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 2021-11-01 to 2025-11-01.)

Article	Citations
Bluebird Bio Chief Financial Officer Resigns as Company Raises “Going Concern” Doubts	94
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	82
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	66
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient	60
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	49
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid	44
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	43
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	43
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	43
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	42
Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease	42
Strategies to Attenuate Myocardial Infarction and No-Reflow Through Preservation of Vascular Integrity by Pigment Epithelium-Derived Factor	35
SNAC: A Single-Nuclei Atlas of Capsid Distribution in Nonhuman Primate Eye	35
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	33
Isolating Natural Adeno-Associated Viruses from Primate Tissues with a High-Fidelity Polymerase	30
Immune Regulatory Effect of Osteopontin Gene Therapy in a Murine Model of Multidrug Resistant Pulmonary Tuberculosis	29
Efficacy and Safety of FX201, a Novel Intra-Articular IL-1Ra Gene Therapy for Osteoarthritis Treatment, in a Rat Model	28
Toxicity and Biodistribution of the Oncolytic Virus VCN-01 Following Intracranial Injection in Syrian Hamsters	28
Meta-Analysis and Optimization of the In Vitro Immortalization Assay for Safety Assessment of Retroviral Vectors in Gene Therapy	27
Characterizing Complex Populations of Endogenous Adeno-Associated Viruses by Single-Genome Amplification	27
Abstracts	27
Adeno-Associated Virus-Mediated Interleukin-12 Gene Expression Alleviates Lung Inflammation and Type 2 T-Helper-Responses in Ovalbumin-Sensitized Asthmatic Mice	27
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop	26
uniQure Gene Therapy Significantly Slows Huntington Disease Progression	26
ESGCT 2021	26

Limitations of Dual-Single Guide RNA CRISPR Strategies for the Treatment of Central Nervous System Genetic Disorders	26
Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-Modified AAV Vector	25
Immune Responses to Adeno-Associated Virus-Mediated CRISPR Therapy	25
Gene Therapeutics for Surfactant Dysfunction Disorders: Targeting the Alveolar Type 2 Epithelial Cell	25
Genome Editing of Pik3cd Impedes Abnormal Retinal Angiogenesis	23
Advancing Precision Medicine with Gene and Cell Therapy in Malaysia: Ethical, Legal, and Social Implications	23
Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases	22
An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature	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	21
Lived Experience with Gene Therapy	20
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	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	18
The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy	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
Quantitation of Residual Host Cell DNA in Recombinant Adeno-Associated Virus Using Droplet Digital Polymerase Chain Reaction	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
Lipid Nanoparticles for Nucleic Acid Delivery Beyond the Liver	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	17
rAAV Production and Titration at the Microscale for High-Throughput Screening	17
Adeno-Associated Virus-Mediated Gene Transfer of Inducible Nitric Oxide Synthase to an Animal Model of Pulmonary Hypertension	16
Acknowledgment of Reviewers 2021	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
Interview with Dr. Ronald Crystal	16
Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for Human Gene Therapy	16
Patient Dies in Beam Trial of Sickle Cell Disease Candidate; Company Cites Conditioning	15
Prediction of Adeno-Associated Virus Fitness with a Protein Language-Based Machine Learning Model	15
Update on Viral Gene Therapy Clinical Trials for Retinal Diseases	15
An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy	15
Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles	14
Development and Delivery of a Hands-On Short Course in Adeno-Associated Virus Manufacturing to Support Growing Workforce Needs in Gene Therapy	14
Genetic Modification of Limbs UsingEx VivoMachine Perfusion	14
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-	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
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
Progress in Respiratory Gene Therapy	14
A Recombinant Oncolytic Influenza Virus Carrying GV1001 Triggers an Antitumor Immune Response	14
Matrix Protein of Vesicular Stomatitis Virus Targets the Mitochondria, Reprograms Glucose Metabolism, and Sensitizes to 2-Deoxyglucose in Glioblastoma	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	13
Onasemnogene Abeparvovec in Type 1 Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis	13
Analysis of HIV-1-Based Lentiviral Vector Particle Composition by PacBio Long-Read Nucleic Acid Sequencing	13
The Implication of Hinge 1 and Hinge 4 in Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy	13
Engineered Human Adenoviruses of Species B and C Report Early, Intermediate Early, and Late Viral Gene Expression	12
Chemical Epigenetic Regulation of Adeno-Associated Virus Delivered Transgenes	12
Akouos, Immusoft Win FDA Clearances for First-of-Their-Kind Gene Therapies	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

