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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: 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...
Immune profiling of adeno-associated virus response identifies B cell-specific targets that enable vector re-administration in mice
Immune profiling of adeno-associated virus response identifies B cell-specific targets that enable vector re-administration in mice
Adeno-associated virus (AAV) vector-based gene therapies can be applied to a wide range of diseases. AAV expression can la...
A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification
A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification
Recombinant adeno-associated virus (rAAV) has become one of the most promising gene delivery systems for both in vitro and...
Taylor Dispersion Analysis to support lipid-nanoparticle formulations for mRNA vaccines
Taylor Dispersion Analysis to support lipid-nanoparticle formulations for mRNA vaccines
Lipid nanoparticles (LNPs) are currently the most advanced non-viral clinically approved messenger ribonucleic acid (mRNA)...
IgG-cleavage protein allows therapeutic AAV gene delivery in passively immunized MPS IIIA mice
IgG-cleavage protein allows therapeutic AAV gene delivery in passively immunized MPS IIIA mice
The widespread pre-existing αAAV-Abs in humans pose a critical challenge in translation of AAV gene therapy. The IgG ...
Activation of homology-directed DNA repair plays key role in CRISPR-mediated genome correction
Activation of homology-directed DNA repair plays key role in CRISPR-mediated genome correction
Gene editing for the cure of inborn errors of metabolism (IEMs) has been limited by inefficiency of adult hepatocyte targe...
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors
A formal screening of self-complementary adeno-associated virus (scAAV) vector serotypes in canine joint tissues has not b...
Influence of donor age and comorbidities on transduced human adipose-derived stem cell in vitro osteogenic potential
Influence of donor age and comorbidities on transduced human adipose-derived stem cell in vitro osteogenic potential
Human adipose-derived mesenchymal stem cells (ASCs) transduced with a lentiviral vector system to express bone morphogenet...
Characterization of a novel Pde6b-deficient rat model of retinal degeneration and treatment with adeno-associated virus (AAV) gene therapy
Characterization of a novel Pde6b-deficient rat model of retinal degeneration and treatment with adeno-associated virus (AAV) gene therapy
In humans, mutations in the beta subunit of cGMP-phosphodiesterase type 6 (PDE6B) cause autosomal recessive retinitis pigm...
Survey of perspectives of people with inherited retinal diseases on ocular gene therapy in Australia
Survey of perspectives of people with inherited retinal diseases on ocular gene therapy in Australia
Many gene therapies are in development for treating people with inherited retinal diseases (IRD). We hypothesized that pot...
Changing trends in the development of AAV-based gene therapies: a meta-analysis of past and present therapies
Changing trends in the development of AAV-based gene therapies: a meta-analysis of past and present therapies
Gene therapy has seen a transformation from a proof-of-concept approach to a clinical reality over the past several decade...
Design, construction and in vivo functional assessment of a hinge truncated sFLT01
Design, construction and in vivo functional assessment of a hinge truncated sFLT01
Gene therapy for the treatment of ocular neovascularization has reached clinical trial phases. The AAV2-sFLT01 construct w...
Multiplex viral tropism assay in complex cell populations with single-cell resolution
Multiplex viral tropism assay in complex cell populations with single-cell resolution
Gene therapy constitutes one of the most promising mode of disease treatments. Two key properties for therapeutic delivery...
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation
A primary goal in transplantation medicine is the induction of a tolerogenic environment for prevention of transplant reje...
rAAV-mediated over-expression of acid ceramidase prevents retinopathy in a mouse model of Farber lipogranulomatosis
rAAV-mediated over-expression of acid ceramidase prevents retinopathy in a mouse model of Farber lipogranulomatosis
Farber disease (FD) is a rare monogenic lysosomal storage disorder caused by mutations in ASAH1 that results in a deficien...
Loss of RAGE prevents chronic intermittent hypoxia-induced nonalcoholic fatty liver disease via blockade of NF-кB pathway
Loss of RAGE prevents chronic intermittent hypoxia-induced nonalcoholic fatty liver disease via blockade of NF-кB pathway
In recent years, receptor for advanced glycation end-products (RAGE) has been documented to induce liver fibrosis and infl...
A novel preclinical model of mucopolysaccharidosis type II for developing human hematopoietic stem cell gene therapy
A novel preclinical model of mucopolysaccharidosis type II for developing human hematopoietic stem cell gene therapy
A hematopoietic stem cell (HSC) gene therapy (GT) using lentiviral vectors has attracted interest as a promising treatment...
Selective attachment of a microtubule interacting peptide to plasmid DNA via a triplex forming oligonucleotide for transfection improvement
Selective attachment of a microtubule interacting peptide to plasmid DNA via a triplex forming oligonucleotide for transfection improvement
In nonviral gene therapy approaches, the linkage of signal molecules to plasmid DNA (pDNA) is of interest for guiding its ...
Cerebral organoids as an in vitro model to study autism spectrum disorders
Cerebral organoids as an in vitro model to study autism spectrum disorders
Autism spectrum disorders (ASDs) are a set of disorders characterised by social and communication deficits caused by numer...
Identification of RPGR ORF15 mutation for X-linked retinitis pigmentosa in a large Chinese family and in vitro correction with prime editor
Identification of RPGR ORF15 mutation for X-linked retinitis pigmentosa in a large Chinese family and in vitro correction with prime editor
X-linked retinitis pigmentosa (XLRP) is the most severe form of Retinitis Pigmentosa (RP) and one of the leading causes of...
Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model
Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model
X-linked adrenoleukodystrophy (ALD) is a genetic disorder of the ABCD1 gene. We aimed to treat ALD via direct intracerebra...
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials
Recombinant adeno-associated virus (AAV) vectors are the leading platform for gene delivery for a variety of clinical appl...
Splicing mutations in the CFTR gene as therapeutic targets
Splicing mutations in the CFTR gene as therapeutic targets
The marketing approval, about ten years ago, of the first disease modulator for patients with cystic fibrosis harboring sp...
Forced activation of dystrophin transcription by CRISPR/dCas9 reduced arrhythmia susceptibility via restoring membrane Nav1.5 distribution
Forced activation of dystrophin transcription by CRISPR/dCas9 reduced arrhythmia susceptibility via restoring membrane Nav1.5 distribution
Dystrophin deficiency due to genetic mutations causes cardiac abnormalities in Duchenne’s muscular dystrophy. Dystro...
Curing SMA: Are we there yet?
Curing SMA: Are we there yet?
Loss or deletion of survival motor neuron 1 gene (SMN1) is causative for a severe and devastating neuromuscular disease, S...
Effects of intracardiac delivery of aldehyde dehydrogenase 2 gene in myocardial salvage
Effects of intracardiac delivery of aldehyde dehydrogenase 2 gene in myocardial salvage
Intrinsic activity of aldehyde dehydrogenase (ALDH)2, a cardiac mitochondrial enzyme, is vital in detoxifying 4-hydroxy-2-...