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
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice	33
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
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
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
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
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop	29
uniQure Gene Therapy Significantly Slows Huntington Disease Progression	28
Abstracts	28

Advancing Precision Medicine with Gene and Cell Therapy in Malaysia: Ethical, Legal, and Social Implications	28
Genome Editing of Pik3cd Impedes Abnormal Retinal Angiogenesis	27