OOIR: Observatory of International Research

Papers

(The TQCC of Human Gene Therapy is 8. 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
First Positive Clinical Data for In Vivo Genome Editing in Humans Opens “New Era of Medicine”	75
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient	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
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	33
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
pNaSS-Grafted PCL Film-Guided rAAV TGF-β Gene Therapy Activates the Chondrogenic Activities in Human Bone Marrow Aspirates	31
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	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
ESGCT 2021	28
Abstracts	28

Meta-Analysis and Optimization of the In Vitro Immortalization Assay for Safety Assessment of Retroviral Vectors in Gene Therapy	28
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop	27
Immune Responses to Adeno-Associated Virus-Mediated CRISPR Therapy	26
Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell	25
An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature	25
Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders	24
Characterizing Complex Populations of Endogenous Adeno-Associated Viruses by Single-Genome Amplification	24
Advancing Precision Medicine with Gene and Cell Therapy in Malaysia: Ethical, Legal, and Social Implications	23
Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-Modified AAV Vector	22
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	22
Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases	22
Genome Editing of Pik3cd Impedes Abnormal Retinal Angiogenesis	22
Gene Therapy, the Immune System, and Vaccine Development: An Interview with Hildegund Ertl	21
uniQure, CSL Behring Launch $2B Hemophilia B Partnership	21
Immune Responses to Recombinant Adenoviruses As Gene Therapy Vectors and COVID-19 Vaccines: A Two-Edged Sword	21
Lived Experience with Gene Therapy	21
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	20
Gene Therapy for Huntington's Disease: The Future Is in Gene Editing	20
Modulation of AAV9 Galactose Binding Yields Novel Gene Therapy Vectors and Predicts Cross-Species Differences in Glycan Avidity	20
AAV1-Mediated shRNA Knockdown of SASH1 in Rat Bronchus Attenuates Hypoxia-Induced Pulmonary Artery Remodeling	20
rAAV Production and Titration at the Microscale for High-Throughput Screening	19
Primum Non Nocere: Should Gene Therapy Be Used to Prevent Potentially Fatal Disease but Enable Potentially Destructive Behavior?	19
The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy	19
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	19
Quantitation of Residual Host Cell DNA in Recombinant Adeno-Associated Virus Using Droplet Digital Polymerase Chain Reaction	18
CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells	18
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	18
Lipid Nanoparticles for Nucleic Acid Delivery Beyond the Liver	17
Acknowledgment of Reviewers 2021	17
Interview with Dr. Ronald Crystal	17
Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for Human Gene Therapy	17
Genetic Modification of Limbs UsingEx VivoMachine Perfusion	16
Adeno-Associated Virus-Mediated Gene Transfer of Inducible Nitric Oxide Synthase to an Animal Model of Pulmonary Hypertension	16
Prediction of Adeno-Associated Virus Fitness with a Protein Language-Based Machine Learning Model	16
Patient Dies in Beam Trial of Sickle Cell Disease Candidate; Company Cites Conditioning	16
A Recombinant Oncolytic Influenza Virus Carrying GV1001 Triggers an Antitumor Immune Response	16
ESGCTCollaborative Virtual Congress19–22 October 2021Abstracts	15
Update on Viral Gene Therapy Clinical Trials for Retinal Diseases	15
Introduction to ESGCT 2024 Special Issue	15
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy	15
An Optimized CRISPR/Cas9 Adenovirus Vector (AdZ-CRISPR) for High-Throughput Cloning of sgRNA, Using Enhanced sgRNA and Cas9 Variants	15
Success Stories and Challenges Ahead in Hematopoietic Stem Cell Gene Therapy: Hemoglobinopathies as Disease Models	15
Development and Delivery of a Hands-On Short Course in Adeno-Associated Virus Manufacturing to Support Growing Workforce Needs in Gene Therapy	14
Progress in Respiratory Gene Therapy	14
The Implication of Hinge 1 and Hinge 4 in Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy	13
Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis	13
Clinical Efficacy and Safety of AdV-tk Gene Therapy for Patients with Cervical Squamous Intraepithelial Lesion: A Prospective Study	13
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-	13
Urocortin 2 Gene Transfer for Systolic and Diastolic Dysfunction Due to Chronically Increased Left Ventricular Pressure	13
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	13

