SCI时时刷

search
+
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...
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...
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...
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...
Expression and distribution of rAAV9 intrathecally administered in juvenile to adolescent mice
Expression and distribution of rAAV9 intrathecally administered in juvenile to adolescent mice
Intrathecal (IT) lumbar puncture delivery of recombinant adeno-associated virus serotype 9 (rAAV9) is a gene therapy appro...
The circRNA circSCAF8 promotes tumor growth and metastasis of gastric cancer via miR-1293/TIMP1signaling
The circRNA circSCAF8 promotes tumor growth and metastasis of gastric cancer via miR-1293/TIMP1signaling
SR-like CTD-associated factor 8 (SCAF8) can regulate transcriptional termination, but the function of circSCAF8 remains un...
Electroporation-mediated novel albumin-fused Flt3L DNA delivery promotes cDC1-associated anticancer immunity
Electroporation-mediated novel albumin-fused Flt3L DNA delivery promotes cDC1-associated anticancer immunity
Dendritic cells (DCs) constitute a distinct type of immune cell found within tumors, serving a central role in mediating t...
Characterization of anti-AAV2 neutralizing antibody levels in sheep prior to and following intravitreal AAV2.7m8 injection
Characterization of anti-AAV2 neutralizing antibody levels in sheep prior to and following intravitreal AAV2.7m8 injection
Gene augmentation therapy is a promising treatment for incurable, blinding inherited retinal diseases, and intravitreal de...
AAV gene therapy for Duchenne Muscular Dystrophy: lessons learned from a phase 3 trial
FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy. 2023. https://www.fda....
Chemogenetics with PSAM4-GlyR decreases excitability and epileptiform activity in epileptic hippocampus
Chemogenetics with PSAM4-GlyR decreases excitability and epileptiform activity in epileptic hippocampus
Despite the availability of new drugs on the clinics in recent years, drug-resistant epilepsy remains an unresolved challe...
Targeting serum response factor (SRF) deactivates ΔFosB and mitigates Levodopa-induced dyskinesia in a mouse model of Parkinson’s disease
Targeting serum response factor (SRF) deactivates ΔFosB and mitigates Levodopa-induced dyskinesia in a mouse model of Parkinson’s disease
L-3,4-dihydroxyphenylalanine (L-DOPA) is currently the preferred treatment for Parkinson’s Disease (PD) and is consi...
Suppression of matrigel-induced choroidal neovascularization by AAV delivery of a novel anti-Scg3 antibody
Suppression of matrigel-induced choroidal neovascularization by AAV delivery of a novel anti-Scg3 antibody
Efforts to develop gene therapy for long-term treatment of neovascular disease are hampered by ongoing concerns that biolo...
Precision medicine: toward restoring fat with gene therapy in inherited lipodystrophy
Mann JP, Savage DB. What lipodystrophies teach us about the metabolic syndrome. J Clin Invest. 2019;129:4009–21. https://d...
Gene therapy of Dent disease type 1 in newborn ClC-5 null mice for sustained transgene expression and gene therapy effects
Gene therapy of Dent disease type 1 in newborn ClC-5 null mice for sustained transgene expression and gene therapy effects
Dent disease type 1 is caused by changes in the chloride voltage-gated channel 5 (CLCN5) gene on chromosome X, resulting i...
BRD9 promotes the progression of gallbladder cancer via CST1 upregulation and interaction with FOXP1 through the PI3K/AKT pathway and represents a therapeutic target
BRD9 promotes the progression of gallbladder cancer via CST1 upregulation and interaction with FOXP1 through the PI3K/AKT pathway and represents a therapeutic target
Gallbladder cancer (GBC) is highly aggressive and has poor prognosis, with most patients only diagnosed at an advanced sta...
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa
Open Access This article is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 ...
Retraction Note: Inhibition of microRNA-495 suppresses chondrocyte apoptosis through activation of the NF-κB signaling pathway by regulating CCL4 in osteoarthritis
Authors and AffiliationsDepartment of Orthopaedics, The Fourth Affiliated Hospital of Harbin Medical University, 150001, H...
Activated factor X delivered by adeno-associated virus significantly inhibited bleeding and alleviated hemophilic synovitis in hemophilic mice
Activated factor X delivered by adeno-associated virus significantly inhibited bleeding and alleviated hemophilic synovitis in hemophilic mice
In hemophilia, deficiency of factor VIII or IX prevents the activation of the common coagulation pathway, and inhibits the...
Retraction Note: miR-503-5p inhibits colon cancer tumorigenesis, angiogenesis, and lymphangiogenesis by directly downregulating VEGF-A
Authors and AffiliationsDepartment of Radiotherapy, Cancer Hospital of China Medical University, Liaoning Cancer Hospital ...
CRISPR/Cas9-mediated exon skipping to restore premature translation termination in a DFNB4 mouse model
CRISPR/Cas9-mediated exon skipping to restore premature translation termination in a DFNB4 mouse model
SLC26A4 encodes pendrin, a crucial anion exchanger essential for maintaining hearing function. Mutations in SLC26A4, inclu...
AAV dose-dependent transduction efficiency in retinal ganglion cells and functional efficacy of optogenetic vision restoration
AAV dose-dependent transduction efficiency in retinal ganglion cells and functional efficacy of optogenetic vision restoration
Optogenetics is a promising approach for restoring vision to the blind after photoreceptor degeneration. The ability to re...
Intravesical nerve growth factor antisense therapy for bladder hypersensitivity induced by psychological stress
Intravesical nerve growth factor antisense therapy for bladder hypersensitivity induced by psychological stress
This study assessed the relationship between NGF expression in the bladder and bladder hypersensitivity caused by psycholo...
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...
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...
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...
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: Andrew C. G. Porter (1955–2023)
Authors and AffiliationsAGCTlab.org, Centre of Gene and Cell Therapy, Department of Biological Sciences, School of Life Sc...
阅读更多
format_indent_increase