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 2021-11-01 to 2025-11-01.)

Article	Citations
Bluebird Bio Chief Financial Officer Resigns as Company Raises “Going Concern” Doubts	94
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	82
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	66
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient	60
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	49
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid	44
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	43
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	43
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	43
Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease	42
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	42
SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye	35
Strategies to Attenuate Myocardial Infarction and No-Reflow Through Preservation of Vascular Integrity by Pigment Epithelium-Derived Factor	35
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	33
Isolating Natural Adeno-Associated Viruses from Primate Tissues with a High-Fidelity Polymerase	30
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	29
Efficacy and Safety of FX201, a Novel Intra-Articular IL-1Ra Gene Therapy for Osteoarthritis Treatment, in a Rat Model	28
Toxicity and Biodistribution of the Oncolytic Virus VCN-01 Following Intracranial Injection in Syrian Hamsters	28
Meta-Analysis and Optimization of the In Vitro Immortalization Assay for Safety Assessment of Retroviral Vectors in Gene Therapy	27
Characterizing Complex Populations of Endogenous Adeno-Associated Viruses by Single-Genome Amplification	27
Abstracts	27
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice	27
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop	26
uniQure Gene Therapy Significantly Slows Huntington Disease Progression	26
ESGCT 2021	26

Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders