OOIR: Observatory of International Research

Papers

(The TQCC of Human Gene Therapy is 7. 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.)

Article	Citations
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	74
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	74
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	73
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 Polymerase	50
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid	49
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient	47
Bluebird Bio Chief Financial Officer Resigns as Company Raises “Going Concern” Doubts	44
Induction of Robust Type I Interferon Levels by Oncolytic Reovirus Requires Both Viral Replication and Interferon-α/β Receptor Signaling	42
Strategies to Attenuate Myocardial Infarction and No-Reflow Through Preservation of Vascular Integrity by Pigment Epithelium-Derived Factor	41
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	37
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	36
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	36
Long-Term Follow-Up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy	36
Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease	35
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	33
Ocular Inflammation with Anti-Vascular Endothelial Growth Factor Treatments	32
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	31
A Cautiously Optimistic Outlook of a Designer Therapy for 1% of Duchenne Muscular Dystrophy Patients	30
Gene Therapy in Global Health: Meeting the Needs of Chinese Patients with Beta-Thalassemia	30
Efficacy and Safety of FX201, a Novel Intra-Articular IL-1Ra Gene Therapy for Osteoarthritis Treatment, in a Rat Model	29
pNaSS-Grafted PCL Film-Guided rAAV TGF-β Gene Therapy Activates the Chondrogenic Activities in Human Bone Marrow Aspirates	28
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice	28
Advancing Precision Medicine with Gene and Cell Therapy in Malaysia: Ethical, Legal, and Social Implications	27
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	27

Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases	26
Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-Modified AAV Vector	26
Meta-Analysis and Optimization of the In Vitro Immortalization Assay for Safety Assessment of Retroviral Vectors in Gene Therapy	25
Global Rare Disease Research Symposium & The Second China Rare Disease Research and Translational Medicine Annual ConferenceShanghai, ChinaMay 23-25, 2025	25
ESGCT 2021	24
An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature	24
Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders	22
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop	22
Characterizing Complex Populations of Endogenous Adeno-Associated Viruses by Single-Genome Amplification	22
Immune Responses to Adeno-Associated Virus-Mediated CRISPR Therapy	22
Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell	22
Genome Editing of Pik3cd Impedes Abnormal Retinal Angiogenesis	21
Development and Validation of a Liquid Chromatography-Tandem Mass Spectrometry Method for Sensitive Analysis of Residual Protein Tat Bh1–101 in Lentiviral Vectors for Gene Therapy	21
rAAV Production and Titration at the Microscale for High-Throughput Screening	21
Lived Experience with Gene Therapy	20
Correction to: An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature, by Ramirez et al. Hum Gene Ther	20
Immune Responses to Recombinant Adenoviruses As Gene Therapy Vectors and COVID-19 Vaccines: A Two-Edged Sword	19
Primum Non Nocere: Should Gene Therapy Be Used to Prevent Potentially Fatal Disease but Enable Potentially Destructive Behavior?	19
Gene Therapy, the Immune System, and Vaccine Development: An Interview with Hildegund Ertl	19
Gene Therapy for Huntington's Disease: The Future Is in Gene Editing	19
uniQure, CSL Behring Launch $2B Hemophilia B Partnership	19
The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy	18
Lipid Nanoparticles for Nucleic Acid Delivery Beyond the Liver	17
CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells	17
Quantitation of Residual Host Cell DNA in Recombinant Adeno-Associated Virus Using Droplet Digital Polymerase Chain Reaction	17
Conversion of the Liver into a Biofactory for DNaseI Using Adeno-Associated Virus Vector Gene Transfer Reduces Neutrophil Extracellular Traps in a Model of Systemic Lupus Erythematosus	17
Modulation of AAV9 Galactose Binding Yields Novel Gene Therapy Vectors and Predicts Cross-Species Differences in Glycan Avidity	17
SP-101, A Novel Adeno-Associated Virus Gene Therapy for the Treatment of Cystic Fibrosis, Mediates Functional Correction of Primary Human Airway Epithelia From Donors with Cystic Fibrosis	16
AAV1-Mediated shRNA Knockdown of SASH1 in Rat Bronchus Attenuates Hypoxia-Induced Pulmonary Artery Remodeling	16
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy	16
Adeno-Associated Virus-Mediated Gene Transfer of Inducible Nitric Oxide Synthase to an Animal Model of Pulmonary Hypertension	15
Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for Human Gene Therapy	15
Interview with Dr. Ronald Crystal	15
Engineered U7 Small Nuclear RNA Restores Correct β-Globin Pre-mRNA Splicing in Mouse β^IVS2-654-Thalassemic Erythroid Progenitor Cells	15
Success Stories and Challenges Ahead in Hematopoietic Stem Cell Gene Therapy: Hemoglobinopathies as Disease Models	15
Acknowledgment of Reviewers 2021	15
Patient Dies in Beam Trial of Sickle Cell Disease Candidate; Company Cites Conditioning	15
A Recombinant Oncolytic Influenza Virus Carrying GV1001 Triggers an Antitumor Immune Response	15
Genetic Modification of Limbs UsingEx VivoMachine Perfusion	14
Update on Viral Gene Therapy Clinical Trials for Retinal Diseases	14
Prediction of Adeno-Associated Virus Fitness with a Protein Language-Based Machine Learning Model	14
Urocortin 2 Gene Transfer for Systolic and Diastolic Dysfunction Due to Chronically Increased Left Ventricular Pressure	14
Generation of a Novel Oncolytic Vaccinia Virus Using the IHD-W Strain	13
ESGCTCollaborative Virtual Congress19–22 October 2021Abstracts	13
Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles	13
Introduction to ESGCT 2024 Special Issue	13
Clinical Efficacy and Safety of AdV-tk Gene Therapy for Patients with Cervical Squamous Intraepithelial Lesion: A Prospective Study	13
The Implication of Hinge 1 and Hinge 4 in Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy	12
Progress in Respiratory Gene Therapy	12
Matrix Protein of Vesicular Stomatitis Virus Targets the Mitochondria, Reprograms Glucose Metabolism, and Sensitizes to 2-Deoxyglucose in Glioblastoma	12

Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease	12
Transduction of Ferret Surface and Basal Cells of Airways, Lung, Liver, and Pancreas via Intratracheal or Intravenous Delivery of Adeno-Associated Virus 1 or 6	12
Pre-Existing Immunity to a Nucleic Acid Contaminant-Derived Antigen Mediates Transaminitis and Resultant Diminished Transgene Expression in a Mouse Model of Hepatic Recombinant Adeno-Associated Virus-	12
An Optimized CRISPR/Cas9 Adenovirus Vector (AdZ-CRISPR) for High-Throughput Cloning of sgRNA, Using Enhanced sgRNA and Cas9 Variants	12
Correction to: Generation of a Novel Oncolytic Vaccinia Virus Using the IHD-W Strain by Jaeil Shin et al. Hum Gene Ther 2021;32:9–10. DOI: 10.1089/hum.2020.050	12
Is Hospital Exemption an Alternative or a Bridge to European Medicines Agency for Developing Academic Chimeric Antigen Receptor T-Cell in Europe? Our Experience with ARI-0001	12
Development and Delivery of a Hands-On Short Course in Adeno-Associated Virus Manufacturing to Support Growing Workforce Needs in Gene Therapy	12
Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis	12
Analysis of HIV-1-Based Lentiviral Vector Particle Composition by PacBio Long-Read Nucleic Acid Sequencing	12
Effects of Urocortin 2 Gene Transfer on Glucose Disposal in Insulin-Resistant db/db Mice on Metformin	12
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article	11
Biodistribution of Transplanted Hematopoietic Precursor Cells Injected Through Different Administration Routes in Newborn Mice	11
Adeno-Associated Virus Type 9-Mediated Gene Therapy of Choline Acetyltransferase-Deficient Mice	11
Chemical Epigenetic Regulation of Adeno-Associated Virus Delivered Transgenes	11
Efficient Delivery of Adeno-Associated Virus into Inner Ear In Vivo Through Trans-Stapes Route in Adult Guinea Pig	11
Recent Advances Using Genetic Therapies Against Infectious Diseases and for Vaccination	11
Engineered Human Adenoviruses of Species B and C Report Early, Intermediate Early, and Late Viral Gene Expression	11
Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond	11
Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy	11
Bluebird Bio Eliminating 30% of Staff in Restructuring	11
Akouos, Immusoft Win FDA Clearances for First-of-Their-Kind Gene Therapies	11
Capsid-Engineering for Central Nervous System-Directed Gene Therapy with Adeno-Associated Virus Vectors	11
Production of Recombinant Adeno-Associated Virus Through High-Cell-Density Transfection of HEK293 Cells Based on Fed-Perfusion Culture	11
Current and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed Gene Therapy	10
Pfizer Marks Phase III Success in Hemophilia A, then Layoffs after Failure in DMD	10
Pfizer Weighs Next Steps after DMD Therapy Linked to Boy’s Death Fails Phase III Trial	10
Leveraging CRISPR-Cas9 for Accurate Detection of AAV-Neutralizing Antibodies: The AAV-HDR Method	10
A Modified Arrestin1 Increases Lactate Production in the Retina and Slows Retinal Degeneration	10
