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The disparate burden of infectious diseases
The disparate burden of infectious diseases
The age of modern medicine brought about groundbreaking methods to combat pathogens and improve our q...
Non-replicative herpes simplex virus genomic and amplicon vectors for gene therapy - an update
Non-replicative herpes simplex virus genomic and amplicon vectors for gene therapy - an update
Two major types of defective vectors have been derived from herpes simplex virus type 1 (HSV-1), non-replicative genomic v...
Retraction Note: Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations
These authors contributed equally: X. Sun, B. Pawlyk.Berman-Gund Laboratory for the Study of Retinal Degenerations, Harvar...
Bridging gene therapy and next-generation vaccine technologies
Bridging gene therapy and next-generation vaccine technologies
Gilbert S, Hatchett R. No one is safe until we are all safe. Sci Transl Med. 2021;13:eabl9900.Article  CAS  PubMed...
Genes for bad backs
Kang JD, Georgescu HI, McIntyre-Larkin L, Stefanovic-Racic M, Evans CH. Herniated cervical intervertebral discs spontaneou...
PCRX-201, a novel IL-1Ra gene therapy treatment approach for low back pain resulting from intervertebral disc degeneration
PCRX-201, a novel IL-1Ra gene therapy treatment approach for low back pain resulting from intervertebral disc degeneration
Low back pain is the leading cause of global disability with intervertebral disc (IVD) degeneration a major cause. However...
Gene-based therapy for the treatment of spinal muscular atrophy types 1 and 2 : a systematic review and meta-analysis
Gene-based therapy for the treatment of spinal muscular atrophy types 1 and 2 : a systematic review and meta-analysis
Despite numerous studies identifying the advantages of therapies for spinal muscular atrophy (SMA), healthcare professiona...
Prime editing: therapeutic advances and mechanistic insights
Prime editing: therapeutic advances and mechanistic insights
We are often confronted with a simple question, “which gene editing technique is the best?”; the simple answer...
Correction: Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications
Correction: Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications
Correction to: Gene Therapy https://doi.org/10.1038/s41434-020-0182-4, published online 17 August 202...
Correction: Andrew C. G. Porter (1955–2023)
Authors and AffiliationsAGCTlab.org, Centre of Gene and Cell Therapy, Department of Biological Sciences, School of Life Sc...
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2
Recombinant adeno-associated virus (rAAV) vectors are currently the only proven vehicles for treating ophthalmological dis...
Correction: Alternative oxidase encoded by sequence-optimized and chemically-modified RNA transfected into mammalian cells is catalytically active
Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which...
Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model
Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model
Almost all attempts to date at gene therapy approaches for monogenetic disease have used the amino acid sequences of the n...
Retraction Note: Oncolysis of pancreatic tumour cells by a γ34.5-deleted HSV-1 does not rely upon Ras-activation, but on the PI 3-kinase pathway
Authors and AffiliationsDepartment of Histology, Microbiology and Medical Biotechnologies, Section of Microbiology and Vir...
Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models
Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models
Mutation-agnostic treatments such as airway gene therapy have the potential to treat any individual with cystic fibrosis (...
Correction: AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses
Correction: AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses
Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which...
Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation
Adoptive cell therapy (ACT), particularly chimeric antigen receptor (CAR)-T cell therapy, has emerged as a promising appro...
Precision ophthalmology: a call for Africa not to be left in the dark
Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, et al. Efficacy and safety of voretigene neparvovec (AAV2-hR...
Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a
Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a
Efficient manufacturing of recombinant Adeno-Associated Viral (rAAV) vectors to meet rising clinical demand remains a majo...
A multinational survey of potential participant perspectives on ocular gene therapy
A multinational survey of potential participant perspectives on ocular gene therapy
Amidst rapid advancements in ocular gene therapy, understanding patient perspectives is crucial for shaping future treatme...
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia
Pseudomonas aeruginosa poses a significant threat to immunocompromised individuals and those with cystic fibrosis. Treatme...
Development of KoRV-pseudotyped lentiviral vectors for efficient gene transfer into freshly isolated immune cells
Development of KoRV-pseudotyped lentiviral vectors for efficient gene transfer into freshly isolated immune cells
Allogeneic cell therapies, such as those involving macrophages or Natural Killer (NK) cells, are of increasing interest fo...
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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