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 2021-02-01 to 2025-02-01.)
ArticleCitations
Development of a cGMP-compliant process to manufacture donor-derived, CD45RA-depleted memory CD19-CAR T cells175
Rs1h−/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation97
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors92
Ghrelin mediated cardioprotection using in vitro models of oxidative stress47
Gene augmentation for autosomal dominant retinitis pigmentosa using rhodopsin genomic loci nanoparticles in the P23H+/− knock-in murine model47
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia45
Automated production of CCR5-negative CD4+-T cells in a GMP-compatible, clinical scale for treatment of HIV-positive patients38
Micro-dystrophin gene constructs for repairing heart and muscle function in rats: the smaller is enough?37
Effect of nanoparticle-mediated delivery of SFRP4 siRNA for treating Dupuytren disease36
Enhanced expression of the human Survival motor neuron 1 gene from a codon-optimised cDNA transgene in vitro and in vivo35
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice33
Precision ophthalmology: a call for Africa not to be left in the dark33
Effects of intracardiac delivery of aldehyde dehydrogenase 2 gene in myocardial salvage32
Schwann cell gene therapies in sight32
Combinatorial gene therapy for epilepsy: Gene sequence positioning and AAV serotype influence expression and inhibitory effect on seizures32
First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model30
AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 129
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings26
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury25
Gene delivery to the hypoglossal motor system: preclinical studies and translational potential25
Correction: RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells24
AAV library screening identifies novel vector for efficient transduction of human aorta24
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model23
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