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Open Access
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Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease
- Guo Ge
- , Barath P. Sivasubramanian
- & Senlin Li
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Article
| Open Access
A multinational survey of potential participant perspectives on ocular gene therapy
- Alexis Ceecee Britten-Jones
- , Myra B. McGuinness
- & Lauren N. Ayton
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Perspective
| Open AccessPrecision ophthalmology: a call for Africa not to be left in the dark
- Lisa Roberts
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Brief Communication
| Open Access
Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
- Maria Elisabeth Kalland
- , Tomas Pose-Boirazian
- & Segundo Mariz
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Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate
- Mathieu Guerriaud
- , Cyril Poupet
- & Nathalie Moiré
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Article
| Open Access
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia
- Ingrid Pabinger
- , Mila Ayash-Rashkovsky
- & John Chapin
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Gene therapy corrects the neurological deficits of mice with sialidosis
- Wuh-Liang Hwu
- , Karine Chang
- & Yin-Hsiu Chien
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AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes
- Brahim Chaqour
- , Thu T. Duong
- & Ahmara G. Ross
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Review Article |
CRISPR/Cas9-mediated base editors and their prospects for mitochondrial genome engineering
- Shahin Eghbalsaied
- , Clancy Lawler
- & Stephen Frankenberg
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Article
| Open Access
Ghrelin mediated cardioprotection using in vitro models of oxidative stress
- Cindy Y. Kok
- , George Ghossein
- & Eddy Kizana
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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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Distributional comparison of different AAV vectors after unilateral cochlear administration
- Shuang Han
- , Zhijiao Xu
- & Yilai Shu
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Review Article
| Open AccessLooking ahead: ethical and social challenges of somatic gene therapy for sickle cell disease in Africa
- Nchangwi Syntia Munung
- , Obiageli E. Nnodu
- & Ambroise Wonkam
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Brief Communication
| Open Access
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver
- Carola J. Maturana
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Brief Communication |
Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement
- A. Mateu-Bosch
- , E. Segur-Bailach
- & C. Fillat
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Overexpression of KCNN4 channels in principal neurons produces an anti-seizure effect without reducing their coding ability
- Evgeny S. Nikitin
- , Tatiana Y. Postnikova
- & Aleksey V. Zaitsev
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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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Article
| Open AccessSuccesses and challenges in clinical gene therapy
- Donald B. Kohn
- , Yvonne Y. Chen
- & Melissa J. Spencer
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Approval and therapeutic value of gene therapies in the US and Europe
- Kerstin N. Vokinger
- , Camille E. G. Glaus
- & Aaron S. Kesselheim
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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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p53 dry gene powder enhances anti-cancer effects of chemotherapy against malignant pleural mesothelioma
- Naomi Muramatsu
- , Misa Ichikawa
- & Hirokazu Okamoto
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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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Long-term effects of a fat-directed FGF21 gene therapy in aged female mice
- Jacqueline M. Anderson
- , W. David Arnold
- & Lei Cao
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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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| Open Access
Targeted shock-and-kill HIV-1 gene therapy approach combining CRISPR activation, suicide gene tBid and retargeted adenovirus delivery
- Sarah Klinnert
- , Corinne D. Schenkel
- & Karin J. Metzner
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Article
| Open Access
CRISPR/sgRNA-directed synergistic activation mediator (SAM) as a therapeutic tool for Parkinson´s disease
- Luis Fernando Narváez-Pérez
- , Francisco Paz-Bermúdez
- & José Segovia
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Article
| Open Access
Repeat or single-dose lentiviral vector administration to mouse lungs? It’s all about the timing
- Martin Donnelley
- , Patricia Cmielewski
- & Nigel Farrow
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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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| Open Access
Combinatorial gene therapy for epilepsy: Gene sequence positioning and AAV serotype influence expression and inhibitory effect on seizures
- Esbjörn Melin
- , My Andersson
- & Merab Kokaia
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Comment |
Comment on: Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets
- Apeksha Dave
- , Cara L. Berkowitz
- & William H. Peranteau
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Novel rAAV vector mediated intrathecal HGF delivery has an impact on neuroimmune modulation in the ALS motor cortex with TDP-43 pathology
- Barış Genç
- , Boram Nho
- & Hande Özdinler
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Removal of the GAA repeat in the heart of a Friedreich’s ataxia mouse model using CjCas9
- Pouiré Yaméogo
- , Catherine Gérard
- & Jacques P. Tremblay
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Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy
- Jiali Nie
- , Yu Han
- & Dao Wen Wang
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Brief Communication |
Ptbp1 knockdown failed to induce astrocytes to neurons in vivo
- Guixiang Yang
- , Zixiang Yan
- & Kailun Fang
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Article
| Open Access
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
- Jey M. Jeyakumar
- , Azadeh Kia
- & Amit Nathwani
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Brief Communication
| Open Access
Glyco-binding domain chimeric antigen receptors as a new option for cancer immunotherapy
- Anna-Katharina Franke
- , Charlotte Wessolowski
- & Kerstin Cornils
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Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors
- Ah Young Kim
- , Felix Michael Duerr
- & Laurie R. Goodrich
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Activation of homology-directed DNA repair plays key role in CRISPR-mediated genome correction
- Gourish Mondal
- , Caitlin J. VanLith
- & Joseph B. Lillegard
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IgG-cleavage protein allows therapeutic AAV gene delivery in passively immunized MPS IIIA mice
- Tierra A. Bobo
- , Preston N. Samowitz
- & Haiyan Fu
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Article
| Open Access
Survey of perspectives of people with inherited retinal diseases on ocular gene therapy in Australia
- Heather G. Mack
- , Alexis Ceecee Britten-Jones
- & Lauren N. Ayton
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Article
| Open Access
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation
- Emma Proics
- , Marion David
- & Céline Dumont
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A novel preclinical model of mucopolysaccharidosis type II for developing human hematopoietic stem cell gene therapy
- Yohta Shimada
- , Natsumi Ishii
- & Hiroshi Kobayashi
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Identification of RPGR ORF15 mutation for X-linked retinitis pigmentosa in a large Chinese family and in vitro correction with prime editor
- Xiujuan Lv
- , Zheng Zheng
- & Feng Gu
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Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model
- Jie Gong
- , Yunyun Liu
- & Lung-Ji Chang
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Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes
- Jayanth Chandran
- , Ekram Ahmed Chowdhury
- & Guy M. L. Meno-Tetang
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Effects of intracardiac delivery of aldehyde dehydrogenase 2 gene in myocardial salvage
- Guodong Pan
- , Bipradas Roy
- & Suresh Selvaraj Palaniyandi
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Brief Communication
| Open Access
Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study
- Ilaria Bitetti
- , Valentina Lanzara
- & Antonio Varone
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Review Article |
Prospects of viral vector-mediated delivery of sequences encoding anti-HBV designer endonucleases
- Ridhwaanah Jacobs
- , Prashika Singh
- & Mohube Betty Maepa
Curing SMA: Are we there yet?