OOIR: Observatory of International Research

Papers

(The TQCC of Human Gene Therapy is 6. 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 2021-04-01 to 2025-04-01.)

Article	Citations
Reuben Matalon, MD, PhD, FACMG (1935–2021)	73
Adeno-Associated Viral Vector-Delivered Pannexin-1 Mimetic Peptide Alleviates Airway Inflammation in an Allergen-Sensitized Mouse Model	72
Efficient Gene Transfer to Kidney Using a Lentiviral Vector Pseudotyped with Zika Virus Envelope Glycoprotein	72
Strategies to Attenuate Myocardial Infarction and No-Reflow Through Preservation of Vascular Integrity by Pigment Epithelium-Derived Factor	70
Correction to: Efficacy and Safety of Adeno-Associated Virus-Based Clinical Gene Therapy for Hemophilia: A Systematic Review and Meta-Analysis, by Han et al. Hum Gene Ther 2024;35(3-4):9	51
Update: Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology by Juliette Hordeaux et al. Hum Gene Ther 2020;31:15–16. DOI: 10.1089/hum.2020.167	48
Heart of the Matter: AAV8 Improves Cardiac Function	46
Letter to the Editor: mRNA Vaccines in Urological Malignancies	45
Effects of Urocortin 2 Gene Transfer on Glucose Disposal in Insulin-Resistant db/db Mice on Metformin	42
Correction to: Induced CD20 Expression on B-Cell Malignant Cells Heightened the Cytotoxic Activity of Chimeric Antigen Receptor Engineered T Cells, by Xu et al. Hum Gene Ther 2019;30(4):497–510	40
Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease	40
Long Non-Coding RNAs, Cell Cycle, and Human Breast Cancer	36
Advances in Off-Target Detection for CRISPR-Based Genome Editing	36
Keys to Delivery: An Interview with Hildegard Büning	36
Ex Vivo Gene Therapy in Organ Transplantation: Considerations and Clinical Translation	36
Characterization and Functional Analysis of CD44v6.CAR T Cells Endowed with a New Low-Affinity Nerve Growth Factor Receptor-Based Spacer	31
Induction of Robust Type I Interferon Levels by Oncolytic Reovirus Requires Both Viral Replication and Interferon-α/β Receptor Signaling	28
Gene Supplementation in Mice Heterozygous for the D477G RPE65 Variant Implicated in Autosomal Dominant Retinitis Pigmentosa	28
First Positive Clinical Data for In Vivo Genome Editing in Humans Opens “New Era of Medicine”	28
Correction to: Inhibition of MicroRNA-9-5p Protects Against Cardiac Remodeling Following Myocardial Infarction in Mice, by Xiao et al. Hum Gene Ther 2019;30(3):286–301; doi: 10.1089/hum.	28
A Review of the Legislation of Direct-to-Consumer Genetic Testing in China	28
ESGCTCollaborative Virtual CongressAbstract Author Index	28
Prophylactic Prednisolone Promotes AAV5 Hepatocyte Transduction Through the Novel Mechanism of AAV5 Coreceptor Platelet-Derived Growth Factor Receptor Alpha Upregulation and Innate Immune Suppression	27
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid	25
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	25

Bluebird Bio Chief Financial Officer Resigns as Company Raises “Going Concern” Doubts	24
Dose-Dependent Microdystrophin Expression Enhancement in Cardiac Muscle by a Cardiac-Specific Regulatory Element	24
Call for Papers: Special Issue on Toxicity And Safety in Clinical AAV Gene Therapy	24
Preclinical Safety and Biodistribution in Mice Following Single-Dose Intramuscular Inoculation of Tumor DNA Vaccine by Electroporation	23
Gene Therapy and the Use of Animal Models: Why Mice Alone Are Not Sufficient	23
Efficacious Androgen Hormone Administration in Combination with Adeno-Associated Virus Vector-Mediated Gene Therapy in Female Mice with Pompe Disease	22
PASTE, Don't Cut: Genome Editing Tool Looks Beyond CRISPR and Prime	22
Preclinical Assessment of a Gene-Editing Approach in a Mouse Model of Mitochondrial Neurogastrointestinal Encephalomyopathy	21
Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies	21
