Perspective
|
Open Access
Featured
-
-
Brief Communication
| Open AccessAdvancing 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
-
Article |
Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate
- Mathieu Guerriaud
- , Cyril Poupet
- & Nathalie Moiré
-
Article
| Open AccessMulticenter 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
-
Article |
Gene therapy corrects the neurological deficits of mice with sialidosis
- Wuh-Liang Hwu
- , Karine Chang
- & Yin-Hsiu Chien
-
Article |
AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes
- Brahim Chaqour
- , Thu T. Duong
- & Ahmara G. Ross
-
Review Article |
CRISPR/Cas9-mediated base editors and their prospects for mitochondrial genome engineering
- Shahin Eghbalsaied
- , Clancy Lawler
- & Stephen Frankenberg
-
Article
| Open AccessGhrelin mediated cardioprotection using in vitro models of oxidative stress
- Cindy Y. Kok
- , George Ghossein
- & Eddy Kizana
-
Article |
CSTB gene replacement improves neuroinflammation, neurodegeneration and ataxia in murine type 1 progressive myoclonus epilepsy
- Emrah Gumusgoz
- , Sahba Kasiri
- & Berge A. Minassian
-
Article |
Distributional comparison of different AAV vectors after unilateral cochlear administration
- Shuang Han
- , Zhijiao Xu
- & Yilai Shu
-
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
-
Brief Communication
| Open AccessEngineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver
- Carola J. Maturana
-
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
-
Article |
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
-
Perspective |
Access to affordable medicines: obligations of universities and academic medical centers
- Steven Joffe
- , Rena M. Conti
- & Matthew S. McCoy
-
Article
| Open AccessSuccesses and challenges in clinical gene therapy
- Donald B. Kohn
- , Yvonne Y. Chen
- & Melissa J. Spencer
-
Article |
Approval and therapeutic value of gene therapies in the US and Europe
- Kerstin N. Vokinger
- , Camille E. G. Glaus
- & Aaron S. Kesselheim
-
Article
| Open AccessPrednisolone 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
-
Article |
p53 dry gene powder enhances anti-cancer effects of chemotherapy against malignant pleural mesothelioma
- Naomi Muramatsu
- , Misa Ichikawa
- & Hirokazu Okamoto
-
Article |
Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo
- Gabriela Toro Cabrera
- , Katharina E. Meijboom
- & Christian Mueller
-
Article |
Long-term effects of a fat-directed FGF21 gene therapy in aged female mice
- Jacqueline M. Anderson
- , W. David Arnold
- & Lei Cao
-
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
-
Article
| Open AccessTargeted 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
-
Article
| Open AccessCRISPR/sgRNA-directed synergistic activation mediator (SAM) as a therapeutic tool for Parkinson´s disease
- Luis Fernando Narváez-Pérez
- , Francisco Paz-Bermúdez
- & José Segovia
-
Article
| Open AccessRepeat or single-dose lentiviral vector administration to mouse lungs? It’s all about the timing
- Martin Donnelley
- , Patricia Cmielewski
- & Nigel Farrow
-
Article
| Open AccessCombinatorial gene therapy for epilepsy: Gene sequence positioning and AAV serotype influence expression and inhibitory effect on seizures
- Esbjörn Melin
- , My Andersson
- & Merab Kokaia
-
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
-
Article |
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
-
Article |
Removal of the GAA repeat in the heart of a Friedreich’s ataxia mouse model using CjCas9
- Pouiré Yaméogo
- , Catherine Gérard
- & Jacques P. Tremblay
-
Article |
Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy
- Jiali Nie
- , Yu Han
- & Dao Wen Wang
-
Brief Communication |
Ptbp1 knockdown failed to induce astrocytes to neurons in vivo
- Guixiang Yang
- , Zixiang Yan
- & Kailun Fang
-
Article
| Open AccessPreclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
- Jey M. Jeyakumar
- , Azadeh Kia
- & Amit Nathwani
-
Brief Communication
| Open AccessGlyco-binding domain chimeric antigen receptors as a new option for cancer immunotherapy
- Anna-Katharina Franke
- , Charlotte Wessolowski
- & Kerstin Cornils
-
Article |
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
-
Article |
Activation of homology-directed DNA repair plays key role in CRISPR-mediated genome correction
- Gourish Mondal
- , Caitlin J. VanLith
- & Joseph B. Lillegard
-
Article |
IgG-cleavage protein allows therapeutic AAV gene delivery in passively immunized MPS IIIA mice
- Tierra A. Bobo
- , Preston N. Samowitz
- & Haiyan Fu
-
Article
| Open AccessSurvey of perspectives of people with inherited retinal diseases on ocular gene therapy in Australia
- Heather G. Mack
- , Alexis Ceecee Britten-Jones
- & Lauren N. Ayton
-
Article
| Open AccessPreclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation
- Emma Proics
- , Marion David
- & Céline Dumont
-
Article |
A novel preclinical model of mucopolysaccharidosis type II for developing human hematopoietic stem cell gene therapy
- Yohta Shimada
- , Natsumi Ishii
- & Hiroshi Kobayashi
-
Article |
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
-
Article |
Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model
- Jie Gong
- , Yunyun Liu
- & Lung-Ji Chang
-
Article |
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
-
-
Article |
Effects of intracardiac delivery of aldehyde dehydrogenase 2 gene in myocardial salvage
- Guodong Pan
- , Bipradas Roy
- & Suresh Selvaraj Palaniyandi
-
Review Article |
Prospects of viral vector-mediated delivery of sequences encoding anti-HBV designer endonucleases
- Ridhwaanah Jacobs
- , Prashika Singh
- & Mohube Betty Maepa
-
Brief Communication
| Open AccessOnasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study
- Ilaria Bitetti
- , Valentina Lanzara
- & Antonio Varone
-
Article |
Efficient CRISPR/Cas9 nickase-mediated genome editing in an in vitro model of mucopolysaccharidosis IVA
- Andrés Felipe Leal
- & Carlos Javier Alméciga-Díaz
-
Article |
CRISPR/Cas9-based genome editing for the modification of multiple duplications that cause Duchenne muscular dystrophy
- Dan-Ni Wang
- , Zhi-Qiang Wang
- & Ning Wang
-
Article
| Open AccessHIV- 1 lentivirus tethering to the genome is associated with transcription factor binding sites found in genes that favour virus survival
- Saqlain Suleman
- , Annette Payne
- & Michael Themis
-
Article |
Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec –routine care evidence
- Sharon Aharoni
- , Jacob Bistritzer
- & Yoram Nevo
Browse broader subjects
Browse narrower subjects
- Cancer
- Cardiovascular diseases
- Dental diseases
- Endocrine system and metabolic diseases
- Eye diseases
- Gastrointestinal diseases
- Haematological diseases
- Immunological disorders
- Infectious diseases
- Kidney diseases
- Metabolic disorders
- Neurological disorders
- Nutrition disorders
- Oral diseases
- Psychiatric disorders
- Reproductive disorders
- Respiratory tract diseases
- Rheumatic diseases
- Skin diseases
- Trauma
- Urogenital diseases