OOIR: Observatory of International Research

Papers

(The H4-Index of Human Gene Therapy is 27. 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 2022-05-01 to 2026-05-01.)

Article	Citations
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	112
SAGA-Q: Gene Expression Differences Enable Detection of Retroviral Vectors with Mutagenic Potential	87
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	80
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	62
Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease	55
SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye	54
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	47
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	46
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	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	40
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	38
AVLAYAH and KRESLADI Win FDA Accelerated Approvals	34
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	34
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	33
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient	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	32
Efficacy and Safety of FX201, a Novel Intra-Articular IL-1Ra Gene Therapy for Osteoarthritis Treatment, in a Rat Model	31
Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders	30
AAV-Mediated Base Editing for Correction of RSPH4A Mutations in Primary Ciliary Dyskinesia: A Proof-of-Concept Study	30
Meta-Analysis and Optimization of the In Vitro Immortalization Assay for Safety Assessment of Retroviral Vectors in Gene Therapy	30
An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature	30
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop	29
Advancing Precision Medicine with Gene and Cell Therapy in Malaysia: Ethical, Legal, and Social Implications	28
uniQure Gene Therapy Significantly Slows Huntington Disease Progression	28

Abstracts	28
Genome Editing of Pik3cd Impedes Abnormal Retinal Angiogenesis	27