Featured
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Article |
CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy
- Emrah Gumusgoz
- , Sahba Kasiri
- & Berge A. Minassian
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Perspective |
Access to affordable medicines: obligations of universities and academic medical centers
- Steven Joffe
- , Rena M. Conti
- & Matthew S. McCoy
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Perspective
| Open Access
Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute
- Lea Witkowsky
- , Matthew Norstad
- & Melinda Kliegman
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Article
| Open Access
The estimated annual financial impact of gene therapy in the United States
- Chi Heem Wong
- , Dexin Li
- & Rena M. Conti
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Article
| Open Access
Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques
- Alexander Kistner
- , Jessica A. Chichester
- & Barbara A. Sullivan
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Article |
Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo
- Gabriela Toro Cabrera
- , Katharina E. Meijboom
- & Christian Mueller
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Article
| Open Access
A pilot study to determine the optimal dose of scAAVIL-1ra in a large animal model of post-traumatic osteoarthritis
- P. Thampi
- , K. A. Seabaugh
- & L. R. Goodrich
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Review Article |
Selection of appropriate non-clinical animal models to ensure translatability of novel AAV-gene therapies to the clinic
- Mark Singh
- , Andrew Brooks
- & Keith McLuckie
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Article |
Safety and biodistribution of XC001 (encoberminogene rezmadenovec) gene therapy in rats: a potential therapy for cardiovascular diseases
- Duncan J. Stewart
- , Albert Gianchetti
- & Rickey R. Reinhardt
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Article
| Open AccessReview of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings
- Deepa H. Chand
- , Rui Sun
- & Francis Fonyuy Tukov
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Review Article |
Empowering gene delivery with protein engineering platforms
- Monika Kizerwetter
- , Kevin Pietz
- & Jamie B. Spangler
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Article |
Taylor Dispersion Analysis to support lipid-nanoparticle formulations for mRNA vaccines
- Camille Malburet
- , Laurent Leclercq
- & Hervé Cottet
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Article |
Changing trends in the development of AAV-based gene therapies: a meta-analysis of past and present therapies
- Tamara Burdett
- & Samir Nuseibeh
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Article
| Open Access
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials
- Liching Cao
- , Annemarie Ledeboer
- & Kathleen Meyer
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Comment |
Micro-dystrophin gene constructs for repairing heart and muscle function in rats: the smaller is enough?
- Petra Lujza Szabo
- & Attila Kiss
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Brief Communication |
Targeting mAKAPβ expression as a therapeutic approach for ischemic cardiomyopathy
- Eliana C. Martinez
- , Jinliang Li
- & Michael S. Kapiloff
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Review Article |
Progress on siRNA-based gene therapy targeting secondary injury after intracerebral hemorrhage
- Daniyah A. Almarghalani
- & Zahoor A. Shah
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News |
Consideration with “Intratumoral gene therapy versus intravenous gene therapy for distant metastasis control with DDMC non-viral vector–p53”
- Y. Onishi
- , P. Zarogoulidis
- & Y. Eshita
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Article |
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 |
Characterization of integration frequency and insertion sites of adenovirus DNA into mouse liver genomic DNA following intravenous injection
- Zhibin Wang
- , Philip J. Troilo
- & Brian J. Ledwith
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Perspective
| Open Access
The Gibco™ CTS™ Rotea™ system story—a case study of industry-academia collaboration
- Anqi Li
- , David James
- & Rebecca Lim
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Brief Communication
| Open Access
Alternative oxidase encoded by sequence-optimized and chemically-modified RNA transfected into mammalian cells is catalytically active
- Luca Giordano
- , Manish K. Aneja
- & Marten Szibor
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Review Article |
Gene therapy and gene correction: targets, progress, and challenges for treating human diseases
- Matthew R. Cring
- & Val C. Sheffield
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Article |
Neutralisation of adeno-associated virus transduction by human vitreous humour
- Sławomir Andrzejewski
- , Peter M. Moyle
- & Christopher J. Layton
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Article |
Treating Bietti crystalline dystrophy in a high-fat diet-exacerbated murine model using gene therapy
- Bin Qu
- , Shijing Wu
- & Wei Li
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Review Article |
Gene therapy for cardiovascular diseases in China: basic research
- Jiali Deng
- , Mengying Guo
- & Junjie Xiao
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Article
| Open Access
Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa
- Sujun Li
- , Shyamtanu Datta
- & Neena B. Haider
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Article |
A novel gene editing system to treat both Tay–Sachs and Sandhoff diseases
- Li Ou
- , Michael J. Przybilla
- & Chester B. Whitley
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Article |
Neonatal nonviral gene editing with the CRISPR/Cas9 system improves some cardiovascular, respiratory, and bone disease features of the mucopolysaccharidosis I phenotype in mice
- Roselena Silvestri Schuh
- , Esteban Alberto Gonzalez
- & Guilherme Baldo
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Brief Communication |
An engineered serum albumin-binding AAV9 capsid achieves improved liver transduction after intravenous delivery in mice
- Quan Jin
- , Chunping Qiao
- & Xiao Xiao
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Comment |
AAV shedding after intracoronary delivery: just a safety concern?
- Kelly P. Yamada
- , Taro Kariya
- & Kiyotake Ishikawa
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Review Article |
A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies
- Wendy White
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Article |
AAVrh-10 transduces outer retinal cells in rodents and rabbits following intravitreal administration
- Yong Zeng
- , Haohua Qian
- & Ronald A. Bush
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Review Article
| Open Access
Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine
- Kris Elverum
- & Maria Whitman
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Article |
Dual-targeted NIS polyplexes—a theranostic strategy toward tumors with heterogeneous receptor expression
- Sarah Urnauer
- , Kathrin A. Schmohl
- & Christine Spitzweg
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Article |
Anti-angiogenic treatment of endometriosis via anti-VEGFA siRNA delivery by means of peptide-based carrier in a rat subcutaneous model
- Anna Egorova
- , Mariya Petrosyan
- & Anton Kiselev
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Brief Communication |
Human carbonic anhydrase-8 AAV8 gene therapy inhibits nerve growth factor signaling producing prolonged analgesia and anti-hyperalgesia in mice
- Gerald Z. Zhuang
- , Udita Upadhyay
- & Roy C. Levitt
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Original Article
| Open Access
Optimization of lentiviral vector production for scale-up in fixed-bed bioreactor
- A J Valkama
- , H M Leinonen
- & H P Lesch
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Editorial |
Compilation of online resources of relevance to ‘Spinraza and advanced therapies: a stakeholder special’ issue of Gene Therapy
- V Prakash
- & R J Yáñez-Muñoz
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Original Article
| Open Access
Efficacy of combining ING4 and TRAIL genes in cancer-targeting gene virotherapy strategy: first evidence in preclinical hepatocellular carcinoma
- A Galal El-Shemi
- , A Mohammed Ashshi
- & C-O Yun
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Original Article |
Oligonucleotide-targeting periostin ameliorates pulmonary fibrosis
- A Tomaru
- , T Kobayashi
- & E C Gabazza
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Original Article
| Open Access
AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease
- J Miniarikova
- , V Zimmer
- & P Konstantinova
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Review
| Open Access
The importance of international collaboration for rare diseases research: a European perspective
- D Julkowska
- , C P Austin
- & S van Weely
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Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
CRISPR editing as a therapeutic strategy for Duchenne muscular dystrophy—anti-Cas9 immune response casts its shadow over safety and efficacy