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AAV2 capsid clearance and neuronal trafficking dynamics in the central nervous system
AAV2 capsid clearance and neuronal trafficking dynamics in the central nervous system
Adeno-associated virus serotype 2 (AAV2) remains one of the most common vectors for CNS gene delivery. Yet, its long-term ...
Clinical response to systemic AAV gene therapy in a large animal model of late-stage lysosomal storage disease
Clinical response to systemic AAV gene therapy in a large animal model of late-stage lysosomal storage disease
The benefit of early diagnosis and treatment has been demonstrated in animal models of several lysosomal storage diseases....
A dose-escalation and safety gene therapy study in a model of CMT4C neuropathy
A dose-escalation and safety gene therapy study in a model of CMT4C neuropathy
Charcot-Marie-Tooth disease type 4C is a demyelinating neuropathy caused by loss of function mutations in the SH3TC2 gene,...
rAAV8 encapsidated HMR-001/z enables high efficiency hepatic transduction and restores hemostasis in hemophilic mice
rAAV8 encapsidated HMR-001/z enables high efficiency hepatic transduction and restores hemostasis in hemophilic mice
Hemophilia A (HA), an X-linked bleeding disorder caused by factor VIII (FVIII) deficiency, is primarily managed with exoge...
Research progress and development strategies of antibody-oligonucleotide conjugates
Research progress and development strategies of antibody-oligonucleotide conjugates
Antibody-oligonucleotide conjugates (AOCs) effectively integrate the delivery capability of antibodies with the specific g...
Characterization of CAR-T cell factors that contribute to myeloid cell activation
Characterization of CAR-T cell factors that contribute to myeloid cell activation
Chimeric antigen receptor (CAR) T cell therapies have shown remarkable success in the treatment of hematologic cancers; ho...
DOCTER: a genetically encoded switchable protein module for ERα-mediated transcriptional regulation
DOCTER: a genetically encoded switchable protein module for ERα-mediated transcriptional regulation
The inhibition of estrogen receptor (ER)-mediated genomic signaling in ER-positive cancer cells has long been a primary fo...
Environmental and developmental factors shape anti-AAV immunity in pigs
Environmental and developmental factors shape anti-AAV immunity in pigs
Use of adeno-associated virus (AAV) vectors has revolutionized in vivo gene therapy, but the presence of pre-existing neut...
Rapid detection of AAV8 binding antibodies in gene therapy candidates: development of a point-of-care approach
Rapid detection of AAV8 binding antibodies in gene therapy candidates: development of a point-of-care approach
Preexisting anti-AAV antibodies pose a significant challenge to the success of Adeno-associated Virus (AAV) mediated gene ...
Identification of AAV variants with improved transduction of human vascular endothelial cells by screening AAV capsid libraries in non-human primates
Identification of AAV variants with improved transduction of human vascular endothelial cells by screening AAV capsid libraries in non-human primates
The development of targeted vector systems for gene therapy has made impressive progress during the last decade. Promising...
AAV-mediated MUC5AC siRNA delivery to prevent mucociliary dysfunction in asthma
AAV-mediated MUC5AC siRNA delivery to prevent mucociliary dysfunction in asthma
The main structural components of mucus produced in the lung are mucin 5B (MUC5B) and mucin 5AC (MUC5AC) where a relativel...
Correction: Identification of AAV variants with improved transduction of human vascular endothelial cells by screening AAV capsid libraries in non-human primates
Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which...
Correction: PPARγ is essential for protection against nonalcoholic steatohepatitis
Correction: PPARγ is essential for protection against nonalcoholic steatohepatitis
Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obta...
Overcoming matrix effects in AAV neutralization assays with a constant serum concentration approach
Overcoming matrix effects in AAV neutralization assays with a constant serum concentration approach
Sensitive quantification of adeno-associated virus (AAV) neutralizing antibodies (NAbs) is essential for gene therapy succ...
