OOIR: Observatory of International Research

Papers

(The median citation count of Human Gene Therapy is 3. 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
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
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
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
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
Quantitation of Residual Host Cell DNA in Recombinant Adeno-Associated Virus Using Droplet Digital Polymerase Chain Reaction	22
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
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	19
Lipid Nanoparticles for Nucleic Acid Delivery Beyond the Liver	19
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
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
Rosalind Franklin Society Proudly Announces the 2023 Award Recipient for Human Gene Therapy	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
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	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
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
Effects of Urocortin 2 Gene Transfer on Glucose Disposal in Insulin-Resistant db/db Mice on Metformin	13
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
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
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
Exploring Development Options of a Polishing Chromatography Step for AAV7 and AAV8	11
Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease, Type C1	10
Therapeutic Strategy for Fabry Disease by Intravenous Administration of Adeno-Associated Virus 9 in a Symptomatic Mouse Model	10
CRISPR-Cas Genome Editing in Ex Vivo Human Lungs to Rewire the Translational Path of Genome-Targeting Therapeutics	10
A Review of the Cost-Effectiveness Evidence for FDA-Approved Cell and Gene Therapies	10
Recent Advances Using Genetic Therapies Against Infectious Diseases and for Vaccination	10
A Modified Arrestin1 Increases Lactate Production in the Retina and Slows Retinal Degeneration	10
Current and Emerging Issues in Adeno-Associated Virus Vector-Mediated Liver-Directed Gene Therapy	10
Hemophilia Gene Therapy: The End of the Beginning?	10
Retroviral Transduction of Human CD4 ⁺ T Cells with Membrane-Attached IL-10 Generates Type 1-Like Regulatory T Cells	10
Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy	10
Efficient Delivery of Adeno-Associated Virus into Inner Ear In Vivo Through Trans-Stapes Route in Adult Guinea Pig	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
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
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
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
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
CRISPR in Medicine: A Systematic Review of Clinical Trials and Therapeutic Applications	7
Gene Therapy for Immunoglobulin E, Complement-Mediated, and Eosinophilic Disorders	7
Akouos Therapy Restores 11-Year-Old Boy's Hearing After 1 Month	7
FDA Investigating Sarepta’s Elevidys® after Second Patient Dies	7
MPZL1 Promotes Lung Adenocarcinoma Progression by Enhancing Tumor Proliferation, Invasion, Migration, and Suppressing Immune Function via Transforming Growth Factor-β1	7
Quantification of Linear Polyethylenimines in Recombinant Adeno-Associated Virus by High-Performance Liquid Chromatography with Charged Aerosol Detection	7
Clustered Regularly Interspaced Short Palindromic Repeats and Clustered Regularly Interspaced Short Palindromic Repeats–Associated Protein 9 System: Factors Affecting Precision Gene Editing Efficiency	7
Blood Cancers Reported in Seven Children Dosed with SKYSONA™	7
Timely Intervention: Navigating Ethical Challenges in OTOF -Gene Therapy Trials	7
Targeting Oncolytic Adenoviruses to Cancer Cells Using a Designed Ankyrin Repeat Protein Lipocalin-2 Fusion Protein	7
Rosalind Franklin Society Proudly Announces the 2024 Award Recipient for Human Gene Therapy	7
HIV Tat-Conjugated Histone H3 Peptides Induce Tumor Cell Death Via Cellular Stress Responses	7
Genome Editing of Mammalian Cells Through RNA Transcript-Mediated Homologous Recombination Repair	7
Gene Therapy Restores Auditory Functions in an Adult Vglut3 Knockout Mouse Model	7
USP7-SOX9-miR-96-5p-NLRP3 Network Regulates Myocardial Injury and Cardiomyocyte Pyroptosis in Sepsis	7
Remembrances of Kenneth Berns, PhD	6
Interview with Dr. David Williams	6
Long Non-Coding RNAs, Cell Cycle, and Human Breast Cancer	6
Prophylactic Prednisolone Promotes AAV5 Hepatocyte Transduction Through the Novel Mechanism of AAV5 Coreceptor Platelet-Derived Growth Factor Receptor Alpha Upregulation and Innate Immune Suppression	6
AAV-p40 Bioengineering Platform for Variant Selection Based on Transgene Expression	6
Direct Comparison of Epifluorescence and Immunostaining for Assessing Viral Mediated Gene Expression in the Primate Brain	6
The Legal Status and Improvement Path of Human Genetic Data in Gene Therapy in China	6
Systemic Administration of AAV9-G266A with Focused Ultrasound Enables Robust Brain Transduction with Minimal Liver Transduction	6
Heart of the Matter: AAV8 Improves Cardiac Function	6
Construct Optimization Enables Oncolytic Virus-Mediated Functional Membrane Localization of Calreticulin and Macrophage Reprogramming	6
Efficient LAMA1 Gene Activation by Epigenome Editing as a Therapeutic Approach for LAMA2-CMD	6
Immune Responses to Muscle-Directed Adeno-Associated Viral Gene Transfer in Clinical Studies	6
Lipid Nanoparticle-Delivered mRNA Therapy Corrects Neonatal Murine MPS I-H	6
A Review of the Legislation of Direct-to-Consumer Genetic Testing in China	6
Suicide Gene Delivery System Mediated by Ultrasound-Targeted Microbubble Destruction: A Promising Strategy for Cancer Therapy	6

International Conference on Lymphocyte Engineering Author Index	6
Call for Papers: Special Issue on Toxicity And Safety in Clinical AAV Gene Therapy	5
Acknowledgment of Reviewers 2022	5
Remembrances of Nicholas Muzyczka, PhD	5
Evaluation of Purification Methods for Minimizing Transgene Expression Background During Viral Manufacturing	5
Hospital Exemption for Advanced Therapy Medicinal Products in Spain	5
An Engineered Adeno-Associated Virus Variant Enables Efficient Gene Editing in Human T Cells	5
Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease	5
Concise Analysis of Single-Stranded DNA of Recombinant Adeno-Associated Virus By Automated Electrophoresis System	5
Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction	5
Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice	5
Central Nervous System-Targeted Gene Therapy for the Treatment of Neurocognitive Deficits in Mucopolysaccharidosis Type II Mice	5
Autologous Production: The Future of Sustainable Antibody Treatments	5
Developing Gene Therapy for Mitigating Multisystemic Pathology in Fabry Disease: Proof of Concept in an Aggravated Mouse Model	5
Acknowledgment of Reviewers 2023	5
Lentiviral Gene Delivery Rescues Ciliary Defects in Patient-Derived Airway Organoids from Primary Ciliary Dyskinesia	5
Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors	5
An Analysis of Biomarkers for the Evaluation of Gene Therapy in Niemann–Pick Disease Type C1 Mice	5
Bone Marrow Mesenchymal Stem Cell-Derived Exosomes microRNA-31-5p Repress Pulmonary Fibrosis via IGFBP7	5
Vertex, Enlaza Launch Up-to-$2B Collaboration to Improve CASGEVY® Conditioning	5
Creating an Innovation Engine to Advance Medicine for Patients with Rare Diseases	5
The Lived Experience of Pediatric Gene Therapy: A Scoping Review	4
Pharmacokinetic and Pharmacodynamic Evaluation of Bidridistrogene Xeboparvovec in an Aged Murine Model of Limb-Girdle Muscular Dystrophy Type 2E/R4	4
Recombinant Adeno-Associated Virus Vector Mediated Gene Editing in Proliferating and Polarized Cultures of Human Airway Epithelial Cells	4
Regeneron Wins FDA Approval for First Neurosensory Gene Therapy	4
Neonatal Fc Receptor Inhibition Enables Adeno-Associated Virus Gene Therapy Despite Pre-Existing Humoral Immunity	4
Strategies and Advances in Site-Specific Integration of Exogenous Large Genes	4
Letter to the Editor: A Favorable Benefit–Risk Balance Maybe Expected with Replication-Defective Adenovirus-Mediated Interferon Gene Therapy for Cancer Treatment	4
Interview with Dr. Florian Eichler	4
Comparison of Physical Perturbation Devices for Enhancing Lentiviral Vector-Mediated Gene Transfer to the Airway Epithelium	4
Correction to: Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors, by Westhaus et al.	4
How Great a Risk Do You Take? A Qualitative Study Exploring Attitudes of Individuals with Friedreich Ataxia Toward Gene Therapy	4
Verve Pauses Enrollment in Base Editing Trial after Adverse Events	4
Long-Term Disease Prevention with a Gene Therapy Targeting Oligodendrocytes in a Mouse Model of Adrenomyeloneuropathy	4
Improvement of Precision in Recombinant Adeno-Associated Virus Infectious Titer Assay with Droplet Digital PCR as an Endpoint Measurement	4
Understanding and Tackling Immune Responses to Adeno-Associated Viral Vectors	4
