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 2022-05-01 to 2026-05-01.)

Article	Citations
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	112
SAGA-Q: Gene Expression Differences Enable Detection of Retroviral Vectors with Mutagenic Potential	87
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	80
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	62
Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease	55
SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye	54
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	47
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	46
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	43
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid	40
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	38
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	34
AVLAYAH and KRESLADI Win FDA Accelerated Approvals	34
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice	33
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	33
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient	33
Toxicity and Biodistribution of the Oncolytic Virus VCN-01 Following Intracranial Injection in Syrian Hamsters	32
Efficacy and Safety of FX201, a Novel Intra-Articular IL-1Ra Gene Therapy for Osteoarthritis Treatment, in a Rat Model	31
Meta-Analysis and Optimization of the In Vitro Immortalization Assay for Safety Assessment of Retroviral Vectors in Gene Therapy	30
An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature	30
Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders	30
AAV-Mediated Base Editing for Correction of RSPH4A Mutations in Primary Ciliary Dyskinesia: A Proof-of-Concept Study	30
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop	29
uniQure Gene Therapy Significantly Slows Huntington Disease Progression	28
Abstracts	28

Advancing Precision Medicine with Gene and Cell Therapy in Malaysia: Ethical, Legal, and Social Implications	28
Genome Editing of Pik3cd Impedes Abnormal Retinal Angiogenesis	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	24
Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell	22
Characterizing Complex Populations of Endogenous Adeno-Associated Viruses by Single-Genome Amplification	22
CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells	21
Metagenomic Reconstruction of Adeno-Associated Virus Genomes	21
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	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	21
The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy	21
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.	21
Quantitation of Residual Host Cell DNA in Recombinant Adeno-Associated Virus Using Droplet Digital Polymerase Chain Reaction	19
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	19
Prenatal Intraportal Delivery of Polymeric Nanoparticles to Fetal Rhesus Monkeys ( Macaca mulatta )	19
Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management	19
Lipid Nanoparticles for Nucleic Acid Delivery Beyond the Liver	18
Modulation of AAV9 Galactose Binding Yields Novel Gene Therapy Vectors and Predicts Cross-Species Differences in Glycan Avidity	18
Intrastriatal Delivery of a Zinc Finger Protein Targeting the Mutant HTT Gene Allele Obviates Lipid Phenotypes in Brain and Plasma in Huntington's Disease Mice	17
Interview with Dr. Ronald Crystal	17
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy	17
Adeno-Associated Virus-Mediated Gene Transfer of Inducible Nitric Oxide Synthase to an Animal Model of Pulmonary Hypertension	17
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
Update on Viral Gene Therapy Clinical Trials for Retinal Diseases	16
Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for Human Gene Therapy	16
Clinical Efficacy and Safety of AdV-tk Gene Therapy for Patients with Cervical Squamous Intraepithelial Lesion: A Prospective Study	16
A Recombinant Oncolytic Influenza Virus Carrying GV1001 Triggers an Antitumor Immune Response	16
Long-Term Follow-Up of Patients Receiving Cell and Gene Therapy Products	15
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-	15
Urocortin 2 Gene Transfer for Systolic and Diastolic Dysfunction Due to Chronically Increased Left Ventricular Pressure	15
Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles	15
Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis	15
The Implication of Hinge 1 and Hinge 4 in Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy	14
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	14
Introduction to ESGCT 2024 Special Issue	14
Development and Delivery of a Hands-On Short Course in Adeno-Associated Virus Manufacturing to Support Growing Workforce Needs in Gene Therapy	13
Progress in Respiratory Gene Therapy	13
Engineered Human Adenoviruses of Species B and C Report Early, Intermediate Early, and Late Viral Gene Expression	13
An Optimized CRISPR/Cas9 Adenovirus Vector (AdZ-CRISPR) for High-Throughput Cloning of sgRNA, Using Enhanced sgRNA and Cas9 Variants	13
Production of Recombinant Adeno-Associated Virus Through High-Cell-Density Transfection of HEK293 Cells Based on Fed-Perfusion Culture	12
A Modified Arrestin1 Increases Lactate Production in the Retina and Slows Retinal Degeneration	12
Effects of Urocortin 2 Gene Transfer on Glucose Disposal in Insulin-Resistant db/db Mice on Metformin	12
Chemical Epigenetic Regulation of Adeno-Associated Virus Delivered Transgenes	12
Pfizer Weighs Next Steps after DMD Therapy Linked to Boy’s Death Fails Phase III Trial	12
Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond	12
Matrix Protein of Vesicular Stomatitis Virus Targets the Mitochondria, Reprograms Glucose Metabolism, and Sensitizes to 2-Deoxyglucose in Glioblastoma	12
Analysis of HIV-1-Based Lentiviral Vector Particle Composition by PacBio Long-Read Nucleic Acid Sequencing	12
A Tripartite AAV System with Engineered Lox Sites Enables Efficient Delivery of the EYS Gene for Retinal Gene Therapy	12

Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease	12
Adeno-Associated Virus Type 9-Mediated Gene Therapy of Choline Acetyltransferase-Deficient Mice	12
Lilly, Seamless Ink Up-to-$1.12B Hearing Loss Collaboration	11
Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy	11
Acknowledgment of Reviewers 2025	11
Therapeutic Strategy for Fabry Disease by Intravenous Administration of Adeno-Associated Virus 9 in a Symptomatic Mouse Model	11
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article	11
Exploring Development Options of a Polishing Chromatography Step for AAV7 and AAV8	11
Efficient Delivery of Adeno-Associated Virus into Inner Ear In Vivo Through Trans-Stapes Route in Adult Guinea Pig	11
Targeted E3 Region Engineering Boosts Antitumor Efficacy of Conditionally Replicating Adenoviruses in an Immunocompetent Tumor Model	10
Process Development of Recombinant Adeno-Associated Virus Production Platform Results in High Production Yield and Purity	10
Leveraging CRISPR-Cas9 for Accurate Detection of AAV-Neutralizing Antibodies: The AAV-HDR Method	10
A Review of the Cost-Effectiveness Evidence for FDA-Approved Cell and Gene Therapies	10
Akouos, Immusoft Win FDA Clearances for First-of-Their-Kind Gene Therapies	10
CRISPR-Cas Genome Editing in Ex Vivo Human Lungs to Rewire the Translational Path of Genome-Targeting Therapeutics	10
Pfizer Marks Phase III Success in Hemophilia A, then Layoffs after Failure in DMD	10
Recent Advances Using Genetic Therapies Against Infectious Diseases and for Vaccination	10
Preclinical Development and Characterization of Novel Adeno-Associated Viral Vectors for the Treatment of Lipoprotein Lipase Deficiency	10
Gene Coexpression and miRNA Regulation: A Path to Early Intervention in Colorectal Cancer	10
Bluebird Bio Eliminating 30% of Staff in Restructuring	10
Current and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed Gene Therapy	9
Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1	9
Retroviral Transduction of Human CD4 ⁺ T Cells with Membrane-Attached IL-10 Generates Type 1-Like Regulatory T Cells	9
The Future of Exon Skipping for Duchenne Muscular Dystrophy	9
Intra-Articular Adeno-Associated Virus-Mediated Proteoglycan 4 Gene Therapy for Preventing Posttraumatic Osteoarthritis	9
Stable and Predictable Lentiviral Vector Production at Clinical Scale	9
Hemophilia Gene Therapy: The End of the Beginning?	9
Enhanced Cochlear Transduction by AAV9 with High-Concentration Sucrose	9
Adeno-Associated Virus-Mediated Knockdown of Agmatinase Attenuates Inflammation and Tumorigenesis in a Mouse Model of Colitis-Associated Colorectal Cancer	9
Trojan Horse-Like Vehicles for CRISPR-Cas Delivery: Engineering Extracellular Vesicles and Virus-Like Particles for Precision Gene Editing in Cystic Fibrosis	9
Personalizing Oncolytic Virotherapy	9
Liver Gene Therapy	9
Marks’ Resignation Sparks Concerns on FDA Regulation of Gene Therapies	9
Progress, Applications and Prospects of CRISPR-Based Genome Editing Technology in Gene Therapy for Cancer and Sickle Cell Disease	9
Engineering Cancer Selective Virotherapies: Are the Pieces of the Puzzle Falling into Place?	9
Unconstrained Precision Mitochondrial Genome Editing with αDdCBEs	9
The Coming of Age of Gene Therapy for the Treatment of Human Diseases: A Regulatory Perspective	8
Ethical and Regulatory Considerations for Developing Gene Therapies Involving Genome Editing	8
A Paradox of the Field's Own Success: Unintended Challenges in Bringing Cutting-Edge Science from the Bench to the Market	8
A Review of the Challenge of Pre-Existing Humoral Immunity in Adeno-Associated Virus Gene Therapy and Potential Solutions	8
AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys	8
SG33, a Vaccine Strain of Myxoma Virus with Oncolytic Potential, Exploits Macropinocytosis and Clathrin-Mediated Endocytosis for Entry into Pancreatic Cancer Cells	8
Interview with Barry Byrne, MD/PhD	8
Ultragenyx Gene Therapies Spark Lawsuit from Lacks Family	8
Correction to: Letter to the Editor: A Favorable Benefit–Risk Balance Maybe Expected with Replication-Defective Adenovirus-Mediated Interferon Gene Therapy for Cancer Treatm	8
Insights into Prime Editing Technology: A Deep Dive into Fundamentals, Potentials, and Challenges	8
Safe and Efficacious Permanent Removal of Large COL7A1 Exons for Gene Reframing as a Reliable Therapeutic Strategy for Recessive Dystrophic Epidermolysis Bullosa	8
ESGCT 29th Annual Congress In collaboration with BSGCT Edinburgh, UK October 11–14, 2022 Abstracts	8
Multidimensional Response Surface Methodology for the Development of a Gene Editing Protocol for p67 ^phox -Deficient Chronic Granulomatous Disease	8