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 2022-08-01 to 2026-08-01.)

Article	Citations
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	106
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	82
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	74
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	62
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	59
SAGA-Q: Gene Expression Differences Enable Detection of Retroviral Vectors with Mutagenic Potential	48
AVLAYAH and KRESLADI Win FDA Accelerated Approvals	46
SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye	46
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	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	42
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	36
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	35
Restoring Cancer Genomes: Functional Mutation Correction as a Platform for Precision Oncology	35
AAV.hBAG3 Gene Therapy Improves Phenotype in a Valosin Containing Protein Mouse Model of Hereditary Inclusion Body Myositis	35
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	33
AAV-Mediated Base Editing for Correction of RSPH4A Mutations in Primary Ciliary Dyskinesia: A Proof-of-Concept Study	33
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice	33
Toxicity and Biodistribution of the Oncolytic Virus VCN-01 Following Intracranial Injection in Syrian Hamsters	33
uniQure Gene Therapy Significantly Slows Huntington Disease Progression	32
Abstracts	32
Genome Editing of Pik3cd Impedes Abnormal Retinal Angiogenesis	30
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop	28
Characterizing Complex Populations of Endogenous Adeno-Associated Viruses by Single-Genome Amplification	28
Advancing Precision Medicine with Gene and Cell Therapy in Malaysia: Ethical, Legal, and Social Implications	27
Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases	25

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	24
Interview with Manlio Fusciello, PhD	23
Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-Modified AAV Vector	23
Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell	23
Modulation of AAV9 Galactose Binding Yields Novel Gene Therapy Vectors and Predicts Cross-Species Differences in Glycan Avidity	22
Meta-Analysis and Optimization of the In Vitro Immortalization Assay for Safety Assessment of Retroviral Vectors in Gene Therapy	22
Quantitation of Residual Host Cell DNA in Recombinant Adeno-Associated Virus Using Droplet Digital Polymerase Chain Reaction	21
Prenatal Intraportal Delivery of Polymeric Nanoparticles to Fetal Rhesus Monkeys ( Macaca mulatta )	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
Optimization of Alpha-1 Antitrypsin Expression from Adeno-Associated Virus Vectors	21
Metagenomic Reconstruction of Adeno-Associated Virus Genomes	20
Lived Experience with Gene Therapy	20
Genome Editing for Familial Hemophagocytic Lymphohistiocytosis: Design Principles, Challenges, and Translational Perspectives	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.	20
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
CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells	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
Interview with Dr. Ronald Crystal	18
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy	17
Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for Human Gene Therapy	17
Patient Dies in Beam Trial of Sickle Cell Disease Candidate; Company Cites Conditioning	17
Evaluation of the Toxicity and Efficacy of an Adeno-Associated Viral Vector Expressing BEST1 Delivered by Subretinal Injection in a Canine Model of Human	16
Adeno-Associated Virus-Mediated Gene Transfer of Inducible Nitric Oxide Synthase to an Animal Model of Pulmonary Hypertension	16
Update on Viral Gene Therapy Clinical Trials for Retinal Diseases	16
Prediction of Adeno-Associated Virus Fitness with a Protein Language-Based Machine Learning Model	16
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	16
An Optimized CRISPR/Cas9 Adenovirus Vector (AdZ-CRISPR) for High-Throughput Cloning of sgRNA, Using Enhanced sgRNA and Cas9 Variants	15
A Recombinant Oncolytic Influenza Virus Carrying GV1001 Triggers an Antitumor Immune Response	15
Clinical Efficacy and Safety of AdV-tk Gene Therapy for Patients with Cervical Squamous Intraepithelial Lesion: A Prospective Study	14
Introduction to ESGCT 2024 Special Issue	14
Urocortin 2 Gene Transfer for Systolic and Diastolic Dysfunction Due to Chronically Increased Left Ventricular Pressure	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
Development and Delivery of a Hands-On Short Course in Adeno-Associated Virus Manufacturing to Support Growing Workforce Needs in Gene Therapy	13
Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis	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
Long-Term Follow-Up of Patients Receiving Cell and Gene Therapy Products	13
Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles	13
The Implication of Hinge 1 and Hinge 4 in Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy	13
Progress in Respiratory Gene Therapy	13
Resolving the Unresolvable: Nanopore Sequencing as a Comprehensive Quality Control Platform for Gene Therapy Vectors	13
Chemical Epigenetic Regulation of Adeno-Associated Virus Delivered Transgenes	12
Engineered Human Adenoviruses of Species B and C Report Early, Intermediate Early, and Late Viral Gene Expression	12
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article	12
Production of Recombinant Adeno-Associated Virus Through High-Cell-Density Transfection of HEK293 Cells Based on Fed-Perfusion Culture	12
Analysis of HIV-1-Based Lentiviral Vector Particle Composition by PacBio Long-Read Nucleic Acid Sequencing	12
Matrix Protein of Vesicular Stomatitis Virus Targets the Mitochondria, Reprograms Glucose Metabolism, and Sensitizes to 2-Deoxyglucose in Glioblastoma	12
Pfizer Marks Phase III Success in Hemophilia A, then Layoffs after Failure in DMD	12
Effects of Urocortin 2 Gene Transfer on Glucose Disposal in Insulin-Resistant db/db Mice on Metformin	12

Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease	12
Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond	12
Adeno-Associated Virus Type 9-Mediated Gene Therapy of Choline Acetyltransferase-Deficient Mice	12
Pfizer Weighs Next Steps after DMD Therapy Linked to Boy’s Death Fails Phase III Trial	11
Acknowledgment of Reviewers 2025	11
Lilly, Seamless Ink Up-to-$1.12B Hearing Loss Collaboration	11
Akouos, Immusoft Win FDA Clearances for First-of-Their-Kind Gene Therapies	11
A Review of the Cost-Effectiveness Evidence for FDA-Approved Cell and Gene Therapies	11
Leveraging CRISPR-Cas9 for Accurate Detection of AAV-Neutralizing Antibodies: The AAV-HDR Method	11
A Tripartite AAV System with Engineered Lox Sites Enables Efficient Delivery of the EYS Gene for Retinal Gene Therapy	11
Liver 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	11
Targeted E3 Region Engineering Boosts Antitumor Efficacy of Conditionally Replicating Adenoviruses in an Immunocompetent Tumor Model	11
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
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
Current and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed Gene Therapy	10
Gene Coexpression and miRNA Regulation: A Path to Early Intervention in Colorectal Cancer	10
Therapeutic Strategy for Fabry Disease by Intravenous Administration of Adeno-Associated Virus 9 in a Symptomatic Mouse Model	10
Process Development of Recombinant Adeno-Associated Virus Production Platform Results in High Production Yield and Purity	10
Exploring Development Options of a Polishing Chromatography Step for AAV7 and AAV8	10
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
Unconstrained Precision Mitochondrial Genome Editing with αDdCBEs	9
Retroviral Transduction of Human CD4 ⁺ T Cells with Membrane-Attached IL-10 Generates Type 1-Like Regulatory T Cells	9
Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1	9
Marks’ Resignation Sparks Concerns on FDA Regulation of Gene Therapies	9
Engineering Cancer Selective Virotherapies: Are the Pieces of the Puzzle Falling into Place?	9
Adeno-Associated Virus-Mediated Knockdown of Agmatinase Attenuates Inflammation and Tumorigenesis in a Mouse Model of Colitis-Associated Colorectal Cancer	9
The Application of Cell and Gene-Modified Cell Therapy in the Treatment of Osteopetrosis	9
Personalizing Oncolytic Virotherapy	9
The Future of Exon Skipping for Duchenne Muscular Dystrophy	9
Trojan Horse-Like Vehicles for CRISPR-Cas Delivery: Engineering Extracellular Vesicles and Virus-Like Particles for Precision Gene Editing in Cystic Fibrosis	9
A Paradox of the Field's Own Success: Unintended Challenges in Bringing Cutting-Edge Science from the Bench to the Market	8
Stable and Predictable Lentiviral Vector Production at Clinical Scale	8
Gene Editing Pioneer Sangamo Files for Chapter 11 Bankruptcy; Agrees to Sell Assets	8
The Coming of Age of Gene Therapy for the Treatment of Human Diseases: A Regulatory Perspective	8
6 ^TH International Conference on Lymphocyte Engineering 15–17 July 2026 Milan, Italy	8
Interview with Barry Byrne, MD/PhD	8
AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys	8
Insights into Prime Editing Technology: A Deep Dive into Fundamentals, Potentials, and Challenges	8
Ethical and Regulatory Considerations for Developing Gene Therapies Involving Genome Editing	8
Multidimensional Response Surface Methodology for the Development of a Gene Editing Protocol for p67 ^phox -Deficient Chronic Granulomatous Disease	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
Safe and Efficacious Permanent Removal of Large COL7A1 Exons for Gene Reframing as a Reliable Therapeutic Strategy for Recessive Dystrophic Epidermolysis Bullosa	8