OOIR: Observatory of International Research

Papers

(The H4-Index of Molecular Therapy-Methods & Clinical Development is 30. 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.)

Article	Citations
Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies	107
Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning	90
CNS Transduction Benefits of AAV-PHP.eB over AAV9 Are Dependent on Administration Route and Mouse Strain	89
Comparison of analytical techniques to quantitate the capsid content of adeno-associated viral vectors	72
CRISPR-Cas9 gene editing of hepatitis B virus in chronically infected humanized mice	70
Acerola exosome-like nanovesicles to systemically deliver nucleic acid medicine via oral administration	63
qPCR and qRT-PCR analysis: Regulatory points to consider when conducting biodistribution and vector shedding studies	60
Mechanistic model for production of recombinant adeno-associated virus via triple transfection of HEK293 cells	55
piggyBac system to co-express NKG2D CAR and IL-15 to augment the in vivo persistence and anti-AML activity of human peripheral blood NK cells	47
High Levels of Frataxin Overexpression Lead to Mitochondrial and Cardiac Toxicity in Mouse Models	47
Quantitative analysis of genome packaging in recombinant AAV vectors by charge detection mass spectrometry	46
Development of a scalable and robust AEX method for enriched rAAV preparations in genome-containing VCs of serotypes 5, 6, 8, and 9	45
Pharmacological interventions enhance virus-free generation of TRAC-replaced CAR T cells	44
Enhanced expression of immune checkpoint receptors during SARS-CoV-2 viral infection	43
Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations	42
Anion-exchange HPLC assay for separation and quantification of empty and full capsids in multiple adeno-associated virus serotypes	42
The impact of lentiviral vector genome size and producer cell genomic to gag-pol mRNA ratios on packaging efficiency and titre	41
AAV-S: A versatile capsid variant for transduction of mouse and primate inner ear	41
In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy	40
Characterization of AAV-mediated dorsal root ganglionopathy	40
Characterization of AAV-Specific Affinity Ligands: Consequences for Vector Purification and Development Strategies	38
Transplantation of miPSC/mESC-derived retinal ganglion cells into healthy and glaucomatous retinas	36
Automated generation of gene-edited CAR T cells at clinical scale	36
Targeted epigenetic repression by CRISPR/dSaCas9 suppresses pathogenic DUX4-fl expression in FSHD	35
Exosomes and organ-specific metastasis	34

A flexible, thermostable nanostructured lipid carrier platform for RNA vaccine delivery	34
Immunogenicity assessment of AAV-based gene therapies: An IQ consortium industry white paper	33
Clinical Development of Gene Therapies: The First Three Decades and Counting	33
Assessment of genome packaging in AAVs using Orbitrap-based charge-detection mass spectrometry	31
Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous β039-thalassemia patients	31
Rapid Lentiviral Vector Producer Cell Line Generation Using a Single DNA Construct	30
Applying machine learning to predict viral assembly for adeno-associated virus capsid libraries	30
Systemic and local immune responses to intraocular AAV vector administration in non-human primates	30