OOIR: Observatory of International Research

Papers

(The H4-Index of Human Gene Therapy is 26. 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 2022-06-01 to 2026-06-01.)

Article	Citations
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	115
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid	92
AVLAYAH and KRESLADI Win FDA Accelerated Approvals	80
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	65
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	56
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	55
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	47
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	45
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	42
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	40
SAGA-Q: Gene Expression Differences Enable Detection of Retroviral Vectors with Mutagenic Potential	36
Toxicity and Biodistribution of the Oncolytic Virus VCN-01 Following Intracranial Injection in Syrian Hamsters	34
SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye	34
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	34
AAV-Mediated Base Editing for Correction of RSPH4A Mutations in Primary Ciliary Dyskinesia: A Proof-of-Concept Study	34
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice	33
Meta-Analysis and Optimization of the In Vitro Immortalization Assay for Safety Assessment of Retroviral Vectors in Gene Therapy	31
Abstracts	31
Characterizing Complex Populations of Endogenous Adeno-Associated Viruses by Single-Genome Amplification	30
uniQure Gene Therapy Significantly Slows Huntington Disease Progression	30
Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell	30
Genome Editing of Pik3cd Impedes Abnormal Retinal Angiogenesis	30
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop	29
Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases	28
Advancing Precision Medicine with Gene and Cell Therapy in Malaysia: Ethical, Legal, and Social Implications	28

Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders	27
An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature	26