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 2022-06-01 to 2026-06-01.)

Article	Citations
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	115
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid	92
AVLAYAH and KRESLADI Win FDA Accelerated Approvals	80
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	65
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	56
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	55
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	47
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	45
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	42
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	40
SAGA-Q: Gene Expression Differences Enable Detection of Retroviral Vectors with Mutagenic Potential	36
SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye	34
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	34
AAV-Mediated Base Editing for Correction of RSPH4A Mutations in Primary Ciliary Dyskinesia: A Proof-of-Concept Study	34
Toxicity and Biodistribution of the Oncolytic Virus VCN-01 Following Intracranial Injection in Syrian Hamsters	34
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice	33
Abstracts	31
Meta-Analysis and Optimization of the In Vitro Immortalization Assay for Safety Assessment of Retroviral Vectors in Gene Therapy	31
Genome Editing of Pik3cd Impedes Abnormal Retinal Angiogenesis	30
Characterizing Complex Populations of Endogenous Adeno-Associated Viruses by Single-Genome Amplification	30
uniQure Gene Therapy Significantly Slows Huntington Disease Progression	30
Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell	30
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop	29
Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases	28
Advancing Precision Medicine with Gene and Cell Therapy in Malaysia: Ethical, Legal, and Social Implications	28

Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders	27
An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature	26
Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-Modified AAV Vector	24
Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management	24
Prenatal Intraportal Delivery of Polymeric Nanoparticles to Fetal Rhesus Monkeys ( Macaca mulatta )	23
Quantitation of Residual Host Cell DNA in Recombinant Adeno-Associated Virus Using Droplet Digital Polymerase Chain Reaction	22
Metagenomic Reconstruction of Adeno-Associated Virus Genomes	22
CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells	22
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
Lived Experience with Gene Therapy	21
Modulation of AAV9 Galactose Binding Yields Novel Gene Therapy Vectors and Predicts Cross-Species Differences in Glycan Avidity	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
Update on Viral Gene Therapy Clinical Trials for Retinal Diseases	19
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	19
Optimization of Alpha-1 Antitrypsin Expression from Adeno-Associated Virus Vectors	19
Lipid Nanoparticles for Nucleic Acid Delivery Beyond the Liver	19
Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for Human Gene Therapy	18
Interview with Dr. Ronald Crystal	18
Patient Dies in Beam Trial of Sickle Cell Disease Candidate; Company Cites Conditioning	18
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy	18
A Recombinant Oncolytic Influenza Virus Carrying GV1001 Triggers an Antitumor Immune Response	18
Prediction of Adeno-Associated Virus Fitness with a Protein Language-Based Machine Learning Model	17
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
Adeno-Associated Virus-Mediated Gene Transfer of Inducible Nitric Oxide Synthase to an Animal Model of Pulmonary Hypertension	17
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	16
Progress in Respiratory Gene Therapy	16
Clinical Efficacy and Safety of AdV-tk Gene Therapy for Patients with Cervical Squamous Intraepithelial Lesion: A Prospective Study	16
An Optimized CRISPR/Cas9 Adenovirus Vector (AdZ-CRISPR) for High-Throughput Cloning of sgRNA, Using Enhanced sgRNA and Cas9 Variants	16
Urocortin 2 Gene Transfer for Systolic and Diastolic Dysfunction Due to Chronically Increased Left Ventricular Pressure	16
Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles	16
The Implication of Hinge 1 and Hinge 4 in Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy	15
Introduction to ESGCT 2024 Special Issue	15
Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis	14
Development and Delivery of a Hands-On Short Course in Adeno-Associated Virus Manufacturing to Support Growing Workforce Needs in Gene Therapy	14
Effects of Urocortin 2 Gene Transfer on Glucose Disposal in Insulin-Resistant db/db Mice on Metformin	13
Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond	13
Long-Term Follow-Up of Patients Receiving Cell and Gene Therapy Products	13
Adeno-Associated Virus Type 9-Mediated Gene Therapy of Choline Acetyltransferase-Deficient Mice	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
Chemical Epigenetic Regulation of Adeno-Associated Virus Delivered Transgenes	13
Matrix Protein of Vesicular Stomatitis Virus Targets the Mitochondria, Reprograms Glucose Metabolism, and Sensitizes to 2-Deoxyglucose in Glioblastoma	13
Production of Recombinant Adeno-Associated Virus Through High-Cell-Density Transfection of HEK293 Cells Based on Fed-Perfusion Culture	12
Leveraging CRISPR-Cas9 for Accurate Detection of AAV-Neutralizing Antibodies: The AAV-HDR Method	12
Pfizer Marks Phase III Success in Hemophilia A, then Layoffs after Failure in DMD	12
Pfizer Weighs Next Steps after DMD Therapy Linked to Boy’s Death Fails Phase III Trial	12
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article	12
Analysis of HIV-1-Based Lentiviral Vector Particle Composition by PacBio Long-Read Nucleic Acid Sequencing	12
Preclinical Development and Characterization of Novel Adeno-Associated Viral Vectors for the Treatment of Lipoprotein Lipase Deficiency	12
Liver 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

