Featured
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Correction of a knock-in mouse model of acrodysostosis with gene therapy using a rAAV9-CAG-human PRKAR1A vector
- Yasemin Özgür-Günes
- , Catherine Le Stunff
- & Pierre Bougnères
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Article
| Open AccessNon-viral delivery of CRISPR–Cas9 complexes for targeted gene editing via a polymer delivery system
- Jonathan O’Keeffe Ahern
- , Irene Lara-Sáez
- & Wenxin Wang
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Perspective
| Open AccessPulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo
- Ewa Janosz
- , Miriam Hetzel
- & Thomas Moritz
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Article
| Open AccessGene therapy for spinal muscular atrophy: the Qatari experience
- Hossamaldein Gaber Ali
- , Khalid Ibrahim
- & Tawfeg Ben-Omran
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Perspective
| Open AccessThe Gibco™ CTS™ Rotea™ system story—a case study of industry-academia collaboration
- Anqi Li
- , David James
- & Rebecca Lim
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Chitosan-coated Zn-metal-organic framework nanocomposites for effective targeted delivery of LNA-antisense miR-224 to colon tumor: in vitro studies
- Negin Mokri
- , Zahra Sepehri
- & Mehrdad Hashemi
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Perspective
| Open AccessPrime editing – an update on the field
- Janine Scholefield
- & Patrick T. Harrison
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Article
| Open AccessRNAi and chemogenetic reporter co-regulation in primate striatal interneurons
- Walter Lerchner
- , Abdullah A. Adil
- & Barry J. Richmond
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Brief Communication
| Open AccessAdvantages of robotic assistance over a manual approach in simulated subretinal injections and its relevance for gene therapy
- Reza Ladha
- , Thijs Meenink
- & Marc D. de Smet
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Article |
Membrane-bound MMP-14 protease-activatable adeno-associated viral vectors for gene delivery to pancreatic tumors
- Susan S. Butler
- , Kenjiro Date
- & Junghae Suh
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Brief Communication
| Open AccessEnhanced CNS transduction from AAV.PHP.eB infusion into the cisterna magna of older adult rats compared to AAV9
- Diptaman Chatterjee
- , David J. Marmion
- & Jeffrey H. Kordower
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Review Article |
Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders
- Yan Chen
- , Ruiting Wen
- & Zhanghui Chen
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Article |
Fabrication of anionic dextran-coated micelles for aptamer targeted delivery of camptothecin and survivin-shRNA to colon adenocarcinoma
- Setareh Sanati
- , Sahar Taghavi
- & Mona Alibolandi
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Article
| Open AccessUse of CRISPR/Cas9-mediated disruption of CNS cell type genes to profile transduction of AAV by neonatal intracerebroventricular delivery in mice
- Tess Torregrosa
- , Sydney Lehman
- & Shih-Ching Lo
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Brief Communication |
AAV-mediated delivery of actoxumab and bezlotoxumab results in serum and mucosal antibody concentrations that provide protection from C. difficile toxin challenge
- Matthew M. Guilleman
- , Brenna A. Y. Stevens
- & Sarah K. Wootton
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Article
| Open AccessAAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1
- Burcak Ozes
- , Morgan Myers
- & Zarife Sahenk
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Article
| Open AccessHuman MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells
- Andrea J. Korecki
- , Jorge L. Cueva-Vargas
- & Elizabeth M. Simpson
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Article
| Open AccessTargeted multi-epitope switching enables straightforward positive/negative selection of CAR T cells
- Laura Mosti
- , Lukas M. Langner
- & Toni Cathomen
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Article
| Open AccessValidation of an IFN-gamma ELISpot assay to measure cellular immune responses against viral antigens in non-human primates
- Fan Yang
- , Kathryn Patton
- & Christian Vettermann
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Article |
FEN1 inhibitor synergizes with low-dose camptothecin to induce increased cell killing via the mitochondria mediated apoptotic pathway
- Ting Wu
- , Hongqiao Zhu
- & Zhigang Guo
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Brief Communication |
Molecular targeting of vulnerable RNA sequences in SARS CoV-2: identifying clinical feasibility
- G. Ian Gallicano
- , John L. Casey
- & Samiksha Mahapatra
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Article |