Genetic mutations in HSV-1 replication-defective vectors: Implications for their safety in gene therapy applications
Genetic mutations in HSV-1 replication-defective vectors: Implications for their safety in gene therapy applications
Beyond its well-known role in orofacial recurrent infections, HSV-1 has garnered significant attention in neuroscience for...
Codon changes challenge PCR-based gene doping detection
Codon changes challenge PCR-based gene doping detection
Genetic/genomic manipulation techniques (gene transfer/delivery, gene editing, etc.) have become more and more mature, and...
Co-delivery of IL-1Ra and SOX9 via AAV inhibits inflammation and promotes cartilage repair in surgically induced osteoarthritis animal models
Co-delivery of IL-1Ra and SOX9 via AAV inhibits inflammation and promotes cartilage repair in surgically induced osteoarthritis animal models
Osteoarthritis (OA), a prevalent joint disorder, can lead to disability, with no effective treatment available. Interleuki...
Incomplete elimination of viral genomes is associated with chronic inflammation in nonhuman primate livers after AAV-mediated gene transfer
Incomplete elimination of viral genomes is associated with chronic inflammation in nonhuman primate livers after AAV-mediated gene transfer
The liver is a unique organ where immunity can be biased toward ineffective response notably in the context of viral infec...
A peptide conjugate enables systemic injection of the morpholino inducer and more durable induction of T3H38 ribozyme-controlled AAV transgene in mice
A peptide conjugate enables systemic injection of the morpholino inducer and more durable induction of T3H38 ribozyme-controlled AAV transgene in mice
Genetic switches that allow for precise control over transgene expression timing or levels may improve the safety and expa...
AAV1.NT3 gene therapy mitigates the severity of autoimmune encephalomyelitis in the mouse model for multiple sclerosis
AAV1.NT3 gene therapy mitigates the severity of autoimmune encephalomyelitis in the mouse model for multiple sclerosis
Multiple sclerosis (MS) is an immune-mediated chronic inflammatory and neurodegenerative disease of the central nervous sy...
A capless hairpin-protected mRNA vaccine encoding the full-length Influenza A hemagglutinin protects mice against a lethal Influenza A infection
A capless hairpin-protected mRNA vaccine encoding the full-length Influenza A hemagglutinin protects mice against a lethal Influenza A infection
The success of mRNA vaccines in controlling the COVID 19 pandemic has confirmed the efficacy of synthetically synthesized ...
Improved induction of ribozyme-controlled AAV transgene via peptide-conjugated morpholino oligos
Improved induction of ribozyme-controlled AAV transgene via peptide-conjugated morpholino oligos
Adeno-associated virus (AAV)-mediated transgene delivery has emerged as a cornerstone in the field of gene therapy [1, 2]....
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver
Small promoters capable of driving potent neuron-restricted gene expression are required to support successful brain circu...
Looking ahead: ethical and social challenges of somatic gene therapy for sickle cell disease in Africa
Somatic gene therapy will be one of the most exciting practices of genetic medicine in Africa and is primed to offer a ...
Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement
Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement
Glutaric Aciduria type I (GA1) is a rare neurometabolic disorder caused by mutations in the GDCH gene encoding for glutary...
Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute
Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute
To the best of our knowledge, this effort represents the most comprehensive attempt to understand and develop solutions to...
Overexpression of KCNN4 channels in principal neurons produces an anti-seizure effect without reducing their coding ability
Overexpression of KCNN4 channels in principal neurons produces an anti-seizure effect without reducing their coding ability
Gene therapy offers a potential alternative to the surgical treatment of epilepsy, which affects millions of people and is...
Implications of maternal-fetal health on perinatal stem cell banking
Implications of maternal-fetal health on perinatal stem cell banking
Cell based therapies are being assessed for their therapeutic potential across a variety of diseases. Gestational tissues ...
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
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
In the rapidly evolving landscape of biotechnologies, cell and gene therapies are being developed and adopted at an unprec...
Cost of gene therapy
Since its earliest days and during the ensuing 5–6 decades, the field of human gene therapy has been char...
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