Matrix Protein of Vesicular Stomatitis Virus Targets the Mitochondria, Reprograms Glucose Metabolism, and Sensitizes to 2-Deoxyglucose in Glioblastoma	21
Orchard Therapeutics Gains First U.S. Approval for a Metachromatic Leukodystrophy Gene Therapy	21
Suicide Gene Delivery System Mediated by Ultrasound-Targeted Microbubble Destruction: A Promising Strategy for Cancer Therapy	20
Optimization of the Administration Strategy for the Armed Oncolytic Adenovirus ZD55-IL-24 in Both Immunocompromised and Immunocompetent Mouse Models	20
ESGCT Abstract Author Index	19
Acknowledgment of Reviewers 2023	19
Production of Recombinant Adeno-Associated Virus Through High-Cell-Density Transfection of HEK293 Cells Based on Fed-Perfusion Culture	19
Ocular Inflammation with Anti-Vascular Endothelial Growth Factor Treatments	18
Roche, Ascidian Launch Up-to-$1.8B RNA Exon Editing Collaboration	17
Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage	17
Chemical Epigenetic Regulation of Adeno-Associated Virus Delivered Transgenes	17
Fusion of Rabies Virus Glycoprotein or gh625 to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II	17
Adeno-Associated Virus Type 9-Mediated Gene Therapy of Choline Acetyltransferase-Deficient Mice	16
An Improved Lentiviral Fluorescent Genetic Barcoding Approach Distinguishes Hematopoietic Stem Cell Properties in MultiplexedIn VivoExperiments	15
Analysis of HIV-1-Based Lentiviral Vector Particle Composition by PacBio Long-Read Nucleic Acid Sequencing	15
Minimally Humanized Ezh2 Exon-18 Mouse Cell Lines Validate Preclinical CRISPR/Cas9 Approach to Treat Weaver Syndrome	15
Hum Gene Therapy Briefs March 2025	15
Concise Analysis of Single-Stranded DNA of Recombinant Adeno-Associated Virus By Automated Electrophoresis System	14
Case Study Cites Immune Reaction to High Adeno-Associated Virus Dose in Explaining Duchenne Muscular Dystrophy Trial Death	14
Isolating Natural Adeno-Associated Viruses from Primate Tissues with a High-Fidelity Polymerase	14
Prevalence Study of Cellular Capsid-Specific Immune Responses to AAV2, 4, 5, 8, 9, and rh10 in Healthy Donors	14
Engineered Human Adenoviruses of Species B and C Report Early, Intermediate Early, and Late Viral Gene Expression	14
Therapeutic Efficacy of an Oncolytic Influenza Virus Carrying an Antibody Against Programmed Cell Death 1 in Hepatocellular Carcinoma	14
Letter to the Editor: Hematopoietic Stem and Progenitor Cell Mobilization and Collection for Patients Diagnosed with Osteopetrosis and Hurler Syndrome	13
Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model	13
Restoring Ciliary Function: Gene Therapeutics for Primary Ciliary Dyskinesia	13
Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases	13
Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System	13
Long-Term Follow-Up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy	13
Capsid-Engineering for Central Nervous System-Directed Gene Therapy with Adeno-Associated Virus Vectors	13
Graphite Bio Pauses Lead Gene Editing Program in Sickle Cell Disease	13
Pancreatic Cancer Cell and Gene Biotherapies: Past, Present, and Future	13
Dexamethasone Transiently Enhances Transgene Expression in the Liver When Administered at Late-Phase Post Long-Term Adeno-Associated Virus Transduction	12
Development and Validation of an Anion Exchange High-Performance Liquid Chromatography Method for Analysis of Empty Capsids and Capsids Encapsidating Genetic Material in a Purified Preparation of Reco	12
Innate Immune Sensing of Adeno-Associated Virus Vectors	12
Prevalence of Pre-Existing Neutralizing Antibodies Against Adeno-Associated Virus Serotypes 1, 2, 5, 6, 8, and 9 in Sera of Different Pig Strains	12
Viral Vectors Expressing Interleukin 2 for Cancer Immunotherapy	12
Peripheral Cellular Immune Responses Induced by Subretinal Adeno-Associated Virus Gene Transfer Can Be Restrained by the Subretinal-Associated Immune Inhibition Mechanism	12
