Human Gene Therapy

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 2021-05-01 to 2025-05-01.)
ArticleCitations
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model74
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime74
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future73
First Positive Clinical Data for In Vivo Genome Editing in Humans Opens “New Era of Medicine”72
Isolating Natural Adeno-Associated Viruses from Primate Tissues with a High-Fidelity Polymerase50
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid49
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient47
Bluebird Bio Chief Financial Officer Resigns as Company Raises “Going Concern” Doubts44
Induction of Robust Type I Interferon Levels by Oncolytic Reovirus Requires Both Viral Replication and Interferon-α/β Receptor Signaling42
Strategies to Attenuate Myocardial Infarction and No-Reflow Through Preservation of Vascular Integrity by Pigment Epithelium-Derived Factor41
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II37
Long-Term Follow-Up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy36
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease36
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies36
Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease35
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation33
Ocular Inflammation with Anti-Vascular Endothelial Growth Factor Treatments32
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases31
Gene Therapy in Global Health: Meeting the Needs of Chinese Patients with Beta-Thalassemia30
A Cautiously Optimistic Outlook of a Designer Therapy for 1% of Duchenne Muscular Dystrophy Patients30
Efficacy and Safety of FX201, a Novel Intra-Articular IL-1Ra Gene Therapy for Osteoarthritis Treatment, in a Rat Model29
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice28
pNaSS-Grafted PCL Film-Guided rAAV TGF-β Gene Therapy Activates the Chondrogenic Activities in Human Bone Marrow Aspirates28
Advancing Precision Medicine with Gene and Cell Therapy in Malaysia: Ethical, Legal, and Social Implications27
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis27
Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases26
Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-Modified AAV Vector26
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