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 2022-08-01 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
AAV.hBAG3 Gene Therapy Improves Phenotype in a Valosin Containing Protein Mouse Model of Hereditary Inclusion Body Myositis	35
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
Toxicity and Biodistribution of the Oncolytic Virus VCN-01 Following Intracranial Injection in Syrian Hamsters	33
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
Abstracts	32
uniQure Gene Therapy Significantly Slows Huntington Disease Progression	32
Genome Editing of Pik3cd Impedes Abnormal Retinal Angiogenesis	30
Characterizing Complex Populations of Endogenous Adeno-Associated Viruses by Single-Genome Amplification	28
Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop	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
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
Interview with Manlio Fusciello, PhD	23
Meta-Analysis and Optimization of the In Vitro Immortalization Assay for Safety Assessment of Retroviral Vectors in Gene Therapy	22
Modulation of AAV9 Galactose Binding Yields Novel Gene Therapy Vectors and Predicts Cross-Species Differences in Glycan Avidity	22
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
Quantitation of Residual Host Cell DNA in Recombinant Adeno-Associated Virus Using Droplet Digital Polymerase Chain Reaction	21
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
Metagenomic Reconstruction of Adeno-Associated Virus Genomes	20
Lived Experience with Gene Therapy	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
Interview with Dr. Ronald Crystal	18
Lipid Nanoparticles for Nucleic Acid Delivery Beyond the Liver	18
Patient Dies in Beam Trial of Sickle Cell Disease Candidate; Company Cites Conditioning	17
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
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
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
A Recombinant Oncolytic Influenza Virus Carrying GV1001 Triggers an Antitumor Immune Response	15
An Optimized CRISPR/Cas9 Adenovirus Vector (AdZ-CRISPR) for High-Throughput Cloning of sgRNA, Using Enhanced sgRNA and Cas9 Variants	15
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
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
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
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
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
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
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
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
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
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
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
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
AAV-Mediated Gene Transfer of WDR45 Corrects Neurological Deficits in the Mouse Model of Beta-Propeller Protein-Associated Neurodegeneration	7
HIV Tat-Conjugated Histone H3 Peptides Induce Tumor Cell Death Via Cellular Stress Responses	7
Medicinal Products Based on Adeno-Associated Viral Vectors: A Regulatory Perspective on the Potential Risk of Insertion-Mediated Tumorigenesis	7
ESGCT 29th Annual Congress In collaboration with BSGCT Edinburgh, UK October 11–14, 2022 Abstracts	7
Celyvir: Oncolytic Virotherapy with Mesenchymal Stromal Cells. From Concept to Clinical Translation	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
Targeting Oncolytic Adenoviruses to Cancer Cells Using a Designed Ankyrin Repeat Protein Lipocalin-2 Fusion Protein	7
Lilly, Ascidian Launch Up-to-$1.9B RNA Exon Editor Collaboration	7
Rosalind Franklin Society Proudly Announces the 2024 Award Recipient for Human Gene Therapy	7
Comprehensive Review of Osteogenesis Imperfecta: Current Treatments and Future Innovations	7
A Review of the Challenge of Pre-Existing Humoral Immunity in Adeno-Associated Virus Gene Therapy and Potential Solutions	7
Quantification of Linear Polyethylenimines in Recombinant Adeno-Associated Virus by High-Performance Liquid Chromatography with Charged Aerosol Detection	7
Genome Editing of Mammalian Cells Through RNA Transcript-Mediated Homologous Recombination Repair	7
Blood Cancers Reported in Seven Children Dosed with SKYSONA™	7
The 2024 Nobel Prize: Impact of the Discovery of miRNA on the Field of Gene Therapy	7
Intracisternal AAV9-MAG- hABCD1 Vector Reverses Motor Deficits in Adult Adrenomyeloneuropathy Mice	7
