Activated factor X delivered by adeno-associated virus significantly inhibited bleeding and alleviated hemophilic synovitis in hemophilic mice

2024-9-10

Retraction Note: Inhibition of microRNA-495 suppresses chondrocyte apoptosis through activation of the NF-κB signaling pathway by regulating CCL4 in osteoarthritis

2024-9-9

Retraction Note: miR-503-5p inhibits colon cancer tumorigenesis, angiogenesis, and lymphangiogenesis by directly downregulating VEGF-A

2024-9-6

AAV dose-dependent transduction efficiency in retinal ganglion cells and functional efficacy of optogenetic vision restoration

2024-9-5

CRISPR/Cas9-mediated exon skipping to restore premature translation termination in a DFNB4 mouse model

2024-9-4

Intravesical nerve growth factor antisense therapy for bladder hypersensitivity induced by psychological stress

2024-9-3

Correction: AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses

2024-8-26

Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models

2024-8-25

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

2024-8-16

Correction: Alternative oxidase encoded by sequence-optimized and chemically-modified RNA transfected into mammalian cells is catalytically active

2024-8-15

Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model

2024-8-15

The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2

2024-8-12

Correction: Andrew C. G. Porter (1955–2023)

2024-8-7

Correction: Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications

2024-8-1

Andrew C. G. Porter (1955–2023)

2024-7-29

Preclinical evaluation of tissue-selective gene therapies for congenital generalised lipodystrophy

2024-7-28

Amelioration of airway and GI disease in G551D-CF ferrets by AAV1 and AAV6

2024-7-28

Gene drives: an alternative approach to malaria control?

2024-7-22

Characterization of brain transduction capability of a BBB-penetrant AAV vector in mice, rats and macaques reveals differences in expression profiles

2024-7-22

Suppression of viral rebound by a Rev-dependent lentiviral particle in SIV-infected rhesus macaques

2024-7-18

The impact of heparin and direct thrombin inhibitors on cell-penetrating polymer siRNA transfection

2024-7-16

Correction: Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65

2024-7-12

Correction: Dual-targeted NIS polyplexes—a theranostic strategy toward tumors with heterogeneous receptor expression

2024-7-11

Administration and detection of a multi-target rAAV gene doping vector in horses using multiple matrices and molecular techniques

2024-7-7

Stable inhibition of choroidal neovascularization by adeno-associated virus 2/8-vectored bispecific molecules

2024-7-3

Retraction Note: Optimization of oncolytic effect of Newcastle disease virus Clone30 by selecting sensitive tumor host and constructing more oncolytic viruses

2024-7-1

Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system

2024-6-25

Gene replacement therapy for spinal muscular atrophy: safety and preliminary efficacy in a Brazilian cohort

2024-6-5

Superoxide dismutase 1-modified dental pulp stem cells alleviate high-altitude pulmonary edema by inhibiting oxidative stress through the Nrf2/HO-1 pathway

2024-6-4

Retinoic acid related orphan receptor α is a genetic modifier that rescues retinal degeneration in a mouse model of Stargardt disease and Dry AMD

2024-5-16

Development of KoRV-pseudotyped lentiviral vectors for efficient gene transfer into freshly isolated immune cells

2024-4-29

AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia

2024-4-27

Towards ethical drug pricing: the European Orphan Genomic Therapies Fund

2024-4-24

Long-term benefits of hematopoietic stem cell-based macrophage/microglia delivery of GDNF to the CNS in a mouse model of Parkinson’s disease

2024-4-16

A multinational survey of potential participant perspectives on ocular gene therapy

2024-4-2

Adeno-associated virus genome quantification with amplification-free CRISPR-Cas12a

2024-3-25

Precision ophthalmology: a call for Africa not to be left in the dark

2024-3-22

Advancing rare disease treatment: EMA’s decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation

2024-3-14

A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice

2024-3-12

Are genetically modified protozoa eligible for ATMP status? Concerning the legal categorization of an oncolytic protozoan drug candidate

2024-3-1

Analytical characterization of full, intermediate, and empty AAV capsids

2024-2-19

Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia

2024-2-14

Gene therapy corrects the neurological deficits of mice with sialidosis

2024-2-7

Development of a stable Sf9 insect cell line to produce VSV-G pseudotyped baculoviruses

2024-1-26

Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model RhoP23H+/−

2024-1-26

Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates

2024-1-12

AAV2 vector optimization for retinal ganglion cell-targeted delivery of therapeutic genes

2024-1-10

Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea

2024-1-10

Retraction Note: Long noncoding RNA RBMS3-AS3 acts as a microRNA-4534 sponge to inhibit the progression of prostate cancer by upregulating VASH1

2024-1-9

Correction: Cost of gene therapy

2024-1-4