Current Update on Severe Acute Respiratory Syndrome Coronavirus 2 Vaccine Development with a Special Emphasis on Gene Therapy Viral Vector Design and Construction for Vaccination	12
Duchenne Muscular Dystrophy Gene Therapy in 2023: Status, Perspective, and Beyond	12
Characterization of Adeno-Associated Virus Capsid Proteins with Two Types of VP3-Related Components by Capillary Gel Electrophoresis and Mass Spectrometry	12

Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy	11
Optimized Adeno-Associated Virus Vectors for Efficient Transduction of Human Retinal Organoids	11
Bone Marrow Mesenchymal Stem Cell-Mediated Radiosensitive Promoter-Combined Sodium Iodide Symporter for the Treatment of Breast Cancer	11
Choosing the Right Tool for Genetic Engineering: Clinical Lessons from Chimeric Antigen Receptor-T Cells	11
M11: A Tropism-Modified Oncolytic Adenovirus Arming with a Tumor-Homing Peptide for Advanced Ovarian Cancer Therapies	11
In Vivo Potency Testing of Subretinal rAAV5.hCNGB1 Gene Therapy in the Cngb1 Knockout Mouse Model of Retinitis Pigmentosa	11
Advances in MicroRNA Therapeutics: From Preclinical to Clinical Studies	11
Beam Results Show First Genetic Correction of Disease-Causing Mutation	11
After Patient Death, FDA Places Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Trial	11
A Review of the Cost-Effectiveness Evidence for FDA-Approved Cell and Gene Therapies	11
Biodistribution of Transplanted Hematopoietic Precursor Cells Injected Through Different Administration Routes in Newborn Mice	10
Gene Therapy in Global Health: Meeting the Needs of Chinese Patients with Beta-Thalassemia	10
pNaSS-Grafted PCL Film-Guided rAAV TGF-β Gene Therapy Activates the Chondrogenic Activities in Human Bone Marrow Aspirates	10
Lentiviral Gene Therapy of Chronic Granulomatous Disease: Functional Assessment of Universal and Tissue-Specific Promoters	10
A Beautiful Mind and the Heart of an Explorer	10
Biotechnological advances in gene therapy of hematopoietic stem cells: Systematic review and meta-analysis	10
Combination Immunotherapy of Oncolytic Flu-Vectored Virus and Programmed Cell Death 1 Blockade Enhances Antitumor Activity in Hepatocellular Carcinoma	10
Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice	10
Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia	10
Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction	10
Adeno-Associated Virus Gene Therapy Development: Early Planning and Regulatory Considerations to Advance the Platform Vector Gene Therapy Program	10
Short-Term Steroid Treatment of Rhesus Macaque Increases Transduction	10
Finnish Society of Gene and Cell Therapy: A Visionary and Creative Player in the Field	10
Remembrances of Nicholas Muzyczka, PhD	10
Vector-Mediated Delivery of Human Major Histocompatibility Complex-I into Hepatocytes Enables Investigation of T Cell Receptor-Redirected Hepatitis B Virus-Specific T Cells in Mice, and in Macaque Cel	9
Multimodular Optimization of Chemically Regulated T Cell Switches Demonstrates Flexible and Interchangeable Nature of Immune Cell Signaling Domains	9
A Cautiously Optimistic Outlook of a Designer Therapy for 1% of Duchenne Muscular Dystrophy Patients	9
pUDK-HGF Gene Therapy to Relieve CLI Rest Pain and Ulcer: A Phase II, Double-Blind, Randomized Placebo-Controlled Trial	9
Recent Advances Using Genetic Therapies Against Infectious Diseases and for Vaccination	9
Treatment of a Hemophilia B Mouse Model with Platelet-Targeted Expression of Factor IX Padua	9
CRISPR-Cas Genome Editing in Ex Vivo Human Lungs to Rewire the Translational Path of Genome-Targeting Therapeutics	9
Acknowledgment of Reviewers 2022	9
Akouos, Immusoft Win FDA Clearances for First-of-Their-Kind Gene Therapies	9
The RNA World: The Best of Times, the Worst of Times	9
Bluebird Bio Eliminating 30% of Staff in Restructuring	9
Seroprevalence of Adeno-Associated Virus Neutralizing Antibodies in Males with Duchenne Muscular Dystrophy	8
