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 2021-06-01 to 2025-06-01.)

Article	Citations
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	77
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient	75
First Positive Clinical Data for In Vivo Genome Editing in Humans Opens “New Era of Medicine”	75
Bluebird Bio Chief Financial Officer Resigns as Company Raises “Going Concern” Doubts	73
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	54
Induction of Robust Type I Interferon Levels by Oncolytic Reovirus Requires Both Viral Replication and Interferon-α/β Receptor Signaling	51
Strategies to Attenuate Myocardial Infarction and No-Reflow Through Preservation of Vascular Integrity by Pigment Epithelium-Derived Factor	48
Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease	46
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	45
Isolating Natural Adeno-Associated Viruses from Primate Tissues with a High-Fidelity Polymerase	42
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	40
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid	37
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	37
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	36
Ocular Inflammation with Anti-Vascular Endothelial Growth Factor Treatments	35
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	33
A Cautiously Optimistic Outlook of a Designer Therapy for 1% of Duchenne Muscular Dystrophy Patients	33
Long-Term Follow-Up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy	33
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	33
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	31
pNaSS-Grafted PCL Film-Guided rAAV TGF-β Gene Therapy Activates the Chondrogenic Activities in Human Bone Marrow Aspirates	31
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice	30
Efficacy and Safety of FX201, a Novel Intra-Articular IL-1Ra Gene Therapy for Osteoarthritis Treatment, in a Rat Model	29
Abstracts	28
Meta-Analysis and Optimization of the In Vitro Immortalization Assay for Safety Assessment of Retroviral Vectors in Gene Therapy	28

ESGCT 2021	28
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop	27