Novel Cystic Fibrosis Ferret Model Enables Visualization of CFTR Expression Cells and Genetic CFTR Reactivation	4
Precision BioSciences, Novartis Launch Up-to-$1.5B In Vivo Genome Editing Collaboration	4
FDA Issues Plausible Mechanism Pathway Draft Guidance	4
Quantification of Lentiviral Vectors with Nucleic Acid Dyes	4
First Prime Editing Clinical Trial Expected in 2024	4
Food and Drug Administration Sets Stage for Approval of First Duchenne Muscular Dystrophy Gene Therapy	4
Interlaboratory Measurement of Adeno-Associated Virus: Comparative Quantification of Full and Empty Capsids	4
Immune Responses and Immunosuppressive Strategies for Adeno-Associated Virus-Based Gene Therapy for Treatment of Central Nervous System Disorders: Current Knowledge and Approaches	4
Development of an Inducible, Replication-Competent Assay Cell Line for Titration of Infectious Recombinant Adeno-Associated Virus Vectors	4
Recombinant Adeno-Associated Virus-Mediated Editing of the G551D Cystic Fibrosis Transmembrane Conductance Regulator Mutation in Ferret Airway Basal Cells	4
Comparative Analysis of Induced Pancreatic Stem Cells Generated with Different Factors	4
Cure Rare Disease: An Initiative to Enable N of 1 Gene Editing	4
International Conference on Lymphocyte Engineering September 12–14 2023 Munich, Germany	4
Call for Special Issue Papers: Genetic and Nongenetic Inflammation Networks in Major Human Diseases	4
Progress in Bioengineering of Myotropic Adeno-Associated Viral Gene Therapy Vectors	4
Risk and Benefit Assessment of Gene Therapy with Lentiviral Vectors and Hematopoietic Stem Cells: The Skysona Case	3
Thrombotic Microangiopathy Associated with Systemic Adeno-Associated Virus Gene Transfer: Review of Reported Cases	3
Enhanced Delivery of Ligand-Conjugated Antisense Oligonucleotides (C16-HA-ASO) Targeting Dystrophia Myotonica Protein Kinase Transcripts for the Treatment of Myotonic Dystrophy Type 1	3
Prevalent and Disseminated Recombinant and Wild-Type Adeno-Associated Virus Integration in Macaques and Humans	3
Vectorized Human Antibody-Mediated Anti-Eosinophil Gene Therapy	3
Cyclosporin H Improves the Transduction of CD34 ⁺ Cells with an Anti-Sickling Globin Vector, a Possible Therapeutic Approach for Sickle Cell Disease	3
Prevention of Portal-Tract Fibrosis in Zfyve19 ^−/− Mouse Model with Adeno-As	3
Innate Immune Sensing of Adeno-Associated Virus Vectors	3
Preclinical Safety and Biodistribution in Mice Following Single-Dose Intramuscular Inoculation of Tumor DNA Vaccine by Electroporation	3
Roche, Ascidian Launch Up-to-$1.8B RNA Exon Editing Collaboration	3
Minimally Humanized Ezh2 Exon-18 Mouse Cell Lines Validate Preclinical CRISPR/Cas9 Approach to Treat Weaver Syndrome	3
Genetic Engineering in CAR T Cells for Solid Tumors: Current State, Barriers and Future Developments	3
Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration	3
Prevalence of Anti-Adeno-Associated Virus Serotype 9 Antibodies in Adult Patients with Spinal Muscular Atrophy	3
Kallistatin Improves High-Fat-Induced Insulin Resistance via Epididymal Adipose Tissue-Derived Exosomes	3
Sarepta Pauses, then Resumes, Shipments of DMD Gene Therapy	3
Developing a Gene Therapy for the Treatment of Autosomal Dominant Alzheimer's Disease	3
Letter to the Editor: mRNA Vaccines in Urological Malignancies	3
Correction to: Inhibition of MicroRNA-9-5p Protects Against Cardiac Remodeling Following Myocardial Infarction in Mice, by Xiao et al. Hum Gene Ther	3
Efficient Gene Transfer to Kidney Using a Lentiviral Vector Pseudotyped with Zika Virus Envelope Glycoprotein	3
Beam Results Show First Genetic Correction of Disease-Causing Mutation	3
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.	3
Case Study Cites Immune Reaction to High Adeno-Associated Virus Dose in Explaining Duchenne Muscular Dystrophy Trial Death	3
Patient Dies after Treatment with Intellia CRISPR Therapy in Phase III Trial	3
Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys	3
Cell-Penetrating Peptides and CRISPR-Cas9: A Combined Strategy for Human Genetic Disease Therapy	3
ESGCT 32nd Annual Congress In collaboration with SETGYC Seville, Spain October 7-10, 2025 Abstracts	3
Ablation of Cbl-b in ROBO1 CAR-NK92 Cells Enhances Their Antitumor Efficacy	3
Current Status and Prospects of Viral Vector-Based Gene Therapy to Treat Kidney Diseases	3
Advances in Off-Target Detection for CRISPR-Based Genome Editing	3
Focused Ultrasounds as an Adeno-Associated Virus Gene Therapy-Empowering Tool in Juvenile Mice via Intracerebroventricular Administration	3
Abstract Author Index	3