SG33, a Vaccine Strain of Myxoma Virus with Oncolytic Potential, Exploits Macropinocytosis and Clathrin-Mediated Endocytosis for Entry into Pancreatic Cancer Cells	7
Ultragenyx Gene Therapies Spark Lawsuit from Lacks Family	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
Timely Intervention: Navigating Ethical Challenges in OTOF -Gene Therapy Trials	6
Construct Optimization Enables Oncolytic Virus-Mediated Functional Membrane Localization of Calreticulin and Macrophage Reprogramming	6
Systemic Administration of AAV9-G266A with Focused Ultrasound Enables Robust Brain Transduction with Minimal Liver Transduction	6
Remembrances of Kenneth Berns, PhD	6
Long Non-Coding RNAs, Cell Cycle, and Human Breast Cancer	6
International Conference on Lymphocyte Engineering Author Index	6
MPZL1 Promotes Lung Adenocarcinoma Progression by Enhancing Tumor Proliferation, Invasion, Migration, and Suppressing Immune Function via Transforming Growth Factor-β1	6
Efficient LAMA1 Gene Activation by Epigenome Editing as a Therapeutic Approach for LAMA2-CMD	6
FDA Investigating Sarepta’s Elevidys® after Second Patient Dies	6
Direct Comparison of Epifluorescence and Immunostaining for Assessing Viral Mediated Gene Expression in the Primate Brain	6
Immune Responses to Muscle-Directed Adeno-Associated Viral Gene Transfer in Clinical Studies	6
Concise Analysis of Single-Stranded DNA of Recombinant Adeno-Associated Virus By Automated Electrophoresis System	6
CRISPR in Medicine: A Systematic Review of Clinical Trials and Therapeutic Applications	6
Lipid Nanoparticle-Delivered mRNA Therapy Corrects Neonatal Murine MPS I-H	6

Interview with Dr. David Williams	6
The Legal Status and Improvement Path of Human Genetic Data in Gene Therapy in China	6
USP7-SOX9-miR-96-5p-NLRP3 Network Regulates Myocardial Injury and Cardiomyocyte Pyroptosis in Sepsis	6
A Review of the Legislation of Direct-to-Consumer Genetic Testing in China	6
Long-Term Functional Correction of Pompe Disease and Increased α-Glucosidase Expression after Gene Therapy with Novel Combinations of Muscle-Targeted Transcriptional Cis	6
Call for Papers: Special Issue on Toxicity And Safety in Clinical AAV Gene Therapy	5
Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease	5
Hospital Exemption for Advanced Therapy Medicinal Products in Spain	5
Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors	5
Autologous Production: The Future of Sustainable Antibody Treatments	5
Vertex, Enlaza Launch Up-to-$2B Collaboration to Improve CASGEVY® Conditioning	5
Central Nervous System-Targeted Gene Therapy for the Treatment of Neurocognitive Deficits in Mucopolysaccharidosis Type II Mice	5
Suicide Gene Delivery System Mediated by Ultrasound-Targeted Microbubble Destruction: A Promising Strategy for Cancer Therapy	5
Lentiviral Gene Delivery Rescues Ciliary Defects in Patient-Derived Airway Organoids from Primary Ciliary Dyskinesia	5
Acknowledgment of Reviewers 2022	5
High Prevalence of Preexisting Neutralizing Antibodies Against Adeno-Associated Virus Capsids in Mongolian People with Hemophilia	5
Remembrances of Nicholas Muzyczka, PhD	5
Acknowledgment of Reviewers 2023	5
Creating an Innovation Engine to Advance Medicine for Patients with Rare Diseases	5
Developing Gene Therapy for Mitigating Multisystemic Pathology in Fabry Disease: Proof of Concept in an Aggravated Mouse Model	5
An Analysis of Biomarkers for the Evaluation of Gene Therapy in Niemann–Pick Disease Type C1 Mice	5
Heart of the Matter: AAV8 Improves Cardiac Function	5
Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice	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
Prophylactic Prednisolone Promotes AAV5 Hepatocyte Transduction Through the Novel Mechanism of AAV5 Coreceptor Platelet-Derived Growth Factor Receptor Alpha Upregulation and Innate Immune Suppression	5
Development of an Inducible, Replication-Competent Assay Cell Line for Titration of Infectious Recombinant Adeno-Associated Virus Vectors	5
An Engineered Adeno-Associated Virus Variant Enables Efficient Gene Editing in Human T Cells	5
Evaluation of Purification Methods for Minimizing Transgene Expression Background During Viral Manufacturing	5
Regeneron Wins FDA Approval for First Neurosensory Gene Therapy	4
Recombinant Adeno-Associated Virus Vector Mediated Gene Editing in Proliferating and Polarized Cultures of Human Airway Epithelial Cells	4