Preclinical Development and Characterization of Novel Adeno-Associated Viral Vectors for the Treatment of Lipoprotein Lipase Deficiency	10
CRISPR-Cas Genome Editing in Ex Vivo Human Lungs to Rewire the Translational Path of Genome-Targeting Therapeutics	10
Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities	9
Gene Coexpression and miRNA Regulation: A Path to Early Intervention in Colorectal Cancer	9
Preclinical Development of a HIV-Based Gene Therapeutic for Cystic Fibrosis Pulmonary Disease	9
Hemophilia Gene Therapy: The End of the Beginning?	9
Process Development of Recombinant Adeno-Associated Virus Production Platform Results in High Production Yield and Purity	9
Enhanced Cochlear Transduction by AAV9 with High-Concentration Sucrose	9
Therapeutic Strategy for Fabry Disease by Intravenous Administration of Adeno-Associated Virus 9 in a Symptomatic Mouse Model	9
Liver Gene Therapy	9
A Review of the Cost-Effectiveness Evidence for FDA-Approved Cell and Gene Therapies	9
Intra-Articular Adeno-Associated Virus-Mediated Proteoglycan 4 Gene Therapy for Preventing Posttraumatic Osteoarthritis	9
Single-Use Capture Purification of Adeno-Associated Viral Gene Transfer Vectors by Membrane-Based Steric Exclusion Chromatography	8
After Sparring with Payers on Pricing, Bluebird Flies Out of Europe	8
Personalizing Oncolytic Virotherapy	8
Modulation of Immune Reaction in Hydrodynamic Gene Therapy for Hemophilia A	8
The Engineered MARCH8-Resistant Vesicular Stomatitis Virus Glycoprotein Enhances Lentiviral Vector Transduction	8
Association of NPC1L1 and HMGCR Gene Polymorphisms with Major Adverse Cardiac and Cerebrovascular Events in Patients with Three-Vessel Disease	8
Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1	8
Engineering Cancer Selective Virotherapies: Are the Pieces of the Puzzle Falling into Place?	8
Bone Marrow Mesenchymal Stem Cells-Derived Exosomal MicroRNA-150-3p Promotes Osteoblast Proliferation and Differentiation in Osteoporosis	8
Advances in the Development and the Applications of Nonviral, Episomal Vectors for Gene Therapy	8
Rational Use of Immunosuppressive Corticosteroids in Liver-Directed Adeno-Associated Virus Gene Therapy Studies	7
The Future of Exon Skipping for Duchenne Muscular Dystrophy	7
ESGCT 29th Annual Congress In collaboration with BSGCT Edinburgh, UK October 11–14, 2022 Abstracts	7
Adeno-Associated Virus-Mediated Knockdown of Agmatinase Attenuates Inflammation and Tumorigenesis in a Mouse Model of Colitis-Associated Colorectal Cancer	7
International Conference on Lymphocyte Engineering Author Index	7
Interview with Barry Byrne, MD/PhD	7
Unconstrained Precision Mitochondrial Genome Editing with αDdCBEs	7
Trojan Horse-Like Vehicles for CRISPR-Cas Delivery: Engineering Extracellular Vesicles and Virus-Like Particles for Precision Gene Editing in Cystic Fibrosis	7
Ultragenyx Gene Therapies Spark Lawsuit from Lacks Family	7
Correction to: Letter to the Editor: A Favorable Benefit–Risk Balance Maybe Expected with Replication-Defective Adenovirus-Mediated Interferon Gene Therapy for Cancer Treatment, by Qin Hum G	7