Gene Therapy

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 2020-11-01 to 2024-11-01.)
ArticleCitations
Current status and perspective of CAR-T and CAR-NK cell therapy trials in Germany168
CARAMBA: a first-in-human clinical trial with SLAMF7 CAR-T cells prepared by virus-free Sleeping Beauty gene transfer to treat multiple myeloma91
Prime editing – an update on the field89
Non-viral delivery of CRISPR–Cas9 complexes for targeted gene editing via a polymer delivery system47
Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions47
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair45
Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells38
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry37
AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy36
Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?33
Gene therapy for spinal muscular atrophy: the Qatari experience33
Fabrication of anionic dextran-coated micelles for aptamer targeted delivery of camptothecin and survivin-shRNA to colon adenocarcinoma32
Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice32
Genome editing for Duchenne muscular dystrophy: a glimpse of the future?29
Gene editing and RNAi approaches for COVID-19 diagnostics and therapeutics28
Successes and challenges in clinical gene therapy25
Rational engineering of a functional CpG-free ITR for AAV gene therapy25
Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 125
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation25
Curing SMA: Are we there yet?25
Enhanced CNS transduction from AAV.PHP.eB infusion into the cisterna magna of older adult rats compared to AAV924
AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 124
Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study23
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