Sensitization of ON-bipolar cells with ambient light activatable multi-characteristic opsin rescues vision in mice
- Subrata Batabyal
- , Sivakumar Gajjeraman
- & Samarendra Mohanty
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Brief Communication |
Engineered AAV8 capsid acquires heparin and AVB sepharose binding capacity but has altered in vivo transduction efficiency
- Laura P. van Lieshout
- , Ashley A. Stegelmeier
- & Sarah K. Wootton
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Preconditioning of surgical pedicle flaps with DNA plasmid expressing hypoxia-inducible factor-1α (HIF-1α) promotes tissue viability
- Kai-Hua Chang
- , Pouria Shoureshi
- & John W. Harmon
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Article
| Open AccessThe stability of envelope-pseudotyped lentiviral vectors
- Iris J. C. Dautzenberg
- , Martijn J. W. E. Rabelink
- & Rob C. Hoeben
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Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery
- K. L. Pietersz
- , R. M. Martier
- & B. Blits
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Brief Communication |
Detection of non-targeted transgenes by whole-genome resequencing for gene-doping control
- Teruaki Tozaki
- , Aoi Ohnuma
- & Shun-ichi Nagata
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Brief Communication |
Sufficiency for inducible Caspase-9 safety switch in human pluripotent stem cells and disease cells
- Toshinobu Nishimura
- , Haojun Xu
- & Hiromitsu Nakauchi
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Review Article |
Gene therapy development in hearing research in China
- Zhen Zhang
- , Jiping Wang
- & Feng Liu
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Article
| Open AccessInvestigation of the safety and feasibility of AAV1/SERCA2a gene transfer in patients with chronic heart failure supported with a left ventricular assist device – the SERCA-LVAD TRIAL
- A. R. Lyon
- , D. Babalis
- & S. E. Harding
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Comment |
Large gene delivery to the retina with AAV vectors: are we there yet?
- Ivana Trapani
- , Patrizia Tornabene
- & Alberto Auricchio
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Brief Communication |
Effect of connective tissue growth factor gene editing using adeno-associated virus–mediated CRISPR–Cas9 on rabbit glaucoma filtering surgery outcomes
- Eun Jung Lee
- , Jong Chul Han
- & Changwon Kee
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Review Article |
An overview of development in gene therapeutics in China
- Dawei Wang
- , Kang Wang
- & Yujia Cai
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Review Article |
Recent development of AAV-based gene therapies for inner ear disorders
- Yiyang Lan
- , Yong Tao
- & Guisheng Zhong
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Article
| Open AccessNatural polyphenol assisted delivery of single-strand oligonucleotides by cationic polymers
- Wanwan Shen
- , Ruojun Wang
- & Yiyun Cheng
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Review Article |
Advances in genome editing: the technology of choice for precise and efficient β-thalassemia treatment
- Gibran Ali
- , Muhammad Akram Tariq
- & Javed Akram
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Article |
Optimizing lentiviral vector transduction of hematopoietic stem cells for gene therapy
- Yoonjeong Jang
- , Yoon-Sang Kim
- & Byoung Y. Ryu
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Comment
| Open AccessToward a new framework for the development of individualized therapies
- Peter Marks
- & Celia Witten
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Article |
AAV3-miRNA vectors for growth suppression of human hepatocellular carcinoma cells in vitro and human liver tumors in a murine xenograft model in vivo
- Ling Yin
- , Geoffrey D. Keeler
- & Arun Srivastava
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Particulate mediators of the bystander effect linked to suicide and interferon-β transgene expression in melanoma cells
- Lucrecia Agnetti
- , Chiara Fondello
- & Liliana M. E. Finocchiaro
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Article
| Open AccessUtility of microminipigs for evaluating liver-mediated gene expression in the presence of neutralizing antibody against vector capsid
- Ryota Watano
- , Tsukasa Ohmori
- & Hiroaki Mizukami
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Transposon-mediated generation of CAR-T cells shows efficient anti B-cell leukemia response after ex vivo expansion
- Leonardo Chicaybam
- , Luiza Abdo
- & Martín Hernán Bonamino
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Heat-shrinking DNA nanoparticles for in vivo gene delivery
- Basil Mathew
- , Raghu Ramanathan
- & Kevin G. Rice
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