Gene Therapy

Papers
(The H4-Index of Gene Therapy is 24. The table below lists those papers that are above that threshold based on CrossRef citation counts [max. 250 papers]. The publications cover those that have been published in the past four years, i.e., from 2020-05-01 to 2024-05-01.)
ArticleCitations
Self-amplifying RNA vaccines for infectious diseases217
Current status and perspective of CAR-T and CAR-NK cell therapy trials in Germany149
Prime editing – an update on the field75
CARAMBA: a first-in-human clinical trial with SLAMF7 CAR-T cells prepared by virus-free Sleeping Beauty gene transfer to treat multiple myeloma70
Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy66
Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters62
Gene therapy and gene correction: targets, progress, and challenges for treating human diseases57
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair41
Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions39
Investigation of the safety and feasibility of AAV1/SERCA2a gene transfer in patients with chronic heart failure supported with a left ventricular assist device – the SERCA-LVAD TRIAL38
AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy33
Non-viral delivery of CRISPR–Cas9 complexes for targeted gene editing via a polymer delivery system33
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry30
Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice29
Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?29
Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells28
Gene therapy for spinal muscular atrophy: the Qatari experience27
Natural polyphenol assisted delivery of single-strand oligonucleotides by cationic polymers27
Fabrication of anionic dextran-coated micelles for aptamer targeted delivery of camptothecin and survivin-shRNA to colon adenocarcinoma26
Gene editing and RNAi approaches for COVID-19 diagnostics and therapeutics26
miR-503-5p inhibits colon cancer tumorigenesis, angiogenesis, and lymphangiogenesis by directly downregulating VEGF-A26
Genome editing for Duchenne muscular dystrophy: a glimpse of the future?25
An overview of development in gene therapeutics in China25
The stability of envelope-pseudotyped lentiviral vectors24
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