Perspective
|
Open Access
Featured
-
-
Article
| Open Access
A multinational survey of potential participant perspectives on ocular gene therapy
- Alexis Ceecee Britten-Jones
- , Myra B. McGuinness
- & Lauren N. Ayton
-
Perspective
| Open AccessPrecision ophthalmology: a call for Africa not to be left in the dark
- Lisa Roberts
-
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 Access
Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model RhoP23H+/−
- Shannon M. McNamee
- , Natalie P. Chan
- & Neena B. Haider
-
Article |
Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea
- Hyeokgyo Jeong
- , Sujata Purja
- & Eunyoung Kim
-
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 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
-
Perspective
| Open Access
Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute
- Lea Witkowsky
- , Matthew Norstad
- & Melinda Kliegman
-
Article |
Approval and therapeutic value of gene therapies in the US and Europe
- Kerstin N. Vokinger
- , Camille E. G. Glaus
- & Aaron S. Kesselheim
-
Review Article
| Open Access
A practical approach for adoption of a hub and spoke model for cell and gene therapies in low- and middle-income countries: framework and case studies
- Shadi Saleh
- , Omar Dabbous
- & Sean Tunis
-
Article
| Open Access
Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates
- Tricia H. Burdo
- , Chen Chen
- & Kamel Khalili
-
Article |
Biodistribution of lentiviral transduced adipose-derived stem cells for “ex-vivo” regional gene therapy for bone repair
- Jennifer A. Bell
- , Kevin Collon
- & Jay R. Lieberman
-
Article |
Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo
- Neda A. M. Nafchi
- , Ellie M. Chilcott
- & Rafael J. Yáñez-Muñoz
-
Article |
Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23H+/− knock-in murine model
- Simna SP
- , Rajendra N. Mitra
- & Zongchao Han
-
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 |
An ectopic enhancer restores CFTR expression through de novo chromatin looping
- Jenny L. Kerschner
- , Alekh Paranjapye
- & Ann Harris
-
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
| 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
-
Article |
rAAV-mediated over-expression of acid ceramidase prevents retinopathy in a mouse model of Farber lipogranulomatosis
- Hanmeng Zhang
- , Murtaza S. Nagree
- & Daniel M. Lipinski
-
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
-
Review Article
| Open Access
Splicing mutations in the CFTR gene as therapeutic targets
- Karine Deletang
- & Magali Taulan-Cadars
-
Article |
Forced activation of dystrophin transcription by CRISPR/dCas9 reduced arrhythmia susceptibility via restoring membrane Nav1.5 distribution
- Ruixin Zhang
- , Junwu Liu
- & Zhenwei Pan
-
-
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 Access
HIV- 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 |
Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy
- Aoife Reilly
- , Marc-Olivier Deguise
- & Rashmi Kothary
-
Article |
Effect of nanoparticle-mediated delivery of SFRP4 siRNA for treating Dupuytren disease
- Rujue Jin
- , Weigang Zhu
- & Aidong Deng
-
Perspective
| Open Access
Insidious Insights: Implications of viral vector engineering for pathogen enhancement
- Jonas B. Sandbrink
- , Ethan C. Alley
- & Kevin M. Esvelt
-
Brief Communication
| Open Access
Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy
- Carlos Fonck
- , Cheng Su
- & Charles A. O’Neill
-
Article |
Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets
- Kelly A. Rich
- , Christopher G. Wier
- & Stephen J. Kolb
-
Article
| Open Access
Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy
- Vahid Farrokhi
- , Jason Walsh
- & Hendrik Neubert
-
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
-
Article
| Open Access
Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models
- Ellie M. Chilcott
- , Evalyne W. Muiruri
- & Rafael J. Yáñez-Muñoz
-
Article |
Oncolytic Newcastle disease virus expressing the co-stimulator OX40L as immunopotentiator for colorectal cancer therapy
- Limin Tian
- , Tianyan Liu
- & Deshan Li
-
Article |
Injection pressure levels for creating blebs during subretinal gene therapy
- Brittni A. Scruggs
- , Huber Martins Vasconcelos Jr.
- & Andreas K. Lauer
-
Article
| Open Access
Development and biological characterization of a clinical gene transfer vector for the treatment of MAK-associated retinitis pigmentosa
- Budd A. Tucker
- , Erin R. Burnight
- & Edwin M. Stone
-
Article
| Open Access
Restoration of RPGR expression in vivo using CRISPR/Cas9 gene editing
- Jessica D. Gumerson
- , Amal Alsufyani
- & Tiansen Li
-
-
Review Article
| Open Access
Retroviral gene therapy in Germany with a view on previous experience and future perspectives
- Michael A. Morgan
- , Melanie Galla
- & Axel Schambach
-
Review Article |
Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders
- Yan Chen
- , Ruiting Wen
- & Zhanghui Chen
-
Article |
Ectopic expression of BBS1 rescues male infertility, but not retinal degeneration, in a BBS1 mouse model
- Matthew R. Cring
- , Kacie J. Meyer
- & Val C. Sheffield
-
Review Article |
Genetic insights, disease mechanisms, and biological therapeutics for Waardenburg syndrome
- Sida Huang
- , Jian Song
- & Yong Feng
-
Review Article |
Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review
- Nisreen Mohammed Al-Namnam
- , Soher Nagi Jayash
- & Mohammed Abdullah Alshawsh
-
Comment
| Open AccessLiver targeting with rAAV7: balancing tropism with immune profiles
- Terence R. Flotte
-
Article
| Open Access
Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse
- Janine A. Gilkes
- , Benjamin L. Judkins
- & Coy D. Heldermon
-
Article |
Intra-cisterna magna delivery of an AAV vector with the GLUT1 promoter in a pig recapitulates the physiological expression of SLC2A1
- Sachie Nakamura
- , Hitoshi Osaka
- & Takanori Yamagata
-
Review Article |
Gene therapy and gene correction: targets, progress, and challenges for treating human diseases
- Matthew R. Cring
- & Val C. Sheffield
Curing SMA: Are we there yet?
Control of gene doping in human and horse sportsBrowse broader subjects
Browse narrower subjects
- Agricultural genetics
- Animal breeding
- Behavioural genetics
- Cancer genetics
- Cancer genomics
- Clinical genetics
- Consanguinity
- CRISPR-Cas systems
- Cytogenetics
- Development
- Epigenetics
- Epigenomics
- Eukaryote
- Evolutionary biology
- Functional genomics
- Gene expression
- Gene regulation
- Genetic association study
- Genetic hybridization
- Genetic interaction
- Genetic linkage study
- Genetic markers
- Genome
- Genomic instability
- Genomics
- Genotype
- Haplotypes
- Heritable quantitative trait
- Immunogenetics
- Inbreeding
- Medical genetics
- Microbial genetics
- Mutation
- Neurodevelopmental disorders
- Plant breeding
- Plant genetics
- Polyploidy
- Population genetics
- Prokaryote
- Quantitative trait
- RNA splicing
- RNAi
- Sequencing
