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Tolerability and tropism of recombinant adeno-associated virus vectors in the African green monkey (Chlorocebus sabaeus) anterior chamber
Tolerability and tropism of recombinant adeno-associated virus vectors in the African green monkey (Chlorocebus sabaeus) anterior chamber
While many studies have investigated the use of recombinant adeno-associated vectors (rAAV) in the posterior chamber for t...
RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells
RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells
Even if adoptive cell transfer (ACT) has already shown great clinical efficiency in different types of disease, such as ca...
Adeno-associated virus vectors and neurotoxicity—lessons from preclinical and human studies
Adeno-associated virus vectors and neurotoxicity—lessons from preclinical and human studies
Over 15 years after hepatotoxicity was first observed following administration of an adeno-associated virus (AAV) vector d...
Repeat or single-dose lentiviral vector administration to mouse lungs? It’s all about the timing
Repeat or single-dose lentiviral vector administration to mouse lungs? It’s all about the timing
Lentiviral vectors are attractive delivery vehicles for cystic fibrosis gene therapy owing to their low immunogenicity and...
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings
Human gene replacement therapies such as onasemnogene abeparvovec (OA) use recombinant adeno-associated virus (rAAV) vecto...
rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality
rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality
Recently safety concerns have been raised in connection with high doses of recombinant adeno-associated viruses (rAAV). Th...
Combinatorial gene therapy for epilepsy: Gene sequence positioning and AAV serotype influence expression and inhibitory effect on seizures
Combinatorial gene therapy for epilepsy: Gene sequence positioning and AAV serotype influence expression and inhibitory effect on seizures
Gene therapy with AAV vectors carrying genes for neuropeptide Y and its receptor Y2 has been shown to inhibit seizures in ...
Correction: Antitumoral gene-based strategy involving nitric oxide synthase type III overexpression in hepatocellular carcinoma
Correction: Antitumoral gene-based strategy involving nitric oxide synthase type III overexpression in hepatocellular carcinoma
These authors share co-first authorship: Á J De la Rosa, Á Rodríguez-Hernández, R González.These authors share co-second a...
Large-scale purification of functional AAV particles packaging the full genome using short-term ultracentrifugation with a zonal rotor
Large-scale purification of functional AAV particles packaging the full genome using short-term ultracentrifugation with a zonal rotor
Adeno-associated virus (AAV) vector-based gene therapy is potentially curative for various genetic diseases; however, the ...
Induced pluripotent stem cell-derived dendritic cell vaccine therapy genetically modified on the ubiquitin-proteasome system
Induced pluripotent stem cell-derived dendritic cell vaccine therapy genetically modified on the ubiquitin-proteasome system
We previously reported that dendritic cells (DCs) transduced with the full-length tumor-associated antigen (TAA) gene indu...
Correction: Adjuvant properties of IFN-γ and GM-CSF in the scFv6.C4 DNA vaccine against CEA-expressing tumors
Correction: Adjuvant properties of IFN-γ and GM-CSF in the scFv6.C4 DNA vaccine against CEA-expressing tumors
Authors and AffiliationsResearch Center for Gene Therapy, Escola Paulista de Medicina, Universidade Federal de São Paulo, ...
Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23H+/− knock-in murine model
Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23H+/− knock-in murine model
Gene therapy for autosomal dominant retinitis pigmentosa (adRP) is challenged by the dominant inheritance of the mutant ge...
Comment on: Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets
Comment on: Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets
Peranteau WH, Flake AW. The Future of In Utero Gene Therapy. Mol Diagn Ther. 2020;24:135–42. https://doi.org/10.1007/s4029...
Novel rAAV vector mediated intrathecal HGF delivery has an impact on neuroimmune modulation in the ALS motor cortex with TDP-43 pathology
Novel rAAV vector mediated intrathecal HGF delivery has an impact on neuroimmune modulation in the ALS motor cortex with TDP-43 pathology
Recombinant adeno-associated virus (rAAV)-based gene therapies offer an immense opportunity for rare diseases, su...
Terminal hairpins improve protein expression in IRES-initiated mRNA in the absence of a cap and polyadenylated tail
Terminal hairpins improve protein expression in IRES-initiated mRNA in the absence of a cap and polyadenylated tail
Synthesizing mRNA in vitro is a standard and simple procedure. Adding the 5′ cap and 3′ polyadenylated (poly(A...
Removal of the GAA repeat in the heart of a Friedreich’s ataxia mouse model using CjCas9
Removal of the GAA repeat in the heart of a Friedreich’s ataxia mouse model using CjCas9
Most Friedreich ataxia (FRDA) cases are caused by the elongation of the GAA repeat (GAAr) sequence in the first intron of ...
Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression
Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression
Transcranial ultrasound combined with intravenous microbubbles can be used to increase blood-brain barrier permeability or...
Reversible stabilization of DNA/PEI complexes by reducible click-linkage between DNA and polymer. A new polyplex concept for lowering polymer quantity
Reversible stabilization of DNA/PEI complexes by reducible click-linkage between DNA and polymer. A new polyplex concept for lowering polymer quantity
Nonviral transfection of mammalian cells can be performed with electrostatic complexes (polyplexes) between a plasmid DNA ...
Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy
Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy
Variants in myosin-binding protein C3 (MYBPC3) gene are a main cause of hypertrophic cardiomyopathy (HCM), accounting for ...
Ptbp1 knockdown failed to induce astrocytes to neurons in vivo
Ptbp1 knockdown failed to induce astrocytes to neurons in vivo
The conversion of non-neuronal cells to neurons is a promising potential strategy for the treatment of neurodegenerative d...
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis
Respiratory syncytial virus (RSV) causes acute lower respiratory tract infections, with potential lower respiratory tract ...
Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters
Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters
Recombinant adeno-associated viral vectors (AAVs) are an effective system for gene transfer. AAV serotype 2 (AAV2) is comm...
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease
Fabry disease is an X-linked lysosomal storage disorder caused by loss of alpha-galactosidase A (α-Gal A) activity an...
Glyco-binding domain chimeric antigen receptors as a new option for cancer immunotherapy
Glyco-binding domain chimeric antigen receptors as a new option for cancer immunotherapy
In the last decade, treatment using Chimeric Antigen Receptor (CAR) are largely studied and demonstrate the potential of i...
Empowering gene delivery with protein engineering platforms
Empowering gene delivery with protein engineering platforms
The repertoire of therapeutic proteins has been substantially augmented by molecular engineering approaches, which have se...
An ectopic enhancer restores CFTR expression through de novo chromatin looping
An ectopic enhancer restores CFTR expression through de novo chromatin looping
Transcription of the cystic fibrosis transmembrane conductance regulator (CFTR) gene is regulated by both ubiquitous and c...
Potential of an anti-bevacizumab idiotype scFv DNA-based immunization to elicit VEGF-binding antibody response
Potential of an anti-bevacizumab idiotype scFv DNA-based immunization to elicit VEGF-binding antibody response
Anti-idiotype antibodies have been considered for vaccination approaches against different diseases, including cancers. Ba...
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model
CRISPR-based gene editing technology represents a promising approach to deliver therapies for inherited disorders, includi...
Correction: Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models
Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which...
Correction: Optimisation of a TALE nuclease targeting the HIV co-receptor CCR5 for clinical application
Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which...
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