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
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
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
Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders	26
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