OOIR: Observatory of International Research

Papers

(The H4-Index of Human Gene Therapy is 25. 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 2022-08-01 to 2026-08-01.)

Article	Citations
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	106
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	82
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	74
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	62
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	59
SAGA-Q: Gene Expression Differences Enable Detection of Retroviral Vectors with Mutagenic Potential	48
AVLAYAH and KRESLADI Win FDA Accelerated Approvals	46
SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye	46
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	43
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid	42
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	36
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	35
Restoring Cancer Genomes: Functional Mutation Correction as a Platform for Precision Oncology	35
AAV.hBAG3 Gene Therapy Improves Phenotype in a Valosin Containing Protein Mouse Model of Hereditary Inclusion Body Myositis	35
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	33
AAV-Mediated Base Editing for Correction of RSPH4A Mutations in Primary Ciliary Dyskinesia: A Proof-of-Concept Study	33
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice	33
Toxicity and Biodistribution of the Oncolytic Virus VCN-01 Following Intracranial Injection in Syrian Hamsters	33
uniQure Gene Therapy Significantly Slows Huntington Disease Progression	32
Abstracts	32
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	28
Characterizing Complex Populations of Endogenous Adeno-Associated Viruses by Single-Genome Amplification	28
Advancing Precision Medicine with Gene and Cell Therapy in Malaysia: Ethical, Legal, and Social Implications	27
Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases	25