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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...
Safety, efficacy, and immunogenicity of a novel IgG degrading enzyme (KJ103): results from two randomised, blinded, phase 1 clinical trials
Safety, efficacy, and immunogenicity of a novel IgG degrading enzyme (KJ103): results from two randomised, blinded, phase 1 clinical trials
The approved intravenous adeno-associated virus (AAV) therapies are limited by the widespread prevalence of pre-existing a...
Identifying novel response markers for spinal muscular atrophy revealed by targeted proteomics following gene therapy
Identifying novel response markers for spinal muscular atrophy revealed by targeted proteomics following gene therapy
Spinal muscular atrophy (SMA) is a progressive disease that affects motor neurons, with symptoms usually starting in infan...
Correction: RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells
Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which...
AAV library screening identifies novel vector for efficient transduction of human aorta
AAV library screening identifies novel vector for efficient transduction of human aorta
Targeted gene delivery to vascular smooth muscle cells (VSMCs) could prevent or improve a variety of diseases affecting th...
Intracisternal vs intraventricular injection of AAV1 result in comparable, widespread transduction of the dog brain
Intracisternal vs intraventricular injection of AAV1 result in comparable, widespread transduction of the dog brain
Widespread distribution of transduced brain cells following delivery of AAV vectors into the cerebrospinal fluid (CSF) of ...
Unlocking patient access to gene therapy: five key practices
Unlocking patient access to gene therapy: five key practices
Zhang JX, Shugarman LR. Value-based payment and financing for cell and gene therapies: challenges and potential solutions....
Placental nanoparticle-mediated IGF1 gene therapy corrects fetal growth restriction in a guinea pig model
Placental nanoparticle-mediated IGF1 gene therapy corrects fetal growth restriction in a guinea pig model
Fetal growth restriction (FGR) caused by placental insufficiency is a major contributor to neonatal morbidity and mortalit...
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...
Adeno-associated virus serotype 2 capsids with proteolytic cuts by trypsin remain intact and potent
Adeno-associated virus serotype 2 capsids with proteolytic cuts by trypsin remain intact and potent
Recombinant adeno-associated viral (AAV) vectors have emerged as prominent gene delivery vehicles for gene therapy. In the...
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...
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...
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 ...
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