OOIR: Observatory of International Research

Papers

(The H4-Index of Human Gene Therapy is 25. 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 2021-08-01 to 2025-08-01.)

Article	Citations
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	84
First Positive Clinical Data for In Vivo Genome Editing in Humans Opens “New Era of Medicine”	77
Bluebird Bio Chief Financial Officer Resigns as Company Raises “Going Concern” Doubts	76
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	58
Strategies to Attenuate Myocardial Infarction and No-Reflow Through Preservation of Vascular Integrity by Pigment Epithelium-Derived Factor	55
Induction of Robust Type I Interferon Levels by Oncolytic Reovirus Requires Both Viral Replication and Interferon-α/β Receptor Signaling	49
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	47
Isolating Natural Adeno-Associated Viruses from Primate Tissues with a High-Fidelity Polymerase	46
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	42
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	39
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	38
Long-Term Follow-Up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy	37
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	36
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient	36
Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease	35
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	33
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid	33
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	32
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice	32
A Cautiously Optimistic Outlook of a Designer Therapy for 1% of Duchenne Muscular Dystrophy Patients	32
Efficacy and Safety of FX201, a Novel Intra-Articular IL-1Ra Gene Therapy for Osteoarthritis Treatment, in a Rat Model	27
pNaSS-Grafted PCL Film-Guided rAAV TGF-β Gene Therapy Activates the Chondrogenic Activities in Human Bone Marrow Aspirates	26
Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders	26
ESGCT 2021	25
Abstracts	25

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