Gutless Helper-Dependent and First-Generation HAdV5 Vectors Have Similar Mechanical Properties and Common Transduction Mechanisms	8
Strategies for Optimization of the Clustered Regularly Interspaced Short Palindromic Repeat-Based Genome Editing System for Enhanced Editing Specificity	8
Efficient Delivery of Adeno-Associated Virus into Inner Ear In Vivo Through Trans-Stapes Route in Adult Guinea Pig	8
Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors	8
Chemical Modifications of the Capsid for Redirecting and Improving the Efficacy of Adeno-Associated Virus Vectors	8
Boy Dosed with Pfizer’s Duchenne Muscular Dystrophy Gene Therapy Dies a Year After Phase II Trial	8
Identification of Broadly Applicable Adeno-Associated Virus Vectors by Systematic Comparison of Commonly Used Capsid Variants In Vitro	8
Process Development of Recombinant Adeno-Associated Virus Production Platform Results in High Production Yield and Purity	8
Drug Repositioning for Amyloid Transthyretin Amyloidosis by Interactome Network Corrected by Graph Neural Networks and Transcriptome Analysis	7
Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy	7
Leveraging CRISPR-Cas9 for Accurate Detection of AAV-Neutralizing Antibodies: The AAV-HDR Method	7
Construction of Plasmid DNA Expressing Two Isoforms of Insulin-Like Growth Factor-1 and Its Effects on Skeletal Muscle Injury Models	7
Modulating Oncolytic Adenovirus Immunotherapy by Driving Two Axes of the Immune System by Expressing 4-1BBL and CD40L	7
Short Time to Market and Forward Planning Will Enable Cell Therapies to Deliver R&D Pipeline Value	7
Repeated Systemic Dosing of Adeno-Associated Virus Vectors in Immunocompetent Mice After Blockade of T Cell Costimulatory Pathways	6
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article	6
Absence of Significant Off-Target Splicing Variation with a U7snRNA Vector Targeting DMD Exon 2 Duplications	6
In Vivo Hematopoietic Stem Cell Gene Therapy for SARS-CoV2 Infection Using a Decoy Receptor	6
ESGCT Abstract Author Index	6
Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vector Gene Therapy Exp	6
Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease	6
Call for Papers: Special Issue on Ocular Gene Therapy	6
Establishment of the Effectiveness of Early Versus Late Stem Cell Gene Therapy in Mucopolysaccharidosis II for Treating Central Versus Peripheral Disease	6
Efficacy and Safety of Dual-Targeting Chimeric Antigen Receptor-T Therapy for Relapsed or Refractory B Cell Lymphoid Malignancies: A Systematic Review and Meta-Analysis	6
Review of Safety Data for Adenovirus 5 as a Delivery Vector for Intratumoral Cancer Gene Therapy	6
Therapeutic Strategy for Fabry Disease by Intravenous Administration of Adeno-Associated Virus 9 in a Symptomatic Mouse Model	6
Pfizer Weighs Next Steps after DMD Therapy Linked to Boy’s Death Fails Phase III Trial	6
Adeno-Associated Virus-Mediated Gene Therapy for Patients' Fibroblasts, Induced Pluripotent Stem Cells, and a Mouse Model of Congenital Adrenal Hyperplasia	6
Hemophilia Gene Therapy: The End of the Beginning?	6
Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities	6
Adeno-Associated Virus Vector-Mediated Expression of Antirespiratory Syncytial Virus Antibody Prevents Infection in Mouse Airways	6
Preclinical Development and Characterization of Novel Adeno-Associated Viral Vectors for the Treatment of Lipoprotein Lipase Deficiency	6
Optimized Protocol for Accurate Titration of Adeno-Associated Virus Vectors	6
Pfizer Marks Phase III Success in Hemophilia A, then Layoffs after Failure in DMD	6
Abstracts	6
Abstract Author Index	6
Efficacy and Safety of Adeno-Associated Virus-Based Clinical Gene Therapy for Hemophilia: A Systematic Review and Meta-Analysis	6
The Effect of Rapamycin and Ibrutinib on Antibody Responses to Adeno-Associated Virus Vector-Mediated Gene Transfer	6