Cell-Penetrating Peptides and CRISPR-Cas9: A Combined Strategy for Human Genetic Disease Therapy

2024-9-14

Unconstrained precision mitochondrial genome editing with αDdCBEs

2024-8-30

Adeno-associated Virus (AAV) vectors - a target of cellular and humoral immunity – are expanding their reach towards hematopoietic stem cell modification and immunotherapies

2024-8-28

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

2024-8-19

Inhalation of SP-101 followed by inhaled doxorubicin results in robust and durable hCFTRΔR transgene expression in the airways of wild-type and cystic fibrosis ferrets

2024-8-19

Alpharetroviral vector-mediated gene therapy for IL7RA deficient SCID

2024-8-16

Advances in MicroRNA Therapeutics: from Preclinical to Clinical Studies

2024-8-16

Gene editing by ferrying of CRISPR/Cas ribonucleoprotein complexes in enveloped virus-derived particles

2024-8-16

Lipid nanoparticles for nucleic acid delivery beyond the liver

2024-8-14

Gene Editing of the Endogenous Cryptic 3′ Splice Site Corrects the RNA Splicing Defect in the β⁶⁵⁴-Thalassemia Mouse Model

2024-8-13

Roche, Ascidian Launch Up-to-$1.8B RNA Exon Editing Collaboration

2024-8-1

Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article

2024-8-1

Genome editing of mammalian cells through RNA transcript-mediated homologous recombination repair

2024-7-24

Intra-Articular Delivery of an AAV-Anti-TNF-α Vector Alleviates the Progress of Arthritis in a RA Mouse Model

2024-7-24

Matrix protein of vesicular stomatitis virus targets the mitochondria, reprograms glucose metabolism，and sensitizes to 2-deoxyglucose in glioblastoma

2024-7-13

The Legal Status and Improvement Path of Human Genetic Data in Gene Therapy in China

2024-7-13

An improved helper plasmid containing deletions within the E4 and E2a genes results in increased AAV productivity

2024-7-13

Modulation of AAV9 Galactose Binding Yields Novel Gene Therapy Vectors and Predicts Cross-Species Differences in Glycan Avidity

2024-7-13

Lipid nanoparticle mRNA therapy improves survival and reduces serum branched-chain amino acids in mouse models of maple syrup urine disease

2024-7-13

Boy Dosed with Pfizer’s Duchenne Muscular Dystrophy Gene Therapy Dies a Year After Phase II Trial

2024-7-9

Have a little heart (or not): highly minimized skeletal muscle regulatory cassettes with low or no activity in heart

2024-7-6

An Open-Label Phase II Study Assessing the Safety of Bilateral, Sequential Administration of Retinal Gene Therapy in Participants with Choroideremia: The GEMINI Study

2024-7-6

Developing gene therapy for mitigating multisystemic pathology in Fabry disease: proof of concept in an aggravated mouse model

2024-7-6

Innate Immune Sensing of AAV Vectors

2024-7-5

Gene Co-Expression and miRNA Regulation: A Path to Early Intervention in Colorectal Cancer

2024-7-4

AAV5 delivery of CRISPR/Cas9 mediates genome editing in the lungs of young rhesus monkeys

2024-7-3

Pfizer Weighs Next Steps after DMD Therapy Linked to Boy’s Death Fails Phase III Trial

2024-7-1

Role of FoxP3⁺ Regulatory T Cells in Modulating Immune Responses to Adeno-Associated Virus Gene Therapy

2024-7-1

Leveraging CRISPR-Cas9 for Accurate Detection of AAV-Neutralizing Antibodies: The AAV-HDR Method

2024-7-1

Peripheral Cellular Immune Responses Induced by Subretinal AAV Gene Transfer can be Restrained by the Subretinal Associated Immune Inhibition Mechanism

2024-7-1

Insights into Prime Editing Technology: A Deep Dive into Fundamentals, Potentials and Challenges

2024-7-1

Development of an Enzyme-Linked Immunosorbent Spot Assay for the Assessment of Adeno-Associated Virus Peptides to Examine Immune Safety

2024-7-1

A Review of the Cost-Effectiveness Evidence for FDA-Approved Cell and Gene Therapies

2024-6-1

CRISPR-Cas genome editing in ex-vivo human lungs to rewire the translational path of genome-targeting therapeutics

2024-6-1

Methodological validation of sedimentation velocity analytical ultracentrifugation method for Adeno-Associated Virus and collaborative calibration of system suitability substance

2024-5-8

Verve Pauses Enrollment in Base Editing Trial after Adverse Events

2024-5-1

Advances in bone-targeting drug delivery: Emerging strategies using adeno-associated virus

2024-4-25

Intravitreal delivery of rAAV2-hSyn-hRS1 results in Retinal Ganglion Cell-specific gene expression and retinal improvement in the Rs1-KO mouse

2024-4-25

Prevalence study of cellular capsid-specific immune responses to AAV2, 4, 5, 8, 9 and rh10 in healthy donors

2024-4-6

Remembrances of Kenneth Berns, PhD

2024-4-1

Orchard Therapeutics Gains First U.S. Approval for a Metachromatic Leukodystrophy Gene Therapy

2024-4-1

Establishment of the Effectiveness of Early Versus Late Stem Cell Gene Therapy in Mucopolysaccharidosis II for Treating Central Versus Peripheral Disease

2024-4-1

<i>Ex Vivo</i> Gene Therapy in Organ Transplantation: Considerations and Clinical Translation

2024-4-1

Multidimensional Response Surface Methodology for the Development of a Gene Editing Protocol for p67^phox-Deficient Chronic Granulomatous Disease

2024-4-1

Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage

2024-4-1

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-Mediated Gene Transfer

2024-3-28

Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases

2024-3-27

<i>Correction to:</i> Efficacy and Safety of Adeno-Associated Virus-Based Clinical Gene Therapy for Hemophilia: A Systematic Review and Meta-Analysis, by Han et al. <i>Hum Gene Ther</i> 2024;35(3-4):93–103; doi: 10.1089/hum.2023.208.

2024-3-25

Knockout and Replacement Gene Surgery to Treat Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa

2024-3-1

Combination Immunotherapy of Oncolytic Flu-Vectored Virus and Programmed Cell Death 1 Blockade Enhances Antitumor Activity in Hepatocellular Carcinoma

2024-3-1