Engineered Human Adenoviruses of Species B and C Report Early, Intermediate Early, and Late Viral Gene Expression	12
Process Development of Recombinant Adeno-Associated Virus Production Platform Results in High Production Yield and Purity	12
Akouos, Immusoft Win FDA Clearances for First-of-Their-Kind Gene Therapies	12
Exploring Development Options of a Polishing Chromatography Step for AAV7 and AAV8	11
Gene Coexpression and miRNA Regulation: A Path to Early Intervention in Colorectal Cancer	11
A Tripartite AAV System with Engineered Lox Sites Enables Efficient Delivery of the EYS Gene for Retinal Gene Therapy	11
Acknowledgment of Reviewers 2025	11
Lilly, Seamless Ink Up-to-$1.12B Hearing Loss Collaboration	11
Targeted E3 Region Engineering Boosts Antitumor Efficacy of Conditionally Replicating Adenoviruses in an Immunocompetent Tumor Model	11
A Modified Arrestin1 Increases Lactate Production in the Retina and Slows Retinal Degeneration	10
Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1	10
Current and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed Gene Therapy	10
Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy	10
Recent Advances Using Genetic Therapies Against Infectious Diseases and for Vaccination	10
Efficient Delivery of Adeno-Associated Virus into Inner Ear In Vivo Through Trans-Stapes Route in Adult Guinea Pig	10
Therapeutic Strategy for Fabry Disease by Intravenous Administration of Adeno-Associated Virus 9 in a Symptomatic Mouse Model	10
Hemophilia Gene Therapy: The End of the Beginning?	10
CRISPR-Cas Genome Editing in Ex Vivo Human Lungs to Rewire the Translational Path of Genome-Targeting Therapeutics	10
Retroviral Transduction of Human CD4 ⁺ T Cells with Membrane-Attached IL-10 Generates Type 1-Like Regulatory T Cells	10
A Review of the Cost-Effectiveness Evidence for FDA-Approved Cell and Gene Therapies	10
Marks’ Resignation Sparks Concerns on FDA Regulation of Gene Therapies	9
Adeno-Associated Virus-Mediated Knockdown of Agmatinase Attenuates Inflammation and Tumorigenesis in a Mouse Model of Colitis-Associated Colorectal Cancer	9
Engineering Cancer Selective Virotherapies: Are the Pieces of the Puzzle Falling into Place?	9
Stable and Predictable Lentiviral Vector Production at Clinical Scale	9
Multidimensional Response Surface Methodology for the Development of a Gene Editing Protocol for p67 ^phox -Deficient Chronic Granulomatous Disease	9
Personalizing Oncolytic Virotherapy	9
The Future of Exon Skipping for Duchenne Muscular Dystrophy	9
Unconstrained Precision Mitochondrial Genome Editing with αDdCBEs	9
Interview with Barry Byrne, MD/PhD	9
The Application of Cell and Gene-Modified Cell Therapy in the Treatment of Osteopetrosis	9
Progress, Applications and Prospects of CRISPR-Based Genome Editing Technology in Gene Therapy for Cancer and Sickle Cell Disease	9
Enhanced Cochlear Transduction by AAV9 with High-Concentration Sucrose	9
Trojan Horse-Like Vehicles for CRISPR-Cas Delivery: Engineering Extracellular Vesicles and Virus-Like Particles for Precision Gene Editing in Cystic Fibrosis	9
Ethical and Regulatory Considerations for Developing Gene Therapies Involving Genome Editing	9
A Paradox of the Field's Own Success: Unintended Challenges in Bringing Cutting-Edge Science from the Bench to the Market	9
AAV-Mediated Gene Transfer of WDR45 Corrects Neurological Deficits in the Mouse Model of Beta-Propeller Protein-Associated Neurodegeneration	8
Medicinal Products Based on Adeno-Associated Viral Vectors: A Regulatory Perspective on the Potential Risk of Insertion-Mediated Tumorigenesis	8
Comprehensive Review of Osteogenesis Imperfecta: Current Treatments and Future Innovations	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
ESGCT 29th Annual Congress In collaboration with BSGCT Edinburgh, UK October 11–14, 2022 Abstracts	8
Intracisternal AAV9-MAG- hABCD1 Vector Reverses Motor Deficits in Adult Adrenomyeloneuropathy Mice	8
Long-Term Functional Correction of Pompe Disease and Increased α-Glucosidase Expression after Gene Therapy with Novel Combinations of Muscle-Targeted Transcriptional Cis	8
Ultragenyx Gene Therapies Spark Lawsuit from Lacks Family	8
Safe and Efficacious Permanent Removal of Large COL7A1 Exons for Gene Reframing as a Reliable Therapeutic Strategy for Recessive Dystrophic Epidermolysis Bullosa	8
SG33, a Vaccine Strain of Myxoma Virus with Oncolytic Potential, Exploits Macropinocytosis and Clathrin-Mediated Endocytosis for Entry into Pancreatic Cancer Cells	8
The 2024 Nobel Prize: Impact of the Discovery of miRNA on the Field of Gene Therapy	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
The Coming of Age of Gene Therapy for the Treatment of Human Diseases: A Regulatory Perspective	8
Insights into Prime Editing Technology: A Deep Dive into Fundamentals, Potentials, and Challenges	8