Verve Pauses Enrollment in Base Editing Trial after Adverse Events	4
How Great a Risk Do You Take? A Qualitative Study Exploring Attitudes of Individuals with Friedreich Ataxia Toward Gene Therapy	4
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
Strategies and Advances in Site-Specific Integration of Exogenous Large Genes	4
Neonatal Fc Receptor Inhibition Enables Adeno-Associated Virus Gene Therapy Despite Pre-Existing Humoral Immunity	4
Understanding and Tackling Immune Responses to Adeno-Associated Viral Vectors	4
FDA Issues Plausible Mechanism Pathway Draft Guidance	4
Comparative Analysis of Induced Pancreatic Stem Cells Generated with Different Factors	4
Precision BioSciences, Novartis Launch Up-to-$1.5B In Vivo Genome Editing Collaboration	4
Food and Drug Administration Sets Stage for Approval of First Duchenne Muscular Dystrophy Gene Therapy	4
Letter to the Editor: A Favorable Benefit–Risk Balance Maybe Expected with Replication-Defective Adenovirus-Mediated Interferon Gene Therapy for Cancer Treatment	4
Progress in Bioengineering of Myotropic Adeno-Associated Viral Gene Therapy Vectors	4
Improvement of Precision in Recombinant Adeno-Associated Virus Infectious Titer Assay with Droplet Digital PCR as an Endpoint Measurement	4
Bone Marrow Mesenchymal Stem Cell-Derived Exosomes microRNA-31-5p Repress Pulmonary Fibrosis via IGFBP7	4
Novel Cystic Fibrosis Ferret Model Enables Visualization of CFTR Expression Cells and Genetic CFTR Reactivation	4
CAR-T Cell Therapy: Manufacturing Platforms and Clinical Consequences	4
Call for Special Issue Papers: Genetic and Nongenetic Inflammation Networks in Major Human Diseases	4
First Prime Editing Clinical Trial Expected in 2024	4
Letter: Exon 19 of the GNPTAB Gene is Indispensable for the Catalytic Activity of the Enzyme	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
Recombinant Adeno-Associated Virus-Mediated Editing of the G551D Cystic Fibrosis Transmembrane Conductance Regulator Mutation in Ferret Airway Basal Cells	4
ESGCT 32nd Annual Congress In collaboration with SETGYC Seville, Spain October 7-10, 2025 Abstracts	3
International Conference on Lymphocyte Engineering September 12–14 2023 Munich, Germany	3
Interlaboratory Measurement of Adeno-Associated Virus: Comparative Quantification of Full and Empty Capsids	3
Cell-Penetrating Peptides and CRISPR-Cas9: A Combined Strategy for Human Genetic Disease Therapy	3
Dichotomies in Ex Vivo and In Vivo Performance of Receptor-Binding Mutants of Adeno-Associated Virus Vecto	3
Risk and Benefit Assessment of Gene Therapy with Lentiviral Vectors and Hematopoietic Stem Cells: The Skysona Case	3
Prevalence of Anti-Adeno-Associated Virus Serotype 9 Antibodies in Adult Patients with Spinal Muscular Atrophy	3
Focused Ultrasounds as an Adeno-Associated Virus Gene Therapy-Empowering Tool in Juvenile Mice via Intracerebroventricular Administration	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
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.	3
Cure Rare Disease: An Initiative to Enable N of 1 Gene Editing	3
Comparison of Physical Perturbation Devices for Enhancing Lentiviral Vector-Mediated Gene Transfer to the Airway Epithelium	3
Nonhuman Primate Genomes Have Limited Relevance for Evaluating Human CRISPR Off-Target Effects	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
Genetic Engineering in CAR T Cells for Solid Tumors: Current State, Barriers and Future Developments	3
Prevention of Portal-Tract Fibrosis in Zfyve19 ^−/− Mouse Model with Adeno-As	3
Quantification of Lentiviral Vectors with Nucleic Acid Dyes	3
Cyclosporin H Improves the Transduction of CD34 ⁺ Cells with an Anti-Sickling Globin Vector, a Possible Therapeutic Approach for Sickle Cell Disease	3
Long-Term Disease Prevention with a Gene Therapy Targeting Oligodendrocytes in a Mouse Model of Adrenomyeloneuropathy	3
Ablation of Cbl-b in ROBO1 CAR-NK92 Cells Enhances Their Antitumor Efficacy	3
Interview with Dr. Florian Eichler	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
Thrombotic Microangiopathy Associated with Systemic Adeno-Associated Virus Gene Transfer: Review of Reported Cases	3
Patient Dies after Treatment with Intellia CRISPR Therapy in Phase III Trial	3
Current Status and Prospects of Viral Vector-Based Gene Therapy to Treat Kidney Diseases	3
Developing a Gene Therapy for the Treatment of Autosomal Dominant Alzheimer's Disease	3
Vectorized Human Antibody-Mediated Anti-Eosinophil Gene Therapy	3