M. Campbell · 2022 · The Journal of the Acoustical Society of America
  50. Photoacoustic transfection of DNA encoding GFP
    Alexandre D. Silva, Carlos Serpa, Luı́s G. Arnaut · 2019 · Scientific Reports
  51. Laser‐assisted nucleic acid delivery: A systematic review
    Sepanta Hosseinpour, Laurence James Walsh · 2020 · Journal of Biophotonics
  52. Overcoming the barriers to optimization of adenovirus delivery using biomaterials: Current status and future perspective
    Dayananda Kasala, JinWoo Hong, Chae‐Ok Yun · 2021 · Journal of Controlled Release
  53. Influence of the media ionic strength on the formation and in vitro biological performance of polycation-DNA complexes
    Filipe Coelho, Cláudia Botelho, Juan Luis Paris, Eduardo Figueira Marques, Bruno F. B. Silva · 2021 · Journal of Molecular Liquids
  54. Gene Therapy for Acute Respiratory Distress Syndrome
    Jing Liu, David A. Dean · 2022 · Frontiers in Physiology
  55. Gene therapy for kidney disease: targeting cystinuria
    Jennifer L. Peek, Matthew H. Wilson · 2021 · Current Opinion in Nephrology & Hypertension
  56. Designing Synthetic Polymers for Nucleic Acid Complexation and Delivery: From Polyplexes to Micelleplexes to Triggered Degradation
    Le Zhou, Miracle Emenuga, Shreya Kumar, Zachary E. Lamantia, Marxa Leão Figueiredo, Todd S. Emrick · 2022 · Biomacromolecules
  57. Introduction to polymeric nanomaterials
    Kuruvalli Gouthami, Lavanya Lakshminarayana, Basheerabegum Faniband, Vishnu Priya Veeraraghavan, Muhammad Bilal, Ram Naresh Bhargava, Luiz Fernando Romanholo Ferreira, Abbas Rahdar, Siddappa Kakkameli, Sikandar Imamsab Mulla · 2023 · Elsevier eBooks
  58. Exploitation of De Novo Helper-Lipids for Effective Gene Delivery.
    Tomoaki Kurosaki, Takashi Kitahara, Mugen Teshima, Koyo Nishida, Junzo Nakamura, Mikiro Nakashima, Hideto To, Hiromitsu Hukuchi, Tomoyuki Hamamoto, Hitoshi Sasaki · 2009 · Journal of Pharmacy & Pharmaceutical Sciences
  59. Gene transfer to skeletal muscle using hydrodynamic limb vein injection: current applications, hurdles and possible optimizations
    Yann Thierry Le Guen, Tony Le Gall, Patrick Midoux, Philippe Guégan, Serge Braun, Tristan Montier · 2019 · The Journal of Gene Medicine
  60. Fundoscopy-directed genetic testing to re-evaluate negative whole exome sequencing results
    Ahra Cho, José Ronaldo Lima de Carvalho, Akemi J. Tanaka, Ruben Jauregui, Sarah R. Levi, Alexander G. Bassuk, Vinit B. Mahajan, Stephen H. Tsang · 2020 · Orphanet Journal of Rare Diseases
  61. Construction and Characterization of a Novel Recombinant Attenuated and Replication-Deficient Candidate Human Adenovirus Type 3 Vaccine: “Adenovirus Vaccine Within an Adenovirus Vector”
    Yuqian Yan, Shuping Jing, Liqiang Feng, Jing Zhang, Zhiwei Zeng, Min Li, Shan Zhao, Junxian Ou, Wendong Lan, Wenyi Guan et al. · 2020 · Virologica Sinica
  62. Exosomes in Dermatology: Emerging Roles in Skin Health and Disease
    Salman Bin Dayel, Ramadan Hussein · 2025 · Pharmaceutics
  63. Biosafety Practices for In Vivo Viral-Mediated Gene Therapy in the Health Care Setting
    Jill E Blind, E. Nicole McLeod, Alex M. Brown, Hinal Patel, Sumit Ghosh · 2020 · Applied Biosafety
  64. Genetic therapies for neurological disorders
    Laura FitzPatrick, Adrian Peter Bird · 2021 · Human Genetics
  65. When does precision matter? Personalized medicine from the perspective of public health
    James Wilson · 2022 · Oxford University Press eBooks
  66. Reversible Assembly of an Artificial Protein Nanocage Using Alkaline Earth Metal Ions
    Naoya Ohara, Norifumi Kawakami, Ryoichi Arai, Naruhiko Adachi, Toshio Moriya, Masato Kawasaki, Kenji Miyamoto · 2022 · Journal of the American Chemical Society
  67. Stimuli‐Responsive Hydrogel Microcapsules Harnessing the COVID‐19 Immune Response for Cancer Therapeutics
    Amit Fischer, Avner Ehrlich, Yevgeni Plotkin, Yu Ouyang, Klil Asulin, Ioannidis Konstantinos, Chunhai Fan, Yaakov Nahmias, Itamar Willner · 2023 · Angewandte Chemie International Edition
  68. Optimization and physicochemical characterization of a cationic lipid-phosphatidylcholine mixed emulsion formulated as a highly efficient vehicle that facilitates adenoviral gene transfer
    Soo-Yeon Kim, Sang Jin Lee, Jin‐Ki Kim, Han‐Gon Choi, Soo‐Jeong Lim · 2017 · International Journal of Nanomedicine
  69. State of art and limitations in genetic engineering to induce stable chondrogenic phenotype
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  70. Overexpression of TG2 enhances the differentiation of ectomesenchymal stem cells into neuron‑like cells and promotes functional recovery in adult rats following spinal cord injury
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  71. Overexpression of ER Protein Processing and Apoptosis Regulator Genes in Human Embryonic Kidney 293 Cells Improves Gene Therapy Vectors Production
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  72. A quantitative PCR screening method for adeno‐associated viral vector 2‐mediated gene doping
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  73. Spermine-modified Antheraea pernyi silk fibroin as a gene delivery carrier