Bluebird Bio Eliminating 30% of Staff in Restructuring	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
Leveraging CRISPR-Cas9 for Accurate Detection of AAV-Neutralizing Antibodies: The AAV-HDR Method	12
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
Pfizer Marks Phase III Success in Hemophilia A, then Layoffs after Failure in DMD	11
Therapeutic Strategy for Fabry Disease by Intravenous Administration of Adeno-Associated Virus 9 in a Symptomatic Mouse Model	11
A Modified Arrestin1 Increases Lactate Production in the Retina and Slows Retinal Degeneration	11
Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities	11
Current and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed Gene Therapy	11
Process Development of Recombinant Adeno-Associated Virus Production Platform Results in High Production Yield and Purity	11
Efficient Delivery of Adeno-Associated Virus into Inner Ear In Vivo Through Trans-Stapes Route in Adult Guinea Pig	11
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article	11
Gene Coexpression and miRNA Regulation: A Path to Early Intervention in Colorectal Cancer	10
Preclinical Development and Characterization of Novel Adeno-Associated Viral Vectors for the Treatment of Lipoprotein Lipase Deficiency	10
Pfizer Weighs Next Steps after DMD Therapy Linked to Boy’s Death Fails Phase III Trial	10
CRISPR-Cas Genome Editing in Ex Vivo Human Lungs to Rewire the Translational Path of Genome-Targeting Therapeutics	10
Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1	9
Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy	9
Modulation of Immune Reaction in Hydrodynamic Gene Therapy for Hemophilia A	9
Liver Gene Therapy	9
Trojan Horse-Like Vehicles for CRISPR-Cas Delivery: Engineering Extracellular Vesicles and Virus-Like Particles for Precision Gene Editing in Cystic Fibrosis	9
Hemophilia Gene Therapy: The End of the Beginning?	9
Unconstrained Precision Mitochondrial Genome Editing with αDdCBEs	8
Engineering Cancer Selective Virotherapies: Are the Pieces of the Puzzle Falling into Place?	8
Adeno-Associated Virus-Mediated Knockdown of Agmatinase Attenuates Inflammation and Tumorigenesis in a Mouse Model of Colitis-Associated Colorectal Cancer	8
International Conference on Lymphocyte Engineering Author Index	8
ESGCT 29th Annual Congress In collaboration with BSGCT Edinburgh, UK October 11–14, 2022 Abstracts	8
Personalizing Oncolytic Virotherapy	8
Progress, Applications and Prospects of CRISPR-Based Genome Editing Technology in Gene Therapy for Cancer and Sickle Cell Disease	8
Enhanced Cochlear Transduction by AAV9 with High-Concentration Sucrose	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
Interview with Barry Byrne, MD/PhD	8
Rational Use of Immunosuppressive Corticosteroids in Liver-Directed Adeno-Associated Virus Gene Therapy Studies	8
The Future of Exon Skipping for Duchenne Muscular Dystrophy	8
Marks’ Resignation Sparks Concerns on FDA Regulation of Gene Therapies	8
Intra-Articular Adeno-Associated Virus-Mediated Proteoglycan 4 Gene Therapy for Preventing Posttraumatic Osteoarthritis	8
Ultragenyx Gene Therapies Spark Lawsuit from Lacks Family	8
AAV-Mediated Gene Transfer of WDR45 Corrects Neurological Deficits in the Mouse Model of Beta-Propeller Protein-Associated Neurodegeneration	7
The 2024 Nobel Prize: Impact of the Discovery of miRNA on the Field of Gene Therapy	7
Insights into Prime Editing Technology: A Deep Dive into Fundamentals, Potentials, and Challenges	7
Adenoviral Transduction ofDickkopf-1Alleviates Silica-Induced Silicosis Development in Lungs of Mice	7
Safe and Efficacious Permanent Removal of Large COL7A1 Exons for Gene Reframing as a Reliable Therapeutic Strategy for Recessive Dystrophic Epidermolysis Bullosa	7
AAV5 Delivery of CRISPR/Cas9 Mediates Genome Editing in the Lungs of Young Rhesus Monkeys	7
Rosalind Franklin Society Proudly Announces the 2024 Award Recipient for Human Gene Therapy	7
Akouos Therapy Restores 11-Year-Old Boy's Hearing After 1 Month	7
Targeting Oncolytic Adenoviruses to Cancer Cells Using a Designed Ankyrin Repeat Protein Lipocalin-2 Fusion Protein	7
A Paradox of the Field's Own Success: Unintended Challenges in Bringing Cutting-Edge Science from the Bench to the Market	7
Development and Application of a Liquid Chromatography-Mass Spectrometry Method for Residual Iodixanol Quantification in AAV-Based Gene Therapy Product Development	7
Comprehensive Review of Osteogenesis Imperfecta: Current Treatments and Future Innovations	7
Genome Editing of Mammalian Cells Through RNA Transcript-Mediated Homologous Recombination Repair	7
Intracisternal AAV9-MAG-hABCD1 Vector Reverses Motor Deficits in Adult Adrenomyeloneuropathy Mice	7
HIV Tat-Conjugated Histone H3 Peptides Induce Tumor Cell Death Via Cellular Stress Responses	7
The Coming of Age of Gene Therapy for the Treatment of Human Diseases: A Regulatory Perspective	7
Biodistribution and Tolerability of AAV-PHP.B-CBh-SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles	7
Multidimensional Response Surface Methodology for the Development of a Gene Editing Protocol for p67^phox-Deficient Chronic Granulomatous Disease	7
Gene Therapy for Immunoglobulin E, Complement-Mediated, and Eosinophilic Disorders	7