OOIR: Observatory of International Research

Papers

(The H4-Index of Gene Therapy is 23. 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 2020-03-01 to 2024-03-01.)

Article	Citations
Self-amplifying RNA vaccines for infectious diseases	196
Current status and perspective of CAR-T and CAR-NK cell therapy trials in Germany	143
Prime editing – an update on the field	71
CARAMBA: a first-in-human clinical trial with SLAMF7 CAR-T cells prepared by virus-free Sleeping Beauty gene transfer to treat multiple myeloma	68
Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy	65
Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters	59
Gene therapy and gene correction: targets, progress, and challenges for treating human diseases	52
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair	38
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 TRIAL	34
Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions	34
Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa	32
Non-viral delivery of CRISPR–Cas9 complexes for targeted gene editing via a polymer delivery system	32
AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy	31
Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?	29
Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice	28
Gene therapy for spinal muscular atrophy: the Qatari experience	27
Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells	27
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry	26
An overview of development in gene therapeutics in China	25
Gene editing and RNAi approaches for COVID-19 diagnostics and therapeutics	24
The stability of envelope-pseudotyped lentiviral vectors	24
Genome editing for Duchenne muscular dystrophy: a glimpse of the future?	24
Natural polyphenol assisted delivery of single-strand oligonucleotides by cationic polymers	23