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  74. Glycosylated carriers for cell-selective and nuclear delivery of nucleic acids
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  75. Gene Therapy for Pancreatic Diseases: Current Status
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  76. Correction of the pathogenic mutation in TGM1 gene by adenine base editing in mutant embryos
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  77. Matrix Mediated Viral Gene Delivery: A Review
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  78. Therapeutic angiogenesis in coronary artery disease: a review of mechanisms and current approaches
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  79. Characterization of integration frequency and insertion sites of adenovirus DNA into mouse liver genomic DNA following intravenous injection
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  80. Protection is not always a good thing: The immune system’s impact on gene therapy
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  81. Interleukin-24-mediated antitumor effects against human glioblastoma via upregulation of P38 MAPK and endogenous TRAIL-induced apoptosis and LC3-II activation-dependent autophagy
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  82. Acoustically targeted noninvasive gene therapy in large brain volumes
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  83. Nanocarrier-based gene delivery for immune cell engineering
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  85. Innovative Strategy for 3D Transfection of Primary Human Stem Cells with BMP-2 Expressing Plasmid DNA: A Clinically Translatable Strategy for Ex Vivo Gene Therapy
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  86. Cellular Metrology: Scoping for a Value Proposition in Extra- and Intracellular Measurements
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  87. Autophagy inhibition changes the disposition of non-viral gene carriers during blood-brain barrier penetration and enhances TRAIL-induced apoptosis in brain metastatic tumor
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  88. Capsid-like biodegradable poly-glycolic acid nanoparticles for a long-time release of nucleic acid molecules
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  89. Continuous countercurrent chromatographic twin‐column purification of oligonucleotides: The role of the displacement effect
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  90. Well-Defined pH-Responsive Self-Assembled Block Copolymers for the Effective Codelivery of Doxorubicin and Antisense Oligonucleotide to Breast Cancer Cells
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  91. Reduction-responsive cross-linked stearyl peptide for effective delivery of plasmid DNA
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  92. Protein-based vehicles for biomimetic RNAi delivery
    Alex Eli Pottash, Christopher J. Kuffner, Madeleine Noonan-Shueh, Steven M. Jay · 2019 · Journal of Biological Engineering
  93. A virus-free vector for the transfection of somatic cells to obtain IPSC
    Hanife Sevgi Varlı, Funda Alkan, Murat Demirbilek, Nelisa Türkoğlu · 2019 · Journal of Nanoparticle Research
  94. Layer‐by‐Layer‐Assembled Capsule Size Affects the Efficiency of Packaging and Delivery of Different Genetic Cargo
    Yana V. Tarakanchikova, Albert Radikovich Muslimov, Mikhail Valeryevich Zyuzin, Irina A. Nazarenko, Alexander S. Timin, Gleb B. Sukhorukov, Kirill Viktorovich Lepik · 2020 · Particle & Particle Systems Characterization
  95. Unlocking the potential: advancements and applications of gene therapy in severe disorders
    Rahul G. Ingle, Gehan M. Elossaily, Mohd Nazam Ansari, Shivani Makhijani · 2025 · Annals medicus
  96. Assembly of Building Blocks by Double-End-Anchored Polymers in the Dilute Regime Mediated by Hydrophobic Interactions at Controlled Distances
    Emily A. Wonder, Kai K. Ewert, Chenyu Liu, Victoria M. Steffes, Jasmin Kwak, Vikar Qahar, Ramsey N. Majzoub, Zhening Zhang, Bridget O Carragher, Clinton S. Potter et al. · 2020 · ACS Applied Materials & Interfaces
  97. Engineered AAV8 capsid acquires heparin and AVB sepharose binding capacity but has altered in vivo transduction efficiency
    L. van Lieshout, A. Stegelmeier, Tara N Rindler, John J Lawder, Debra L. Sorensen, Kathy L. Frost, S. Booth, James P. Bridges, S. Wootton · 2020 · Gene Therapy
  98. Advances in Development of mRNA-Based Therapeutics
    Lei Huang, Luyao Zhang, Weiwei Li, Shiqiang Li, Jianguo Wen, Hangwen Li, Zhongmin Liu · 2020 · Current topics in microbiology and immunology
  99. Improvement of DNA Vector Delivery of DOTAP Lipoplexes by Short-Chain Aminolipids
    Jonas Buck, Dennis Mueller, Ute Mettal, Miriam Ackermann, Hiu Man Grisch‐Chan, Beat Thöny, Andreas Zumbuehl, Jörg Huwyler, Dominik Witzigmann · 2020 · ACS Omega
  100. Aptamer-Functionalized Natural Protein-Based Polymers as Innovative Biomaterials
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