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
Effects of Urocortin 2 Gene Transfer on Glucose Disposal in Insulin-Resistant db/db Mice on Metformin	12
Matrix Protein of Vesicular Stomatitis Virus Targets the Mitochondria, Reprograms Glucose Metabolism, and Sensitizes to 2-Deoxyglucose in Glioblastoma	12
Bluebird Bio Eliminating 30% of Staff in Restructuring	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
Production of Recombinant Adeno-Associated Virus Through High-Cell-Density Transfection of HEK293 Cells Based on Fed-Perfusion Culture	12
Chemical Epigenetic Regulation of Adeno-Associated Virus Delivered Transgenes	12
Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease	12
Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles	12
Analysis of HIV-1-Based Lentiviral Vector Particle Composition by PacBio Long-Read Nucleic Acid Sequencing	12
Capsid-Engineering for Central Nervous System-Directed Gene Therapy with Adeno-Associated Virus Vectors	12
Adeno-Associated Virus Type 9-Mediated Gene Therapy of Choline Acetyltransferase-Deficient Mice	12
Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond	12
Engineered Human Adenoviruses of Species B and C Report Early, Intermediate Early, and Late Viral Gene Expression	12
Gene Coexpression and miRNA Regulation: A Path to Early Intervention in Colorectal Cancer	11
Liver Gene Therapy	11
Pfizer Marks Phase III Success in Hemophilia A, then Layoffs after Failure in DMD	11
Leveraging CRISPR-Cas9 for Accurate Detection of AAV-Neutralizing Antibodies: The AAV-HDR Method	11
Hemophilia Gene Therapy: The End of the Beginning?	11
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article	11
Pfizer Weighs Next Steps after DMD Therapy Linked to Boy’s Death Fails Phase III Trial	11
Therapeutic Strategy for Fabry Disease by Intravenous Administration of Adeno-Associated Virus 9 in a Symptomatic Mouse Model	11
A Review of the Cost-Effectiveness Evidence for FDA-Approved Cell and Gene Therapies	11
Recent Advances Using Genetic Therapies Against Infectious Diseases and for Vaccination	11
Akouos, Immusoft Win FDA Clearances for First-of-Their-Kind Gene Therapies	11
Preclinical Development and Characterization of Novel Adeno-Associated Viral Vectors for the Treatment of Lipoprotein Lipase Deficiency	11
Current and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed Gene Therapy	11
Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy	10
Process Development of Recombinant Adeno-Associated Virus Production Platform Results in High Production Yield and Purity	10
Association of NPC1L1 and HMGCR Gene Polymorphisms with Major Adverse Cardiac and Cerebrovascular Events in Patients with Three-Vessel Disease	10
Efficient Delivery of Adeno-Associated Virus into Inner Ear In Vivo Through Trans-Stapes Route in Adult Guinea Pig	10
A Modified Arrestin1 Increases Lactate Production in the Retina and Slows Retinal Degeneration	10
The Engineered MARCH8-Resistant Vesicular Stomatitis Virus Glycoprotein Enhances Lentiviral Vector Transduction	10
Preclinical Development of a HIV-Based Gene Therapeutic for Cystic Fibrosis Pulmonary Disease	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	10
After Sparring with Payers on Pricing, Bluebird Flies Out of Europe	10
Rational Use of Immunosuppressive Corticosteroids in Liver-Directed Adeno-Associated Virus Gene Therapy Studies	9
Trojan Horse-Like Vehicles for CRISPR-Cas Delivery: Engineering Extracellular Vesicles and Virus-Like Particles for Precision Gene Editing in Cystic Fibrosis	9
Progress, Applications and Prospects of CRISPR-Based Genome Editing Technology in Gene Therapy for Cancer and Sickle Cell Disease	9
Modulation of Immune Reaction in Hydrodynamic Gene Therapy for Hemophilia A	9
Engineering Cancer Selective Virotherapies: Are the Pieces of the Puzzle Falling into Place?	9
Marks’ Resignation Sparks Concerns on FDA Regulation of Gene Therapies	9
Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1	9
Enhanced Cochlear Transduction by AAV9 with High-Concentration Sucrose	9
International Conference on Lymphocyte Engineering Author Index	8
ESGCT 29th Annual Congress In collaboration with BSGCT Edinburgh, UK October 11–14, 2022 Abstracts	8
Unconstrained Precision Mitochondrial Genome Editing with αDdCBEs	8
Intra-Articular Adeno-Associated Virus-Mediated Proteoglycan 4 Gene Therapy for Preventing Posttraumatic Osteoarthritis	8
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	8
Single-Use Capture Purification of Adeno-Associated Viral Gene Transfer Vectors by Membrane-Based Steric Exclusion Chromatography	8
Personalizing Oncolytic Virotherapy	8
Adeno-Associated Virus-Mediated Knockdown of Agmatinase Attenuates Inflammation and Tumorigenesis in a Mouse Model of Colitis-Associated Colorectal Cancer	8
Advances in the Development and the Applications of Nonviral, Episomal Vectors for Gene Therapy	8
Ultragenyx Gene Therapies Spark Lawsuit from Lacks Family	8
The Future of Exon Skipping for Duchenne Muscular Dystrophy	8
Bone Marrow Mesenchymal Stem Cells-Derived Exosomal MicroRNA-150-3p Promotes Osteoblast Proliferation and Differentiation in Osteoporosis	8